Fiche société
University of California, San Francisco
Traitements, essais et publications liés.
Traitements33programmes
Essais17liés
Publications13liées
SourceDBlocale
Traitements
33| Molécule | Indication / population | Phase | Objectif | Pays | Résultat |
|---|---|---|---|---|---|
| Brain Development Research ProgramDr. Elliott Sherr and his collaborators at University of California, San Francisco (UCSF) are studying the genetic causes of disorders of cognition and epilepsy, in particular disorders of brain development that affect the corpus callosum, such as Aicardi syndrome, as well as two additional brain malformations, polymicrogyria and Dandy-Walker malformation. The goal of the investigators' research is to use a better understanding of the underlying genetic causes as a foundation to develop better treatments for these groups of patients. | Épilepsie | À vérifier | À vérifier | United States | À vérifier |
| ClemastineThe main purpose of this study is to assess clemastine as a remyelinating agent in patients with relapsing forms of multiple sclerosis. The study will also evaluate the tolerability of clemastine, originally approved as first-generation antihistamine, in patients with multiple sclerosis. Study procedures will include assessments for evidence of remyelination in the anterior visual pathway and in the brain using electrophysiologic techniques and magnetic resonance imaging. The study will also assess the robustness and stability of this clinical effect in patients taking clemastine for up to 3 months. Patients in this study can remain on their standard disease modifying treatment during the course of the study. However, patients cannot participate in any other investigational new drug research study concurrently. | SEP | Phase 2 | Remyélinisation | United States | À vérifier |
| Clemastine FumarateThe clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis. Clemastine fumarate was identified along with a series of other antimuscarinic medications as a potential remyelinating agent using the micropillar screen (BIMA) developed at the University of California, San Francisco (UCSF). Following in vivo validation, an FDA IND exemption was granted to investigate clemastine for the treatment of multiple sclerosis in the context of chronic optic neuropathy. That pilot study was recently completed and is the first randomized control trial documenting efficacy for a putative remyelinating agent for the treatment of MS. The preselected primary efficacy endpoint (visual evoked potential) was met and a strong trend to benefit was seen for the principal secondary endpoint assessing function (low contrast visual acuity). That trial number was 13-11577. This study seeks to follow up on that study and examine clemastine fumarate's protective and reparative effects in the context of chronic demyelinating brain lesions as imaged by multi-parametric MRI assessments. The investigators will be assessing the effects of clemastine fumarate as a remyelinating therapy and assessing its effect on MRI metrics of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis. In addition to using conventional multi-parametric MRI assessments, this study will also evaluate a new MRI technique called Ultrashort Echo Time (UTE) MRI to assess the effects of clemastine fumarate as a remyelinating therapy of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis and compare it to the other assessments. | SEP | Phase 1/2 | Remyélinisation | United States | À vérifier |
| CNKSR2 Natural History StudyThis prospective natural history study is being conducted to define the electroclinical, neurodevelopmental, and behavioral characteristics of CNKSR2 epilepsy aphasia syndrome (EAS) and intellectual disability (ID) in children aged 6 to 21 years old with CNKSR2 mutations. The data collected from this study will serve as an external control to eventual clinical trials examining precision medicine investigational therapeutics that aim to improve the seizure burden and neurodevelopmental outcomes in patients with CNKSR2 EAS/ID. | Épilepsie | À vérifier | À vérifier | United States | À vérifier |
| IL1 Inhibition in FOPThis is an observational pre-post study to observe if the off label use of anti-IL1 therapies, such as anakinra or canakinumab, can block ACVR1-induced flare activity and heterotopic ossification in FOP. It will also generate key tools and preliminary data that are needed to design a future Phase II study. This study specifically focuses on patients with severe FOP who are being considered by their medical team for rescue therapy with anti-IL1 therapy. Preliminary data suggests patients experience significant decreases in flare frequency when taking anti-IL1 therapy, but other measures of efficacy remain unassessed, such as changes in heterotopic ossification formation, changes in pain medication use, and changes in functionality. | Myopathies | À vérifier | Traitement symptomatique | United States | À vérifier |
| Laser Interstitial Thermal Therapy (LITT)This study aims to evaluate the efficacy of Laser Interstitial Thermal Therapy (LITT) in treating recurrent or progressive, low-grade gliomas (LGG) in pediatric, adolescent and young adult patients. | Cancer | Non applicable | À vérifier | United States | À vérifier |
| Magnetic resonance imaging (MRI)This study aims to evaluate the efficacy of Laser Interstitial Thermal Therapy (LITT) in treating recurrent or progressive, low-grade gliomas (LGG) in pediatric, adolescent and young adult patients. | Cancer | Non applicable | À vérifier | United States | À vérifier |
| Medtronic Percept Deep Brain Stimulation (adaptive DBS)Parkinson's disease (PD) affects \~1% of people over 60 years old, is highly disabling and represents a large economic burden. While dopaminergic medications effectively treat motor symptoms early in the disease, most patients develop complications, including motor fluctuations and dyskinesias, which can be partially managed by deep brain stimulation (DBS). This surgical therapy consists of delivering continuous electrical stimulation through electrodes permanently implanted in basal ganglia nuclei, with a pulse generator and battery unit implanted in the chest. However, conventional DBS therapy is delivered with constant stimulation parameters, referred to as constant deep brain stimulation (cDBS), that are unresponsive to patient activities or to variations in the severity of symptoms during daily life. This leaves many patients under- or over-stimulated during parts of the day. To address the shortcomings of cDBS, adaptive DBS (aDBS) uses real-time detection of neural signals to automatically adjust stimulation amplitude or other parameters in response to patients' dynamic clinical needs. aDBS was approved by the U.S. Food and Drugs Administration (FDA) for clinical treatment of PD in the Percept PC and RC (Medtronic) device in February 2025. Fully leveraging this therapy in the real world is limited by technical challenges, in particular the fact that: while the investigators developed a consistent pipeline for implementing aDBS, there were several critical control parameters that strongly influenced algorithm performance and required prolonged trial-and-error based testing, to achieve successful control. In this new study, the investigators seek to significantly extend this work and address the major barriers to widespread, easy adoption of aDBS by groups without specialized knowledge of neurophysiology or feedback control. Here the investigators aim to test an automated, data-driven pipeline for the recommendation of the adaptive control parameters. | Parkinson | Non applicable | Traitement symptomatique | United States | À vérifier |
| Medtronic Percept Deep Brain Stimulation (cDBS)Parkinson's disease (PD) affects \~1% of people over 60 years old, is highly disabling and represents a large economic burden. While dopaminergic medications effectively treat motor symptoms early in the disease, most patients develop complications, including motor fluctuations and dyskinesias, which can be partially managed by deep brain stimulation (DBS). This surgical therapy consists of delivering continuous electrical stimulation through electrodes permanently implanted in basal ganglia nuclei, with a pulse generator and battery unit implanted in the chest. However, conventional DBS therapy is delivered with constant stimulation parameters, referred to as constant deep brain stimulation (cDBS), that are unresponsive to patient activities or to variations in the severity of symptoms during daily life. This leaves many patients under- or over-stimulated during parts of the day. To address the shortcomings of cDBS, adaptive DBS (aDBS) uses real-time detection of neural signals to automatically adjust stimulation amplitude or other parameters in response to patients' dynamic clinical needs. aDBS was approved by the U.S. Food and Drugs Administration (FDA) for clinical treatment of PD in the Percept PC and RC (Medtronic) device in February 2025. Fully leveraging this therapy in the real world is limited by technical challenges, in particular the fact that: while the investigators developed a consistent pipeline for implementing aDBS, there were several critical control parameters that strongly influenced algorithm performance and required prolonged trial-and-error based testing, to achieve successful control. In this new study, the investigators seek to significantly extend this work and address the major barriers to widespread, easy adoption of aDBS by groups without specialized knowledge of neurophysiology or feedback control. Here the investigators aim to test an automated, data-driven pipeline for the recommendation of the adaptive control parameters. | Parkinson | Non applicable | Traitement symptomatique | United States | À vérifier |
| Medtronic Percept Deep Brain Stimulation (daytime aDBS)The purpose of this study is to test a new way to treat Parkinson's disease (PD). Subjects will be implanted with deep brain stimulator (DBS) devices and electrodes placed under the scalp. The main questions it aims to answer are: * Is there a less invasive method to collect useful brain signals? Find out if these brain signals can be related to movement and/or sleep symptoms. * How to use these brain signals to tailor adaptive deep brain stimulation settings for movement and/or sleep symptoms Researchers will compare study derived adaptive DBS settings to subject's clinically programmed continuous DBS settings to see which is better at treating patients PD symptoms. | Parkinson | Non applicable | Traitement symptomatique | United States | À vérifier |
| Medtronic Percept Deep Brain Stimulation (nighttime aDBS)The purpose of this study is to test a new way to treat Parkinson's disease (PD). Subjects will be implanted with deep brain stimulator (DBS) devices and electrodes placed under the scalp. The main questions it aims to answer are: * Is there a less invasive method to collect useful brain signals? Find out if these brain signals can be related to movement and/or sleep symptoms. * How to use these brain signals to tailor adaptive deep brain stimulation settings for movement and/or sleep symptoms Researchers will compare study derived adaptive DBS settings to subject's clinically programmed continuous DBS settings to see which is better at treating patients PD symptoms. | Parkinson | Non applicable | Traitement symptomatique | United States | À vérifier |
| MS FIT — Multiple Sclerosis Falls Insight TrackThe purpose of this study is to develop an application: MS Falls Insight Track (MS FIT) which allows patients to log their falls and near falls, view their MS relevant data and responses to the clinic intake survey as well as communicate with their care team about falls and receive educational material on falls prevention. | SEP | Non applicable | À vérifier | United States | À vérifier |
| MS-CATCH — A Closed Loop, Doctor to Patient, Mobile Application for Depression in People With Multiple SclerosisThe researchers want to find out if an electronic application called MS CATCH can enhance patients' and doctors' experiences during and in between clinical visits. MS CATCH is a smartphone-based tool which allows patients to enter their mood related symptoms at regular intervals, which is then available to their Neurologist in their electronic medical record. The neurologist is also able to view additional information from their medical record, and receives alerts for changes reported by the patient that raise concern for the patient's mental health. | SEP | Non applicable | Traitement symptomatique | United States | À vérifier |
| N-acetyl cysteineThis is a 4-week randomized, placebo-controlled, parallel group, double-blind, single center trial on effect of N-acetyl cysteine versus placebo on fatigue in patients with progressive MS defined by McDonald criteria. Subjects who enter the treatment phase of study, will be randomly assigned to either N-acetyl cysteine (1250 mg three times a day) or placebo (three times a day) for 4 weeks. There will be 3 in-person study visits (screening, baseline, and week 4) and 2 visits over the phone (week 2, and week 6 which is 2 weeks after completing last study drug dose). Visits will all occur in the morning to maximize consistency of assessments and evaluate main outcomes within 2 hours of morning dose of study medication. Fatigue questionnaires, and research samples will be obtained before neurological examination, or magnetic resonance imaging. Research blood draws will be obtained just after fatigue questionnaire completion. Brain spectroscopy will be obtained less than 2 hours after morning dose of study drug to maximize detection of the biological effect of study medication. | SEP | Phase 2 | Traitement symptomatique | United States | À vérifier |
| Pregnancy Registry, Infants, Serum/Milk Analysis (PRISMA)PRISMA, is a pregnancy registry study, focused on comprehensively collecting information about pregnancy in women with chronic neurological conditions from across the United States and internationally. Depending on their specific condition (MS, CIS, NMOSD, or other) and their specific treatment, participants will be asked to contribute to different aspects of the study. (1) The biosamples will be blood, breast milk, infant stool, maternal stool and vaginal swab samples, collected at specific time points. (2) The online surveys will be collected at specific time points. All study activities will be discussed with participants upon enrollment. By collecting this information, the investigators hope to gain deeper insights into the relationship between pregnancy, the neurological condition, and maternal and infant health. For example, one of the sub-studies focuses on breast milk collection for women planning postpartum treatment with Ocrevus, Rituxan, Briumvi or Kesimpta. This study is fully remote and all sample collection is optional, so participants can choose which types of samples they wish to provide. For blood draws, participants can schedule a home visit through ExamOne, making participation even more convenient. The investigators aim to enroll women with chronic neurological conditions who are planning pregnancy, currently pregnant, or within one year postpartum. | Myasthénie, SEP | À vérifier | À vérifier | United States | À vérifier |
| Prospective Registry of Histotripsy for Soft Tissue TumorsUltrasons focalisés produisant une cavitation contrôlée et une destruction mécanique non thermique du tissu tumoral. | Cancer | À vérifier | Ablation tumorale non thermique | United States | Dispositif/procédure : résultats à interpréter par essai et indication. |
| RifaximinThe clinical phenotype of Parkinson's disease (PD) is quite variable, as is the response to and side effects from medications. While many patients respond to carbidopa/levodopa early on, motor fluctuations and dyskinesias can become a problem as the condition progresses, causing significant impairment in function and quality of life. The gut microbiome is of increasing interest in PD, potentially contributing to pathophysiology and clinical phenotype. Furthermore, gut bacteria are capable of metabolizing levodopa, which may decrease its ability to reach the central nervous system and could explain the variable effect seen clinically. Altering the population of drug-metabolizing bacteria could improve the clinical symptoms of PD and the benefit seen with medications. The investigators hypothesize that the gut microbiome in people with PD correlates with their phenotypic characteristics, which can be improved with targeting the microbiome through dietary or therapeutic interventions. The investigators propose a two-part clinical trial. First, a cross-sectional analysis will correlate the microbiome profile with (a) the clinical phenotype of PD and (b) medication response. Second, a randomized, controlled trial, will evaluate the effect of microbiome manipulation on clinical phenotype and medication response. The investigators plan to reduce the level of bacteria through antibiotic use, resetting the potentially disadvantageous microbiome population. Outcomes will include changes in clinical symptoms, alterations in the the microbiome, and changes in serum markers of inflammation. This thorough characterization will broaden our understanding of the gut-brain axis significantly in PD in clinically relevant ways that have yet to be explored. | Parkinson | Phase 1 | Immunomodulation + Traitement symptomatique | United States | À vérifier |
| Pregnancy Registry, Infants, Serum/Milk Analysis (PRISMA)PRISMA, is a pregnancy registry study, focused on comprehensively collecting information about pregnancy in women with chronic neurological conditions from across the United States and internationally. Depending on their specific condition (MS, CIS, NMOSD, or other) and their specific treatment, participants will be asked to contribute to different aspects of the study. (1) The biosamples will be blood, breast milk, infant stool, maternal stool and vaginal swab samples, collected at specific time points. (2) The online surveys will be collected at specific time points. All study activities will be discussed with participants upon enrollment. By collecting this information, the investigators hope to gain deeper insights into the relationship between pregnancy, the neurological condition, and maternal and infant health. For example, one of the sub-studies focuses on breast milk collection for women planning postpartum treatment with Ocrevus, Rituxan, Briumvi or Kesimpta. This study is fully remote and all sample collection is optional, so participants can choose which types of samples they wish to provide. For blood draws, participants can schedule a home visit through ExamOne, making participation even more convenient. The investigators aim to enroll women with chronic neurological conditions who are planning pregnancy, currently pregnant, or within one year postpartum. | Myasthénie | À vérifier | À vérifier | United States | À vérifier |
| RifaximinThe clinical phenotype of Parkinson's disease (PD) is quite variable, as is the response to and side effects from medications. While many patients respond to carbidopa/levodopa early on, motor fluctuations and dyskinesias can become a problem as the condition progresses, causing significant impairment in function and quality of life. The gut microbiome is of increasing interest in PD, potentially contributing to pathophysiology and clinical phenotype. Furthermore, gut bacteria are capable of metabolizing levodopa, which may decrease its ability to reach the central nervous system and could explain the variable effect seen clinically. Altering the population of drug-metabolizing bacteria could improve the clinical symptoms of PD and the benefit seen with medications. The investigators hypothesize that the gut microbiome in people with PD correlates with their phenotypic characteristics, which can be improved with targeting the microbiome through dietary or therapeutic interventions. The investigators propose a two-part clinical trial. First, a cross-sectional analysis will correlate the microbiome profile with (a) the clinical phenotype of PD and (b) medication response. Second, a randomized, controlled trial, will evaluate the effect of microbiome manipulation on clinical phenotype and medication response. The investigators plan to reduce the level of bacteria through antibiotic use, resetting the potentially disadvantageous microbiome population. Outcomes will include changes in clinical symptoms, alterations in the the microbiome, and changes in serum markers of inflammation. This thorough characterization will broaden our understanding of the gut-brain axis significantly in PD in clinically relevant ways that have yet to be explored. | Parkinson | Phase 1 | Immunomodulation + Traitement symptomatique | United States | À vérifier |
| MS FIT — Multiple Sclerosis Falls Insight TrackThe purpose of this study is to develop an application: MS Falls Insight Track (MS FIT) which allows patients to log their falls and near falls, view their MS relevant data and responses to the clinic intake survey as well as communicate with their care team about falls and receive educational material on falls prevention. | SEP | Non applicable | À vérifier | United States | À vérifier |
| Clemastine FumarateThe clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis. Clemastine fumarate was identified along with a series of other antimuscarinic medications as a potential remyelinating agent using the micropillar screen (BIMA) developed at the University of California, San Francisco (UCSF). Following in vivo validation, an FDA IND exemption was granted to investigate clemastine for the treatment of multiple sclerosis in the context of chronic optic neuropathy. That pilot study was recently completed and is the first randomized control trial documenting efficacy for a putative remyelinating agent for the treatment of MS. The preselected primary efficacy endpoint (visual evoked potential) was met and a strong trend to benefit was seen for the principal secondary endpoint assessing function (low contrast visual acuity). That trial number was 13-11577. This study seeks to follow up on that study and examine clemastine fumarate's protective and reparative effects in the context of chronic demyelinating brain lesions as imaged by multi-parametric MRI assessments. The investigators will be assessing the effects of clemastine fumarate as a remyelinating therapy and assessing its effect on MRI metrics of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis. In addition to using conventional multi-parametric MRI assessments, this study will also evaluate a new MRI technique called Ultrashort Echo Time (UTE) MRI to assess the effects of clemastine fumarate as a remyelinating therapy of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis and compare it to the other assessments. | SEP | Phase 1 | Remyélinisation | United States | À vérifier |
| CNKSR2 Natural History StudyThis prospective natural history study is being conducted to define the electroclinical, neurodevelopmental, and behavioral characteristics of CNKSR2 epilepsy aphasia syndrome (EAS) and intellectual disability (ID) in children aged 6 to 21 years old with CNKSR2 mutations. The data collected from this study will serve as an external control to eventual clinical trials examining precision medicine investigational therapeutics that aim to improve the seizure burden and neurodevelopmental outcomes in patients with CNKSR2 EAS/ID. | Épilepsie | À vérifier | À vérifier | United States | À vérifier |
| IL1 Inhibition in FOPThis is an observational pre-post study to observe if the off label use of anti-IL1 therapies, such as anakinra or canakinumab, can block ACVR1-induced flare activity and heterotopic ossification in FOP. It will also generate key tools and preliminary data that are needed to design a future Phase II study. This study specifically focuses on patients with severe FOP who are being considered by their medical team for rescue therapy with anti-IL1 therapy. Preliminary data suggests patients experience significant decreases in flare frequency when taking anti-IL1 therapy, but other measures of efficacy remain unassessed, such as changes in heterotopic ossification formation, changes in pain medication use, and changes in functionality. | Myopathies | À vérifier | Traitement symptomatique | United States | À vérifier |
| Prospective Registry of Histotripsy for Soft Tissue TumorsUltrasons focalisés produisant une cavitation contrôlée et une destruction mécanique non thermique du tissu tumoral. | Cancer | À vérifier | Ablation tumorale non thermique | United States | Dispositif/procédure : résultats à interpréter par essai et indication. |
| N-acetyl cysteineThis is a 4-week randomized, placebo-controlled, parallel group, double-blind, single center trial on effect of N-acetyl cysteine versus placebo on fatigue in patients with progressive MS defined by McDonald criteria. Subjects who enter the treatment phase of study, will be randomly assigned to either N-acetyl cysteine (1250 mg three times a day) or placebo (three times a day) for 4 weeks. There will be 3 in-person study visits (screening, baseline, and week 4) and 2 visits over the phone (week 2, and week 6 which is 2 weeks after completing last study drug dose). Visits will all occur in the morning to maximize consistency of assessments and evaluate main outcomes within 2 hours of morning dose of study medication. Fatigue questionnaires, and research samples will be obtained before neurological examination, or magnetic resonance imaging. Research blood draws will be obtained just after fatigue questionnaire completion. Brain spectroscopy will be obtained less than 2 hours after morning dose of study drug to maximize detection of the biological effect of study medication. | SEP | Phase 2 | Traitement symptomatique | United States | À vérifier |
| Clemastine FumarateThe clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis. Clemastine fumarate was identified along with a series of other antimuscarinic medications as a potential remyelinating agent using the micropillar screen (BIMA) developed at the University of California, San Francisco (UCSF). Following in vivo validation, an FDA IND exemption was granted to investigate clemastine for the treatment of multiple sclerosis in the context of chronic optic neuropathy. That pilot study was recently completed and is the first randomized control trial documenting efficacy for a putative remyelinating agent for the treatment of MS. The preselected primary efficacy endpoint (visual evoked potential) was met and a strong trend to benefit was seen for the principal secondary endpoint assessing function (low contrast visual acuity). That trial number was 13-11577. This study seeks to follow up on that study and examine clemastine fumarate's protective and reparative effects in the context of chronic demyelinating brain lesions as imaged by multi-parametric MRI assessments. The investigators will be assessing the effects of clemastine fumarate as a remyelinating therapy and assessing its effect on MRI metrics of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis. In addition to using conventional multi-parametric MRI assessments, this study will also evaluate a new MRI technique called Ultrashort Echo Time (UTE) MRI to assess the effects of clemastine fumarate as a remyelinating therapy of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis and compare it to the other assessments. | SEP | Phase 3 | Remyélinisation | Netherlands | À vérifier |
| Clemastine FumarateThe clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis. Clemastine fumarate was identified along with a series of other antimuscarinic medications as a potential remyelinating agent using the micropillar screen (BIMA) developed at the University of California, San Francisco (UCSF). Following in vivo validation, an FDA IND exemption was granted to investigate clemastine for the treatment of multiple sclerosis in the context of chronic optic neuropathy. That pilot study was recently completed and is the first randomized control trial documenting efficacy for a putative remyelinating agent for the treatment of MS. The preselected primary efficacy endpoint (visual evoked potential) was met and a strong trend to benefit was seen for the principal secondary endpoint assessing function (low contrast visual acuity). That trial number was 13-11577. This study seeks to follow up on that study and examine clemastine fumarate's protective and reparative effects in the context of chronic demyelinating brain lesions as imaged by multi-parametric MRI assessments. The investigators will be assessing the effects of clemastine fumarate as a remyelinating therapy and assessing its effect on MRI metrics of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis. In addition to using conventional multi-parametric MRI assessments, this study will also evaluate a new MRI technique called Ultrashort Echo Time (UTE) MRI to assess the effects of clemastine fumarate as a remyelinating therapy of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis and compare it to the other assessments. | SEP | Phase 1/2 | Remyélinisation | United States | À vérifier |
| Brain Development Research ProgramDr. Elliott Sherr and his collaborators at University of California, San Francisco (UCSF) are studying the genetic causes of disorders of cognition and epilepsy, in particular disorders of brain development that affect the corpus callosum, such as Aicardi syndrome, as well as two additional brain malformations, polymicrogyria and Dandy-Walker malformation. The goal of the investigators' research is to use a better understanding of the underlying genetic causes as a foundation to develop better treatments for these groups of patients. | Épilepsie | À vérifier | À vérifier | United States | À vérifier |
| ClemastineThe main purpose of this study is to assess clemastine as a remyelinating agent in patients with relapsing forms of multiple sclerosis. The study will also evaluate the tolerability of clemastine, originally approved as first-generation antihistamine, in patients with multiple sclerosis. Study procedures will include assessments for evidence of remyelination in the anterior visual pathway and in the brain using electrophysiologic techniques and magnetic resonance imaging. The study will also assess the robustness and stability of this clinical effect in patients taking clemastine for up to 3 months. Patients in this study can remain on their standard disease modifying treatment during the course of the study. However, patients cannot participate in any other investigational new drug research study concurrently. | SEP | Phase 2 | Remyélinisation | United States | À vérifier |
| Laser Interstitial Thermal Therapy (LITT)This study aims to evaluate the efficacy of Laser Interstitial Thermal Therapy (LITT) in treating recurrent or progressive, low-grade gliomas (LGG) in pediatric, adolescent and young adult patients. | Cancer | Non applicable | À vérifier | United States | À vérifier |
| Medtronic Percept Deep Brain Stimulation (adaptive DBS)Parkinson's disease (PD) affects \~1% of people over 60 years old, is highly disabling and represents a large economic burden. While dopaminergic medications effectively treat motor symptoms early in the disease, most patients develop complications, including motor fluctuations and dyskinesias, which can be partially managed by deep brain stimulation (DBS). This surgical therapy consists of delivering continuous electrical stimulation through electrodes permanently implanted in basal ganglia nuclei, with a pulse generator and battery unit implanted in the chest. However, conventional DBS therapy is delivered with constant stimulation parameters, referred to as constant deep brain stimulation (cDBS), that are unresponsive to patient activities or to variations in the severity of symptoms during daily life. This leaves many patients under- or over-stimulated during parts of the day. To address the shortcomings of cDBS, adaptive DBS (aDBS) uses real-time detection of neural signals to automatically adjust stimulation amplitude or other parameters in response to patients' dynamic clinical needs. aDBS was approved by the U.S. Food and Drugs Administration (FDA) for clinical treatment of PD in the Percept PC and RC (Medtronic) device in February 2025. Fully leveraging this therapy in the real world is limited by technical challenges, in particular the fact that: while the investigators developed a consistent pipeline for implementing aDBS, there were several critical control parameters that strongly influenced algorithm performance and required prolonged trial-and-error based testing, to achieve successful control. In this new study, the investigators seek to significantly extend this work and address the major barriers to widespread, easy adoption of aDBS by groups without specialized knowledge of neurophysiology or feedback control. Here the investigators aim to test an automated, data-driven pipeline for the recommendation of the adaptive control parameters. | Parkinson | Non applicable | Traitement symptomatique | United States | À vérifier |
| MS-CATCH — A Closed Loop, Doctor to Patient, Mobile Application for Depression in People With Multiple SclerosisThe researchers want to find out if an electronic application called MS CATCH can enhance patients' and doctors' experiences during and in between clinical visits. MS CATCH is a smartphone-based tool which allows patients to enter their mood related symptoms at regular intervals, which is then available to their Neurologist in their electronic medical record. The neurologist is also able to view additional information from their medical record, and receives alerts for changes reported by the patient that raise concern for the patient's mental health. | SEP | Non applicable | Traitement symptomatique | United States | À vérifier |
| Medtronic Percept Deep Brain Stimulation (cDBS)Parkinson's disease (PD) affects \~1% of people over 60 years old, is highly disabling and represents a large economic burden. While dopaminergic medications effectively treat motor symptoms early in the disease, most patients develop complications, including motor fluctuations and dyskinesias, which can be partially managed by deep brain stimulation (DBS). This surgical therapy consists of delivering continuous electrical stimulation through electrodes permanently implanted in basal ganglia nuclei, with a pulse generator and battery unit implanted in the chest. However, conventional DBS therapy is delivered with constant stimulation parameters, referred to as constant deep brain stimulation (cDBS), that are unresponsive to patient activities or to variations in the severity of symptoms during daily life. This leaves many patients under- or over-stimulated during parts of the day. To address the shortcomings of cDBS, adaptive DBS (aDBS) uses real-time detection of neural signals to automatically adjust stimulation amplitude or other parameters in response to patients' dynamic clinical needs. aDBS was approved by the U.S. Food and Drugs Administration (FDA) for clinical treatment of PD in the Percept PC and RC (Medtronic) device in February 2025. Fully leveraging this therapy in the real world is limited by technical challenges, in particular the fact that: while the investigators developed a consistent pipeline for implementing aDBS, there were several critical control parameters that strongly influenced algorithm performance and required prolonged trial-and-error based testing, to achieve successful control. In this new study, the investigators seek to significantly extend this work and address the major barriers to widespread, easy adoption of aDBS by groups without specialized knowledge of neurophysiology or feedback control. Here the investigators aim to test an automated, data-driven pipeline for the recommendation of the adaptive control parameters. | Parkinson | Non applicable | Traitement symptomatique | United States | À vérifier |
Essais cliniques
17| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| Medtronic Percept Deep Brain Stimulation (cDBS) | Parkinson | Non applicable | NCT07398157 | Subgaleal aDBS — Simplified, Scalable, 24-hour Adaptive DBS in Parkinson's Disease | RECRUITING |
| MS-CATCH — A Closed Loop, Doctor to Patient, Mobile Application for Depression in People With Multiple Sclerosis | SEP | Non applicable | NCT05865405 | MS-CATCH — A Closed Loop, Doctor to Patient, Mobile Application for Depression in People With Multiple Sclerosis | ACTIVE_NOT_RECRUITING |
| Medtronic Percept Deep Brain Stimulation (adaptive DBS) | Parkinson | Non applicable | NCT07682311 | Auto aDBS — Automated Daytime Adaptive Deep Brain Stimulation Parameter Optimization in Patients Implanted With Percept Neurostimulator | RECRUITING |
| Laser Interstitial Thermal Therapy (LITT) | Cancer | Non applicable | NCT07506239 | Efficacy of Laser Interstitial Thermal Therapy in Young Persons With Low-grade Glioma | RECRUITING |
| Clemastine | SEP | Phase 2 | NCT02040298 | ReBUILD — Assessment of Clemastine Fumarate as a Remyelinating Agent in Multiple Sclerosis | COMPLETED |
| Brain Development Research Program | Épilepsie | À vérifier | NCT00305305 | Brain Development Research Program | RECRUITING |
| Clemastine Fumarate | SEP | Phase 1/2 | NCT06065670 | ReINFORCE — Assessing Changes in Multi-parametric MRI in Patients With Acute Demyelinating Lesions Taking Clemastine Fumarate as a Myelin Repair Therapy | NOT_YET_RECRUITING |
| Clemastine Fumarate | SEP | Phase 3 | NCT05338450 | RESTORE — Clemastine Fumarate as Remyelinating Treatment in Internuclear Ophthalmoparesis and Multiple Sclerosis | TERMINATED |
| N-acetyl cysteine | SEP | Phase 2 | NCT02804594 | A Study of Oxidative Pathways in MS Fatigue | COMPLETED |
| Prospective Registry of Histotripsy for Soft Tissue Tumors | Cancer | À vérifier | NCT07470008 | Prospective Registry of Histotripsy for Soft Tissue Tumors | RECRUITING |
| IL1 Inhibition in FOP | Myopathies | À vérifier | NCT06724562 | IL1 Inhibition in FOP | RECRUITING |
| CNKSR2 Natural History Study | Épilepsie | À vérifier | NCT06500260 | CNKSR2 Natural History Study | ACTIVE_NOT_RECRUITING |
| Galcanezumab | Migraine | À vérifier | NCT06085144 | Emgality for Migraine in Breastmilk | RECRUITING |
| Clemastine Fumarate | SEP | Phase 1 | NCT05359653 | ReVIVE — Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair Therapy | RECRUITING |
| MS FIT — Multiple Sclerosis Falls Insight Track | SEP | Non applicable | NCT05837949 | MS FIT — Multiple Sclerosis Falls Insight Track | ACTIVE_NOT_RECRUITING |
| Rifaximin | Parkinson | Phase 1 | NCT03575195 | MICRO-PD — Microbiota Intervention to Change the Response of Parkinson's Disease | SUSPENDED |
| Pregnancy Registry, Infants, Serum/Milk Analysis (PRISMA) | Myasthénie | À vérifier | NCT06940323 | Pregnancy Registry, Infants, Serum/Milk Analysis (PRISMA) | RECRUITING |
Publications
13| Molécule | Indication / population | Titre | Journal | Date |
|---|---|---|---|---|
| Medtronic Percept Deep Brain Stimulation (cDBS) | Effects of theta burst stimulation on the Parkinsonian gait disorder and cortical gait-network activity. | Journal of Parkinson's disease | ||
| Medtronic Percept Deep Brain Stimulation (cDBS) | Chronic adaptive deep brain stimulation in Parkinson's disease: ADAPT-START findings and programming principles. | NPJ Parkinson's disease | ||
| CNKSR2 Natural History Study | CNKSR2-related neurodevelopmental and epilepsy disorder: a cohort of 13 new families and literature review indicating a predominance of loss of function pathogenic variants. | BMC medical genomics | ||
| Rifaximin | The role of systemic inflammation in hepatic encephalopathy: advances in inflammatory mechanisms, prevention and treatment research. | Annals of medicine | ||
| Rifaximin | Muribaculum as a microbial contributor of rifaximin-induced mucosal protection during chemotherapy. | Gut microbes | ||
| IL1 Inhibition in FOP | Long-term use of interleukin-1 inhibitors reduce flare activity in patients with fibrodysplasia ossificans progressiva. | Rheumatology (Oxford, England) | ||
| IL1 Inhibition in FOP | Innovative valsartan-loaded self-nanoemulsifying drug delivery system combat liver inflammation and oxidative stress in streptozotocin-induced diabetic rats. | Molecular and cellular biochemistry | ||
| Rifaximin | Electroacupuncture-mediated lncRNA TUG1 regulates the miR-127-3p/NF-κB p65 axis to inhibit IBS-D low-grade intestinal inflammation. | Frontiers in immunology | ||
| Rifaximin | Correction: Nishimura et al. Rifaximin Attenuates Liver Fibrosis and Hepatocarcinogenesis in a Rat MASH Model by Suppressing the Gut-Liver Axis and Epiregulin-IL-8-Associated Angiogenesis. 2025, , 6710. | International journal of molecular sciences | ||
| Clemastine Fumarate | Clemastine fumarate promotes myelin repair in a nonhuman primate model of demyelination characterized by absent spontaneous remyelination. | Proceedings of the National Academy of Sciences of the United States of America | ||
| Clemastine Fumarate | Green spectrofluorimetric determination of clemastine using nitrogen and phosphorus Co-doped carbon quantum dots with face-centered design optimization and sustainability assessment. | RSC advances | ||
| Rifaximin | Therapeutic drift or formulation drift: avoiding misconceptions about rifaximin and antimicrobial resistance in cirrhosis - Authors' reply. | The lancet. Gastroenterology & hepatology | ||
| Rifaximin | From Dysbiosis to systemic health: Microbiome modulation as a unified therapeutic framework. | Acta microbiologica et immunologica Hungarica |