Sarepta Therapeutics, Inc. — Biomedical Watch V2

Traitements16programmes

Essais7liés

Publications14liées

SourceDBlocale

Traitements

16

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
AntibioticsThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.	Myopathies	Phase 4	À vérifier	United States	À vérifier
CasimersenThe main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).	Myopathies	Phase 3	À vérifier	United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, …	À vérifier
ELEVIDYSThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.	Myopathies	Phase 4	À vérifier	United States	À vérifier
GlucocorticoidsThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.	Myopathies	Phase 4	À vérifier	United States	À vérifier
GolodirsenThe main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).	Myopathies	Phase 3	À vérifier	United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, …	À vérifier
SirolimusThis study will evaluate the safety and effectiveness of a new immunosuppressive drug, sirolimus, in reducing the amount of protein in the urine in patients with membranous nephropathy. This condition involves damage to the walls of tiny blood vessel filters in the kidneys called glomeruli, which allows blood proteins to leak into the urine. Patients have low blood protein levels and high blood cholesterol. Some patients may have leg swelling, impaired kidney function, blood vessel and heart disease, and a risk of emboli (blood clots that travel to the lungs). Drugs currently used to treat membranous nephropathy vary in their effectiveness among patients and can cause severe side effects. The Food and Drug Administration has approved sirolimus for suppressing the immune system of patients who have had a kidney transplant to reduce the risk of organ rejection. The drug does not have certain side effects that have caused problems for patients treated with other immunosuppressants, such as: prednisone (weight gain, round face, diabetes, weak and fractured bones, and cataracts); cyclophosphamide (fertility problems, bladder injury and bladder cancer, and other cancers); chlorambucil (fertility problems, seizures, acute leukemia, and other cancers); and cyclosporine (kidney toxicity, increased facial hair, and seizures). Patients 13 years of age or older with idiopathic membranous nephropathy or lupus membranous nephropathy may be eligible for this study. Candidates must have completed at least one month of treatment with a stable dose of angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs). They will be screened with a medical history, physical examination, blood tests, skin test for exposure to tuberculosis, and an examination for infection, cancers, and other conditions that can cause membranous nephropathy. Participants will take sirolimus once a day for 1 year, except for the first day of treatment, when they will take three doses to quickly bring their blood levels of the drug up to a therapeutic level. They will undergo evaluations at the NIH in Bethesda, Maryland, at baseline (before starting treatment) and again at 1- to 4-month intervals during the study. In addition, they will have blood tests every week for the first month and every 2 weeks for the second month; then blood and urine tests once a month for the next 10 months of treatment and then every 4 months for a 12-month period after treatment stops. These tests will evaluate drug side effects and the response to therapy, and will determine if the therapeutic benefits persist long-term when treatment stops. Patients will also be asked to have optional kidney function tests during the baseline evaluation and at the end of the follow-up period to measure kidney filtration and blood flow rates. Those who participate will be given fluids and other substances by vein to accurately measure kidney function. They will then have blood and urine samples collected about four times over a 1-hour period after drinking fluids to increase urine output. Patients who experience a substantial increase in proteinuria or substantial decrease in kidney function during the course of treatment will stop taking sirolimus and be taken off the study.	Lupus, Myopathies	Phase 4	Traitement symptomatique	United States	À vérifier
SRP-1001 for InjectionThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.	Myopathies	Phase 1/2	À vérifier	Australia, Canada, Germany, Italy, Netherlands, New Zealand, Spain	À vérifier
SRP-1003 IV InfusionThis is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.	Myopathies	Phase 1/2	À vérifier	Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, …	À vérifier
SRP-1003 SC InjectionThis is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.	Myopathies	Phase 1/2	À vérifier	Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, …	À vérifier
SRP-9003The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.	Myopathies	Phase 1	À vérifier	United States	À vérifier
CasimersenThe main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).	Myopathies	Phase 3	À vérifier	United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, Italy, Poland, Spain, Sweden, United Kingdom	À vérifier
SRP-9003The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.	Myopathies	Phase 1	À vérifier	United States	À vérifier
SirolimusThis study will evaluate the safety and effectiveness of a new immunosuppressive drug, sirolimus, in reducing the amount of protein in the urine in patients with membranous nephropathy. This condition involves damage to the walls of tiny blood vessel filters in the kidneys called glomeruli, which allows blood proteins to leak into the urine. Patients have low blood protein levels and high blood cholesterol. Some patients may have leg swelling, impaired kidney function, blood vessel and heart disease, and a risk of emboli (blood clots that travel to the lungs). Drugs currently used to treat membranous nephropathy vary in their effectiveness among patients and can cause severe side effects. The Food and Drug Administration has approved sirolimus for suppressing the immune system of patients who have had a kidney transplant to reduce the risk of organ rejection. The drug does not have certain side effects that have caused problems for patients treated with other immunosuppressants, such as: prednisone (weight gain, round face, diabetes, weak and fractured bones, and cataracts); cyclophosphamide (fertility problems, bladder injury and bladder cancer, and other cancers); chlorambucil (fertility problems, seizures, acute leukemia, and other cancers); and cyclosporine (kidney toxicity, increased facial hair, and seizures). Patients 13 years of age or older with idiopathic membranous nephropathy or lupus membranous nephropathy may be eligible for this study. Candidates must have completed at least one month of treatment with a stable dose of angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs). They will be screened with a medical history, physical examination, blood tests, skin test for exposure to tuberculosis, and an examination for infection, cancers, and other conditions that can cause membranous nephropathy. Participants will take sirolimus once a day for 1 year, except for the first day of treatment, when they will take three doses to quickly bring their blood levels of the drug up to a therapeutic level. They will undergo evaluations at the NIH in Bethesda, Maryland, at baseline (before starting treatment) and again at 1- to 4-month intervals during the study. In addition, they will have blood tests every week for the first month and every 2 weeks for the second month; then blood and urine tests once a month for the next 10 months of treatment and then every 4 months for a 12-month period after treatment stops. These tests will evaluate drug side effects and the response to therapy, and will determine if the therapeutic benefits persist long-term when treatment stops. Patients will also be asked to have optional kidney function tests during the baseline evaluation and at the end of the follow-up period to measure kidney filtration and blood flow rates. Those who participate will be given fluids and other substances by vein to accurately measure kidney function. They will then have blood and urine samples collected about four times over a 1-hour period after drinking fluids to increase urine output. Patients who experience a substantial increase in proteinuria or substantial decrease in kidney function during the course of treatment will stop taking sirolimus and be taken off the study.	Lupus	Phase 2	Traitement symptomatique	United States	À vérifier
SRP-1003 IV InfusionThis is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.	Myopathies	Phase 1/2	À vérifier	Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan, Thailand, United Kingdom	À vérifier
SRP-1001 for InjectionThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.	Myopathies	Phase 1/2	À vérifier	Australia, Canada, Germany, Italy, Netherlands, New Zealand, Spain	À vérifier
ELEVIDYSThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.	Myopathies	Phase 4	À vérifier	United States	À vérifier

Essais cliniques

7

Molécule	Indication / population	Phase	NCT	Titre	Statut
ELEVIDYS	Myopathies	Phase 4	NCT07542314	ENHANCE — Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting	NOT_YET_RECRUITING
delandistrogene moxeparvovec	Myopathies	Phase 1	NCT04626674	ENDEAVOR — A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort	RECRUITING
SRP-1001 for Injection	Myopathies	Phase 1/2	NCT06131983	Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1	RECRUITING
SRP-1003 IV Infusion	Myopathies	Phase 1/2	NCT06138743	Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy	RECRUITING
Sirolimus	Lupus	Phase 2	NCT00050713	Sirolimus Therapy for Idiopathic and Lupus Membranous Nephropathy	COMPLETED
SRP-9003	Myopathies	Phase 1	NCT05876780	A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)	ACTIVE_NOT_RECRUITING
Casimersen	Myopathies	Phase 3	NCT03532542	An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy	TERMINATED

Publications

14

Molécule	Indication / population	Titre	Journal	Date
Antibiotics		Non-prescription antibiotic dispensing in community pharmacies in jordan: a simulated patient study.	The Libyan journal of medicine
Antibiotics		Biomimetic stress granules replenish lysosomal repair to reinstate macrophage immunometabolic antibacterial programs.	Bioactive materials
Casimersen		A Real-World Target Trial Emulation of Eteplirsen, Casimersen, and Golodirsen to Evaluate Survival Among Patients with Duchenne Muscular Dystrophy.	Advances in therapy
Sirolimus		Efficacy and safety of TPO-RA combined with sirolimus in the treatment of relapsed immune thrombocytopenia.	Hematology (Amsterdam, Netherlands)
Sirolimus		Antibody levels and immune memory in children with kaposiform hemangioendothelioma treated with sirolimus after catch up vaccination.	Human vaccines & immunotherapeutics
SRP-1003 SC Injection		Degradation-Robust Hue Prior Network for Low-Light Rainy Image Restoration.	Sensors (Basel, Switzerland)
SRP-1003 SC Injection		Elegans-Inspired Magnetic Polyurethane Soft Robot (MPSR) for Closed Visceral Secondary Hemostasis In Situ.	Advanced healthcare materials
SRP-1003 IV Infusion		Transient Neonatal Myasthenia Gravis in a Newborn Infant Presenting to an Emergency Department.	Cureus
SRP-1003 IV Infusion		Remifentanil attenuates LPS-induced hepatic injury by modulating NRF2/HO-1 and necroptosis-related gene expression.	Naunyn-Schmiedeberg's archives of pharmacology
SRP-9003		Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development.	Neurology. Clinical practice
Sirolimus		Survival and Homeostasis of Alveolar Macrophages in Vivo Depend on mTOR Signaling.	American journal of respiratory cell and molecular biology
Sirolimus		Fetal developmental venous anomaly with varix mimicking an intracranial hemorrhage.	American journal of obstetrics and gynecology
Casimersen		FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.	RNA biology
Casimersen		RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.	Biomolecules