Fondation Ophtalmologique Adolphe de Rothschild — Biomedical Watch V2

Traitements10programmes

Essais5liés

Publications1liées

SourceDBlocale

Traitements

10

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
MOVE — Study of Brain-spinal Cord Neural Connectivity in SpasticityLittle is known about the peripheral and central mechanisms of action of selective dorsal rhizotomy surgery for the treatment of spasticity. A better understanding of these mechanisms will enable us to improve the surgical procedure. This will require cortico-medullo-radiculo-muscular recordings never before performed and published in the literature, and the identification of variations in connectivity correlated with the clinic.	Myopathies	À vérifier	Traitement symptomatique	France	À vérifier
MS-Health — MS and Health CohortThis study aims to identify clinical, biological, imaging, and environmental factors that predict the progression and severity of Multiple Sclerosis (MS). We will establish a prospective, highly phenotyped cohort of patients diagnosed with MS according to the 2024 criteria, regardless of disease form or stage. We hypothesize that combining neurological, vascular, metabolic, neuropsychological, environmental, and imaging data (from the central nervous system and the eye) will improve the identification of markers associated with MS progression. This integrative approach will help clarify the respective roles of inflammation, vascular dysfunction, myelin repair, and neurodegeneration in disability accumulation. The study will also evaluate the impact of MS, disability, and treatments on patients' physical, mental, and social health, as defined by the World Health Organization (WHO). These results are expected to support personalized patient management and identify modifiable risk factors to reduce disability and inform future therapeutic strategies. The primary objective is to identify factors that worsen neurological disability in MS patients, including disease-related, comorbidity, and environmental factors. The main outcome measure is time to confirmed disability accumulation (CDA), defined as an increase in the EDSS (Expanded Disability Status Scale) score confirmed after at least 3 months. This single-center, 5-year prospective cohort study will be conducted at Hôpital Fondation Adolphe de Rothschild (Paris, France), with annual visits. A linkage with national health data (SNDS) will be established for both MS patients and a matched control group (5:1 ratio). Additional research procedures include: Ophthalmologic exams (OCT, angio-OCT, fundus photography, pupillometry) at baseline, year 1, 3, and 5. Brain MRI with additional non-contrast research sequences (annual). Clinical assessments including arterial stiffness and hearing tests. Blood sampling (up to 40 mL) for biomarker analyses and long-term biobanking (25 years). Lumbar puncture if clinically indicated at baseline (with extra samples for research). Physical activity and circadian rhythm monitoring using a wrist accelerometer for 9 consecutive days. Standardized questionnaires assessing quality of life, education, and social/professional impact. Inclusion criteria: Age ≥ 18 years Diagnosis of MS according to 2024 criteria	SEP	À vérifier	Remyélinisation		À vérifier
Ocrelizumab Injection [Ocrevus]Multiple sclerosis (MS) is a chronic autoimmune disease of the central nervous system and the leading cause of severe non-traumatic disability in young people, affecting 110,000 people in France. Ocrelizumab, a humanized anti-CD20 monoclonal antibody, has shown remarkable efficacy in Phase III trials on the inflammatory component of the disease, reducing the annualized relapse rate by 46% and the rate of new T2 lesions by 80% compared with interferon-β 1a. The use of anti-CD20 agents, including ocrelizumab, is associated with an infectious risk that increases with duration of exposure, part of which is due to the development of hypo-gammaglobulinemia in relation to cumulative dose. Several reports suggest a persistent effect of anti-CD20 drugs in MS, with no resumption of inflammatory activity after discontinuation: * During the development of ocrelizumab, at the end of phase 2, after having received 3 or 4 semi-annual cycles of ocrelizumab, a safety period with a therapeutic window of 18 months was planned, before re-administration in the extension study. During this therapeutic window, the annualized relapse rate remained stable, and patients showed no radiological disease activity. * Scandinavian observational studies of "off-label" use of anti-CD20 in MS provide real-life evidence of the absence of recovery of clinical and radiological activity after prolonged interruption of treatment. After 2 years of treatment, and with disease activity under control, spacing administration intervals could reduce the risk of infection without reducing treatment efficacy. This would facilitate the decision to maintain highly active immunotherapy over the long term. In addition, this therapeutic de-escalation, by reducing the frequency of infusions and associated day hospitalizations, would help to reduce treatment management costs. Our aim is to evaluate the non-inferiority of 12-monthly spacing of ocrelizumab infusions versus the conventional 6-monthly regimen, in a population of active MS patients over 18 years of age who have already received 4 or more semi-annual cycles of treatment for 2 years.	SEP	Phase 3	Immunomodulation	France	À vérifier
Ocular Mysathenia Gravis GeneralizationSecondary ocular myasthenia gravis (OMG) generalization represents a pejorative evolution and no validated generalization prevention strategy exists. The aim of this observational study was to determine the percentage of patients with OMG generalization and identify factors predictive of that pejorative evolution. Data from patients with OMG registered in the Fondation Hospital A. de Rothschild database between January 1990 and January 2017 were collected. Among the 183 patients registered in this database, 151 patients with available informations were analyzed.	Myasthénie	À vérifier	À vérifier	France	À vérifier
Optical Coherence Tomography and Optic Neuritis (OCTON)Optic neuritis (ON) can remain isolated or reveal a widespread and chronic disease of the central nervous system (CNS), a multiple sclerosis (MS) or, more rarely, a Devic's neuromyelitis optica (NMO) or a systemic disease. The optical coherence tomography (OCT) is a retinal imaging technique to measure the thickness of the retina and its different layers with an accuracy of 4-6 µM. Costello et al have shown that approximately 75% of 54 MS patients have developed within 3 to 6 months after a ON a loss of 10 to 40 µM in the thickness of the peripapillary retinal nerve fiber layer (RNFL). The etiologic diagnosis of ON has been transformed in recent years. MS can now be diagnosed by McDonald's MRI criteria and NMO by the AQP4 antibodies (anti-aquaporin- 4) antibodies and the anti-MOG (myelin-oligodendrocyte glycoprotein) antibodies. The diagnosis and prognosis value of the OCT in patients with ON is not well known	SEP	À vérifier	Remyélinisation	France	À vérifier
Ocular Mysathenia Gravis GeneralizationSecondary ocular myasthenia gravis (OMG) generalization represents a pejorative evolution and no validated generalization prevention strategy exists. The aim of this observational study was to determine the percentage of patients with OMG generalization and identify factors predictive of that pejorative evolution. Data from patients with OMG registered in the Fondation Hospital A. de Rothschild database between January 1990 and January 2017 were collected. Among the 183 patients registered in this database, 151 patients with available informations were analyzed.	Myasthénie	À vérifier	À vérifier	France	À vérifier
MOVE — Study of Brain-spinal Cord Neural Connectivity in SpasticityLittle is known about the peripheral and central mechanisms of action of selective dorsal rhizotomy surgery for the treatment of spasticity. A better understanding of these mechanisms will enable us to improve the surgical procedure. This will require cortico-medullo-radiculo-muscular recordings never before performed and published in the literature, and the identification of variations in connectivity correlated with the clinic.	Myopathies	À vérifier	Traitement symptomatique	France	À vérifier
MS-Health — MS and Health CohortThis study aims to identify clinical, biological, imaging, and environmental factors that predict the progression and severity of Multiple Sclerosis (MS). We will establish a prospective, highly phenotyped cohort of patients diagnosed with MS according to the 2024 criteria, regardless of disease form or stage. We hypothesize that combining neurological, vascular, metabolic, neuropsychological, environmental, and imaging data (from the central nervous system and the eye) will improve the identification of markers associated with MS progression. This integrative approach will help clarify the respective roles of inflammation, vascular dysfunction, myelin repair, and neurodegeneration in disability accumulation. The study will also evaluate the impact of MS, disability, and treatments on patients' physical, mental, and social health, as defined by the World Health Organization (WHO). These results are expected to support personalized patient management and identify modifiable risk factors to reduce disability and inform future therapeutic strategies. The primary objective is to identify factors that worsen neurological disability in MS patients, including disease-related, comorbidity, and environmental factors. The main outcome measure is time to confirmed disability accumulation (CDA), defined as an increase in the EDSS (Expanded Disability Status Scale) score confirmed after at least 3 months. This single-center, 5-year prospective cohort study will be conducted at Hôpital Fondation Adolphe de Rothschild (Paris, France), with annual visits. A linkage with national health data (SNDS) will be established for both MS patients and a matched control group (5:1 ratio). Additional research procedures include: Ophthalmologic exams (OCT, angio-OCT, fundus photography, pupillometry) at baseline, year 1, 3, and 5. Brain MRI with additional non-contrast research sequences (annual). Clinical assessments including arterial stiffness and hearing tests. Blood sampling (up to 40 mL) for biomarker analyses and long-term biobanking (25 years). Lumbar puncture if clinically indicated at baseline (with extra samples for research). Physical activity and circadian rhythm monitoring using a wrist accelerometer for 9 consecutive days. Standardized questionnaires assessing quality of life, education, and social/professional impact. Inclusion criteria: Age ≥ 18 years Diagnosis of MS according to 2024 criteria	SEP	À vérifier	Remyélinisation		À vérifier
Ocrelizumab Injection [Ocrevus]Multiple sclerosis (MS) is a chronic autoimmune disease of the central nervous system and the leading cause of severe non-traumatic disability in young people, affecting 110,000 people in France. Ocrelizumab, a humanized anti-CD20 monoclonal antibody, has shown remarkable efficacy in Phase III trials on the inflammatory component of the disease, reducing the annualized relapse rate by 46% and the rate of new T2 lesions by 80% compared with interferon-β 1a. The use of anti-CD20 agents, including ocrelizumab, is associated with an infectious risk that increases with duration of exposure, part of which is due to the development of hypo-gammaglobulinemia in relation to cumulative dose. Several reports suggest a persistent effect of anti-CD20 drugs in MS, with no resumption of inflammatory activity after discontinuation: * During the development of ocrelizumab, at the end of phase 2, after having received 3 or 4 semi-annual cycles of ocrelizumab, a safety period with a therapeutic window of 18 months was planned, before re-administration in the extension study. During this therapeutic window, the annualized relapse rate remained stable, and patients showed no radiological disease activity. * Scandinavian observational studies of "off-label" use of anti-CD20 in MS provide real-life evidence of the absence of recovery of clinical and radiological activity after prolonged interruption of treatment. After 2 years of treatment, and with disease activity under control, spacing administration intervals could reduce the risk of infection without reducing treatment efficacy. This would facilitate the decision to maintain highly active immunotherapy over the long term. In addition, this therapeutic de-escalation, by reducing the frequency of infusions and associated day hospitalizations, would help to reduce treatment management costs. Our aim is to evaluate the non-inferiority of 12-monthly spacing of ocrelizumab infusions versus the conventional 6-monthly regimen, in a population of active MS patients over 18 years of age who have already received 4 or more semi-annual cycles of treatment for 2 years.	SEP	Phase 3	Immunomodulation	France	À vérifier
Optical Coherence Tomography and Optic Neuritis (OCTON)Optic neuritis (ON) can remain isolated or reveal a widespread and chronic disease of the central nervous system (CNS), a multiple sclerosis (MS) or, more rarely, a Devic's neuromyelitis optica (NMO) or a systemic disease. The optical coherence tomography (OCT) is a retinal imaging technique to measure the thickness of the retina and its different layers with an accuracy of 4-6 µM. Costello et al have shown that approximately 75% of 54 MS patients have developed within 3 to 6 months after a ON a loss of 10 to 40 µM in the thickness of the peripapillary retinal nerve fiber layer (RNFL). The etiologic diagnosis of ON has been transformed in recent years. MS can now be diagnosed by McDonald's MRI criteria and NMO by the AQP4 antibodies (anti-aquaporin- 4) antibodies and the anti-MOG (myelin-oligodendrocyte glycoprotein) antibodies. The diagnosis and prognosis value of the OCT in patients with ON is not well known	SEP	À vérifier	Remyélinisation	France	À vérifier

Essais cliniques

5

Molécule	Indication / population	Phase	NCT	Titre	Statut
Optical Coherence Tomography and Optic Neuritis (OCTON)	SEP	À vérifier	NCT02573792	Optical Coherence Tomography and Optic Neuritis (OCTON)	COMPLETED
Ocrelizumab Injection [Ocrevus]	SEP	Phase 3	NCT05999604	WINDOCRE — Impact of Annual Versus Biannual Infusions of Ocrelizumab in Patients With Active MS,After 2 Years of Initial Treatment, on Freedom From Radiological Disease Activity at Two Years: a Multicenter Randomized Controlled Non-inferiority Trial	ACTIVE_NOT_RECRUITING
MS-Health — MS and Health Cohort	SEP	À vérifier	NCT07645703	MS-Health — MS and Health Cohort	NOT_YET_RECRUITING
MOVE — Study of Brain-spinal Cord Neural Connectivity in Spasticity	Myopathies	À vérifier	NCT06281223	MOVE — Study of Brain-spinal Cord Neural Connectivity in Spasticity	RECRUITING
Ocular Mysathenia Gravis Generalization	Myasthénie	À vérifier	NCT04667650	Ocular Mysathenia Gravis Generalization	COMPLETED

Publications

1

Molécule	Indication / population	Titre	Journal	Date
Ocular Mysathenia Gravis Generalization		[Effectiveness of plasmapheresis in adolescent myasthenia gravis treatment].	Przeglad lekarski