Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse
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 6 Trials 
8 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Cances, Claude
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Active, not recruiting
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
10/24
12/24
NusiMFM, NCT04602195: Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen

Completed
N/A
33
Europe
Hospices Civils de Lyon
Spinal Muscular Atrophy
11/23
11/23
NCT04003922: Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term

Recruiting
N/A
40
Europe
Adalimumab treatment
Assistance Publique Hopitaux De Marseille
Rasmussen Encephalitis
04/24
10/25
IMPROVE, NCT04802135: Creation of a Register of Patients With Neonatal-onset Epileptic Encephalopathy

Recruiting
N/A
200
Europe
Survey
Assistance Publique Hopitaux De Marseille
Epileptic Encephalopathy
09/25
09/25
DOPADEF, NCT05211609: Prevalence of High Plasmatic 3OMethyldopa Level in a Specific Population of Patients With a Symptomatology Compatible With AADC Deficiency

Recruiting
N/A
388
Europe
Plasmatic 3-O-Methyldopa dosage
University Hospital, Montpellier
Aromatic L-Amino Acid Decarboxylase Deficiency
05/24
11/24
PHENO SMART, NCT06321965: Characterization of New Phenotypes of Patients with Spinal Muscular Atrophy Treated with SMN Restoring Therapy

Recruiting
N/A
60
Europe
evaluation of muscle function, First-line cognitive assessment, second-line cognitive assessment, Cardiac evaluation, MRI, Assessment of activity and muscle fatigue, Assessment of bulbar function, Evaluation of body composition and metabolism, Questionnaires, Biocollection, Skinfold measurement
Hospices Civils de Lyon
Spinal Muscular Atrophy
01/28
01/28
RetroNusiMFM, NCT04644393: Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen

Not yet recruiting
N/A
40
Europe
Hospices Civils de Lyon
Spinal Muscular Atrophy
03/21
03/21
MFM-Play, NCT05227274: Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules

Completed
N/A
70
Europe
Items completion with or without a MFM-Play
Hospices Civils de Lyon
Neuromuscular Diseases
07/24
07/24

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