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Results for “Charcot-Marie-Tooth Disease, Type 1”

21 trials

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Showing 20 of 21 results

Testing effectiveness (Phase 2)Ended earlyNCT03124459
What this trial is testing

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

Who this might be right for
Charcot-Marie-Tooth Disease
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA 63
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Not applicableStudy completedNCT06218134
What this trial is testing

Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E

Who this might be right for
Charcot-Marie-Tooth Disease, Type 1
Samsung Medical Center 3
Not applicableLooking for participantsNCT06092346
What this trial is testing

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Who this might be right for
AMPD3, OMIM*102772, AMP Deaminase DeficiencyAK1, OMIM *103000, Adenylate Kinase DeficiencyAMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency+25 more
National Human Genome Research Institute (NHGRI)
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

Who this might be right for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Testing effectiveness (Phase 2)Ended earlyNCT03943290
What this trial is testing

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Who this might be right for
Facioscapulohumeral Muscular DystrophyCharcot-Marie-Tooth Disease
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA 62
Large-scale testing (Phase 3)Active Not RecruitingNCT04695080
What this trial is testing

ChariotMS - Cladribine to Halt Deterioration in People With Advanced Multiple Sclerosis

Who this might be right for
Advanced Multiple SclerosisProgressive Multiple Sclerosis
Queen Mary University of London 204
Not applicableLooking for participantsNCT07136844
What this trial is testing

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Who this might be right for
Neuromuscular DiseasesObesity (Disorder)Myotonic Dystrophy 1+9 more
Centre Hospitalier Universitaire de Liege 300
Not applicableLooking for participantsNCT06708468
What this trial is testing

Personalized Training for People With Rare Neuromuscular Disorders

Who this might be right for
Neuromuscular Diseases (NMD)Charcot Marie Tooth Disease (CMT)Facioscapulohumeral Muscular Dystrophy+1 more
Oslo University Hospital 120
Not applicableUnknownNCT03550300
What this trial is testing

Muscle MRI in Charcot Mary Tooth Disease: a Prospective Cohort Study

Who this might be right for
Charcot-Marie-Tooth Disease, Type IACharcot-Marie-Tooth Disease Type 2ACharcot-Marie-Tooth Disease, Type IB+1 more
University College, London 130
Not applicableStudy completedNCT02532244
What this trial is testing

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

Who this might be right for
Spinal Muscular AtrophyCharcot-Marie-Tooth DiseaseMuscular Dystrophy+5 more
Nemours Children's Clinic 230
Early research (Phase 1)Study completedNCT05947578
What this trial is testing

The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease.

Who this might be right for
Charcot Marie Tooth Disease, Type 1
Cellatoz Therapeutics, Inc 9
Not applicableLooking for participantsNCT05982119
What this trial is testing

Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Who this might be right for
Duchenne Muscular DystrophyFascioscapulohumeral Muscular DystrophyMyotonic Dystrophy 1+3 more
Centre Hospitalier Universitaire de Liege 300
Not applicableStudy completedNCT07263256
What this trial is testing

Median Nerve Decompression for Complex Regional Pain Syndrome: A Clinical Study

Who this might be right for
Complex Regional Pain Syndrome I (CRPS I)Nerve Entrapment Syndrome
Jitka Fricova 74
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Early research (Phase 1)Study completedNCT05333406
What this trial is testing

Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A

Who this might be right for
Charcot-Marie-Tooth Disease, Type IA
ENCell 9
Testing effectiveness (Phase 2)Study completedNCT06482437
What this trial is testing

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

Who this might be right for
Charcot-Marie-Tooth Disease
NMD Pharma A/S 81
Large-scale testing (Phase 3)Active Not RecruitingNCT04762758
What this trial is testing

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

Who this might be right for
Charcot-Marie-Tooth Disease
Pharnext S.C.A. 350
Not applicableLooking for participantsNCT01668186
What this trial is testing

Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)

Who this might be right for
Peroxisome Biogenesis DisorderZellweger Spectrum DisorderRCDP - Rhizomelic Chondrodysplasia Punctata+8 more
McGill University Health Centre/Research Institute of the McGill University Health Centre 244
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