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Results for “Rare Genetic Muscle Diseases”

28 trials

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Showing 20 of 28 results

Large-scale testing (Phase 3)Study completedNCT00004646
What this trial is testing

Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
National Center for Research Resources (NCRR) 20
Not applicableLooking for participantsNCT06839469
What this trial is testing

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

Who this might be right for
Spinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy (DMD)
Columbia University 106
Not applicableLooking for participantsNCT07127978
What this trial is testing

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchene Muscular Dystrophy
ITF Therapeutics LLC 300
Not applicableStudy completedNCT00004685
What this trial is testing

Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Facioscapulohumeral
Ohio State University 90
Not applicableUnknownNCT05092230
What this trial is testing

Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)

Who this might be right for
Pompe Disease
University Hospital Center of Martinique 100
Not applicableAvailableNCT06931834
What this trial is testing

Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

Who this might be right for
Mitochondrial DiseaseChronic Kidney Disease
RiboNova Inc.
Not applicableActive Not RecruitingNCT05720923
What this trial is testing

Analysis of Muscular Properties in Patients With MFS and EDS

Who this might be right for
Rare DiseasesMarfan SyndromeEhlers-Danlos Syndrome
IRCCS Policlinico S. Donato 65
Post-approval studies (Phase 4)Study completedNCT00673608
What this trial is testing

Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Who this might be right for
HemoglobinopathiesMyelodysplastic SyndromesOther Inherited or Acquired Anaemia+4 more
Novartis 118
Testing effectiveness (Phase 2)Looking for participantsNCT06285539
What this trial is testing

Drug Rediscovery for Rare Immune Mediated Inflammatory Diseases

Who this might be right for
Behcet's DiseaseIdiopathic Inflammatory MyopathiesIgG4-related Disease
UMC Utrecht 60
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT00004353
What this trial is testing

Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia

Who this might be right for
Mitochondrial MyopathyMELAS SyndromeLactic Acidosis
National Center for Research Resources (NCRR) 35
Not applicableLooking for participantsNCT06795152
What this trial is testing

Rare Glycogen Storage Diseases Natural History Study

Who this might be right for
Glycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more
Duke University 200
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 applicableStudy completedNCT00004770
What this trial is testing

Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Who this might be right for
Mitochondrial Myopathy
National Center for Research Resources (NCRR) 1
Not applicableStudy completedNCT00004769
What this trial is testing

Study of Muscle Wasting and Altered Metabolism in Patients With Myotonic Dystrophy

Who this might be right for
Myotonic Muscular Dystrophy
University of Rochester 130
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Not applicableStudy completedNCT03713151
What this trial is testing

Feasibility of a Blended Therapy Approach

Who this might be right for
TelemedicineExerciseFeasibility+2 more
University of Zurich 30
Not applicableEnrolling By InvitationNCT03836300
What this trial is testing

Parent and Infant Inter(X)Action Intervention (PIXI)

Who this might be right for
Fragile X SyndromeAngelman SyndromePrader-Willi Syndrome+11 more
RTI International 120
Testing effectiveness (Phase 2)Study completedNCT01761292
What this trial is testing

Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
Italfarmaco 20
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