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Results for “Rare Genetic Muscle Diseases”

31 trials

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Showing 20 of 31 results

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
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 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)Study completedNCT00004771
What this trial is testing

Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease

Who this might be right for
Spinal Muscular AtrophyAmyotrophic Lateral SclerosisSpinobulbar Muscular Atrophy
National Center for Research Resources (NCRR) 40
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
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 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
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
Not applicableActive Not RecruitingNCT03655223
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 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
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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990
What this trial is testing

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 60
Not applicableActive Not RecruitingNCT07558213
What this trial is testing

A Multi-Dimensional Model of cAre and transItion for Patients With cOmplex RAre Diseases

Who this might be right for
Rare DiseasesMitochondrial DiseasesMuscular Dystrophies+1 more
Fondazione Policlinico Universitario Agostino Gemelli IRCCS 136
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT02867592
What this trial is testing

Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

Who this might be right for
Adrenal Cortical CarcinomaAlveolar Soft Part SarcomaCentral Nervous System Neoplasm+41 more
National Cancer Institute (NCI) 109
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 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 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 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
Not applicableUnknownNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
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