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Results for “Rare Genetic Muscle Diseases”

29 trials

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Showing 20 of 29 results

Not applicableLooking for participantsNCT06539169
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
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 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 completedNCT03680365
What this trial is testing

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

Who this might be right for
Duchenne Muscular DystrophyBurden, DependencyDisability Physical+3 more
Jett Foundation, Inc. 60
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
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 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 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 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
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
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
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 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.
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
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
Early research (Phase 1)Active Not RecruitingNCT03478462
What this trial is testing

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

Who this might be right for
Pediatric Solid TumorPediatric LymphomaPediatric Brain Tumor+5 more
Cellectar Biosciences, Inc. 30
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 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
Large-scale testing (Phase 3)Study completedNCT00004802
What this trial is testing

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders

Who this might be right for
Paralysis, Hyperkalemic PeriodicHypokalemic Periodic ParalysisParamyotonia Congenita
National Center for Research Resources (NCRR) 64
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
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