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Results for “Hereditary Inclusion Body Myopathy”

29 trials

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Showing 20 of 29 results

Large-scale testing (Phase 3)Study completedNCT02377921
What this trial is testing

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka Type+1 more
Ultragenyx Pharmaceutical Inc 89
Early research (Phase 1)Study completedNCT01519349
What this trial is testing

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis

Who this might be right for
Becker Muscular DystrophySporadic Inclusion Body Myositis
Nationwide Children's Hospital 15
Not applicableStudy completedNCT04009226
What this trial is testing

International GNE Myopathy Patient Registry

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy
Newcastle University 430
Testing effectiveness (Phase 2)Study completedNCT01830972
What this trial is testing

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy (HIBM)
Ultragenyx Pharmaceutical Inc 59
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 completedNCT01902940
What this trial is testing

Natural History in CCFDN and IBM Syndromes

Who this might be right for
Inclusion Body Myositis, SporadicInclusion Body Myopathy, Autosomal-recessiveInclusion Body Myopathy, Autosomal-dominant+1 more
Ludwig-Maximilians - University of Munich 350
Testing effectiveness (Phase 2)Study completedNCT01517880
What this trial is testing

Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy
Ultragenyx Pharmaceutical Inc 46
Not applicableStudy completedNCT02196909
What this trial is testing

Clinical, Biological and NMR Outcome Measures Study for Hereditary Inclusion Body Myopathy Due to Mutation of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine Kinase Gene (GNE)

Who this might be right for
HIBM
Institut de Myologie, France 20
Not applicableStudy completedNCT01784679
What this trial is testing

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body MyopathyGNE MyopathyNonaka Disease+2 more
Ultragenyx Pharmaceutical Inc 319
Testing effectiveness (Phase 2)Study completedNCT02346461
What this trial is testing

An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy

Who this might be right for
GNE Myopathy
National Human Genome Research Institute (NHGRI) 12
Early research (Phase 1)Study completedNCT01634750
What this trial is testing

Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body Myopathy (HIBM)GNE Myopathy
National Human Genome Research Institute (NHGRI) 26
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)Active Not RecruitingNCT04231266
What this trial is testing

Multi-Center Study of ManNAc for GNE Myopathy

Who this might be right for
GNE Myopathy
Leadiant Biosciences, Inc. 54
Testing effectiveness (Phase 2)Looking for participantsNCT07511556
What this trial is testing

First-in-human Study of UX016 in GNEM

Who this might be right for
GNE Myopathy
Ultragenyx Pharmaceutical Inc 24
Not applicableStudy completedNCT02810028
What this trial is testing

Acceptance and Commitment Therapy for Muscle Disease

Who this might be right for
Muscle Diseases
King's College Hospital NHS Trust 155
Early research (Phase 1)Study completedNCT00195637
What this trial is testing

Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy

Who this might be right for
Hereditary Inclusion Body Myopathy
National Human Genome Research Institute (NHGRI) 4
Not applicableLooking for participantsNCT07478172
What this trial is testing

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Who this might be right for
Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more
University of Missouri-Columbia 50
Not applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
Not applicableStudy completedNCT05890833
What this trial is testing

The Risk of Falls Index for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyFacioscapulohumeral Muscular Dystrophies+3 more
LMU Klinikum 30
Not applicableStudy completedNCT03317171
What this trial is testing

Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies

Who this might be right for
Muscular DystrophiesMuscle Inflammation
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico 29
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