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Results for “Hereditary Inclusion Body Myopathy”

27 trials

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Showing 20 of 27 results

Early research (Phase 1)Study completedNCT01236898
What this trial is testing

Pharmacokinetic Study on N-acetylneuraminic Acid

Who this might be right for
Nonaka MyopathyHereditary Inclusion Body Myopathy
Tohoku University 6
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 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
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 applicableLooking for participantsNCT01417533
What this trial is testing

A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases

Who this might be right for
GNE MyopathyGNE Related Diseases
National Human Genome Research Institute (NHGRI) 125
Early research (Phase 1)Study completedNCT01359319
What this trial is testing

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body Myopathy (HIBM)
Ultragenyx Pharmaceutical Inc 26
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 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
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
Not applicableUnknownNCT04009226
What this trial is testing

International GNE Myopathy Patient Registry

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy
Newcastle University 430
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
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
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
Large-scale testing (Phase 3)Study completedNCT04671472
What this trial is testing

Efficacy Confirmation Study of NPC-09

Who this might be right for
GNE MyopathyDistal Myopathy With Rimmed Vacuoles (DMRV)Hereditary Inclusion Body Myopathy (hIBM)+1 more
Nobelpharma 14
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
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
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
Large-scale testing (Phase 3)Ended earlyNCT02736188
What this trial is testing

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets 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+2 more
Ultragenyx Pharmaceutical Inc 143
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
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