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Results for “Inclusion Body Myopathy, Autosomal-recessive”

19 trials

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Showing 19 of 19 results

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
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 completedNCT01417533
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) 78
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
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 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 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
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
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
Testing effectiveness (Phase 2)Ended earlyNCT02731690
What this trial is testing

Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Who this might be right for
Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka Type+3 more
Ultragenyx Pharmaceutical Inc 42
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 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 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
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
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
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
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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07511556
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