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Results for “Hereditary Inclusion Body Myopathy”

29 trials

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Showing 20 of 29 results

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 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 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)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)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
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 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
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
Not applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
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
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
Post-approval studies (Phase 4)Not Yet RecruitingNCT07240649
What this trial is testing

Outcomes From Hyperbaric Oxygen (HBO2) Treatment for Emerging Indications

Who this might be right for
Post-COVID-19 ConditionUlcerative ColitisCrohn Disease+38 more
Jay C. Buckey Jr. 100
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 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
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
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
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
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