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Showing 1–13 of 13 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Hereditary Inclusion Body Myopathy (HIBM) Clear all
Completed No phase listed Observational

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

NCT01417533
View directory record Official record
Conditions
GNE Myopathy, GNE Related Diseases
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 21, 2026, 10:50 AM EDT
Terminated Phase 3 Interventional Results available

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)

NCT02736188
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy
Interventions
Aceneuramic Acid Extended-Release Tablets
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
143 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
5
States / cities
Los Angeles, California • Orange, California • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2023 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 1 Interventional

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

NCT01634750
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy (HIBM), GNE Myopathy
Interventions
ManNAc
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 18, 2017 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 2 Interventional

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

NCT01517880
View directory record Official record
Conditions
GNE Myopathy, Hereditary Inclusion Body Myopathy
Interventions
Sialic Acid Extended Release (SA-ER), Placebo
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
3
States / cities
Los Angeles, California • St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 15, 2016 · Synced Jul 21, 2026, 10:50 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

First-in-human Study of UX016 in GNEM

NCT07511556
View directory record Official record
Conditions
GNE Myopathy
Interventions
UX016, Placebo
Drug · Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Orange, California • Iselin, New Jersey
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 2 Interventional Results available

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

NCT01830972
View directory record Official record
Conditions
GNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM)
Interventions
SA-ER 500 mg, SA-IR 500 mg
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
3
States / cities
Los Angeles, California • St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 10, 2018 · Synced Jul 21, 2026, 10:50 AM EDT
Terminated Phase 2 Interventional Results available

A Study to 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

NCT02731690
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy, Inclusion Body Myopathy 2
Interventions
Aceneuramic Acid Extended-Release
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Orange, California • St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 18, 2019 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 1 Interventional

Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy

NCT00195637
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy
Interventions
Immune Globulin
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 3 Interventional Results available

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)

NCT02377921
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
Interventions
aceneuramic acid extended-release (Ace-ER), Placebo
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Irvine, California • St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 26, 2019 · Synced Jul 21, 2026, 10:50 AM EDT
Active, not recruiting Phase 2 Interventional

Multi-Center Study of ManNAc for GNE Myopathy

NCT04231266
View directory record Official record
Conditions
GNE Myopathy
Interventions
ManNAc, Placebo
Drug · Other
Lead sponsor
Leadiant Biosciences, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Los Angeles, California • Iowa City, Iowa • Kansas City, Kansas + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2025 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 1 Interventional

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

NCT01359319
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy (HIBM)
Interventions
Sialic Acid Extended Release (SA-ER) Tablets, Sialic Acid Extended Release (SA-ER) Tables
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 70 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Costa Mesa, California • New York, New York
Source: ClinicalTrials.gov public record
Updated May 20, 2012 · Synced Jul 21, 2026, 10:50 AM EDT
Completed No phase listed Observational

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)

NCT01784679
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM), Distal Myopathy With Rimmed Vacuoles (DMRV)
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
319 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Apr 26, 2018 · Synced Jul 21, 2026, 10:50 AM EDT
Completed Phase 2 Interventional Results available

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

NCT02346461
View directory record Official record
Conditions
GNE Myopathy
Interventions
ManNAc
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 15, 2019 · Synced Jul 21, 2026, 10:50 AM EDT