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NCT01634750 · ClinicalTrials.gov registry record · Phase 1
Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
A Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 1
- Development phase
- 26
- Enrollment target
NCT01634750: Completed Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
NCT01634750 is a Phase 1 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 26 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01634750, a Phase 1 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 26 participants
- Enrollment target
Study Summary
Background: \- Hereditary inclusion body myopathy (HIBM) is a genetic disorder caused by mutations in a gene called GNE. This gene is responsible for producing a sugar called sialic acid. Low levels of sialic acid may cause muscle problems. Symptoms of HIBM include walking difficulties and muscle weakness, which usually start in a person s 20s or 30s and become worse over time. Researchers are studying a drug called ManNAc. It may be useful for treating HIBM. However, this drug is still being tested. Researchers want to see how ManNAc is absorbed into and removed from the blood. They will not be looking specifically at whether ManNAc can stop or slow the symptoms of HIBM. Objectives: * \<TAB\>To study how MaNAc is absorbed into and removed from the blood in people with HIBM. * \<TAB\>To study of safety of ManNAc in people with HIBM. Eligibility: \- Individuals between 18 and 70 years of age who have HIBM. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. * Participants will have a 3 to 4-day inpatient stay for the main part of the study. * Participants will be divided into groups of six. In each group, four will take ManNAc and two will take a placebo. Participants will not know which one they will receive. * Participants will have a single dose of either ManNAc or placebo. They will be monitored for any possible side effects. Frequent blood samples will be collected during the 4-day stay. * No treatment for HIBM will be provided as part of this study.
Interventions
- DRUG ManNAc
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2012-09-11 |
| Est. Completion | 2013-05-29 |
| Phase | Phase 1 |
What the finished NCT01634750 record still lists
NCT01634750 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower).
The record links to 0 conditions, and to 1 intervention - of which ManNAc is the first listed.
NCT01634750 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01634750 about?
NCT01634750 is a clinical study titled "Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)". Background: \- Hereditary inclusion body myopathy (HIBM) is a genetic disorder caused by mutations in a gene called GNE. This gene is responsible for producing a sugar called sialic acid. Low levels of sialic acid may cause muscle problems. Symptoms of HIBM include walking difficulties and muscle w...
What is the current status of trial NCT01634750?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 26 participants. The study started on 2012-09-11. Estimated completion is 2013-05-29.
What interventions are being tested in trial NCT01634750?
The interventions under investigation include: ManNAc (DRUG).
Who is sponsoring clinical trial NCT01634750?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01634750's enrollment target sits among peer trials
26 1482nd of 2000 higher than 506 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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