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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 is a Phase 1 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 26 participants.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01634750
The ClinicalTrials.gov registry entry for NCT01634750 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Human Genome Research Institute (NHGRI), which has 185 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which ManNAc is the first listed.
NCT01634750 reports 0 study locations.
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.
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