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NCT01417533 · ClinicalTrials.gov registry record
A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
A clinical trial of GNE Myopathy and GNE Related Diseases, sponsored by National Human Genome Research Institute (NHGRI).
- Recruiting
- Registry status
- 125
- Enrollment target
- 1
- Study location
NCT01417533 is a study of GNE Myopathy and GNE Related Diseases that is actively recruiting participants, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 125 participants. The trial reports 1 study location across 1 state.
The verdict
NCT01417533, a study of GNE Myopathy and GNE Related Diseases, is actively recruiting participants, sponsored by National Human Genome Research Institute (NHGRI).
- RECRUITING
- Registry status
- 125 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: \- GNE Myopathy is a disease that causes walking difficulties and increasing muscle weakness. It usually develops in young adults (between 20 and 30 years of age), and affects arm and leg muscles. HIBM is caused by mutations in a gene that may affect how the muscles function. Researchers want to learn more about the causes, symptoms, and effects of HIBM. Objectives: \- To collect genetic and medical information from people with GNE Myopathy . Eligibility: \- Individuals between 18 and 80 years of age who have GNE Myopathy and do not use a wheelchair. - Participants must be willing to stop any current treatment of HIBM while enrolled in the study. Design: * Participants will be screened with a medical history, physical exam, and neurological exam. * At the first visit, participants will have the following tests: * Questionnaires about the impact of HIBM on daily activities, mood, and quality of life * 24-hour urine collection * Blood samples * Heart function tests * Muscle strength and endurance tests, including walking * Imaging study of the muscles * Participants will return for followup visits at 6, 12, and 18 months. They may be asked to return for a final visit at 24 months. Not all tests will be performed at each visit. * Treatment will not be provided as part of this protocol. For more information, visit our website: http://hibmstudy.nhgri.nih.gov/
Conditions Studied
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 125 participants |
| Start Date | 2011-09-14 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01417533
The ClinicalTrials.gov registry entry for NCT01417533 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 125 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 2 conditions, with GNE Myopathy appearing as the primary indexed condition, and to 0 interventions.
NCT01417533 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT01417533 about?
NCT01417533 is a clinical study titled "A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases". Background: \- GNE Myopathy is a disease that causes walking difficulties and increasing muscle weakness. It usually develops in young adults (between 20 and 30 years of age), and affects arm and leg muscles. HIBM is caused by mutations in a gene that may affect how the muscles function. Researcher...
What is the current status of trial NCT01417533?
This trial is currently recruiting. The enrollment target is 125 participants. The study started on 2011-09-14.
What conditions does trial NCT01417533 study?
This clinical trial studies the following conditions: GNE Myopathy, GNE Related Diseases.
Who is sponsoring clinical trial NCT01417533?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01417533 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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