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NCT01784679 · ClinicalTrials.gov registry record
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)
A clinical trial, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- 319
- Enrollment target
NCT01784679 is a clinical trial that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 319 participants.
The verdict
NCT01784679 has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- 319 participants
- Enrollment target
Study Summary
HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and ultimately a wheelchair-bound state. The rate of progression is gradual and variable over the course of 10-20 years or longer. There is a need to better understand the disease-specific features of HIBM to heighten disease awareness; facilitate early diagnosis; identify patients; expand knowledge of the clinical presentation, progression and variation of the disease; identify and validate biomarkers and other efficacy measures; inform on the design and interpretation of clinical studies of investigational products; and eventually to optimize patient management.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 319 participants |
| Start Date | 2013-04-05 |
| Est. Completion | 2017-11-30 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01784679
The ClinicalTrials.gov registry entry for NCT01784679 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 319 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 0 interventions.
NCT01784679 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01784679 about?
NCT01784679 is a clinical study titled "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)". HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and ultimately a wheelchair-bound state. The rate of progression is gradual and va...
What is the current status of trial NCT01784679?
This trial is currently completed. The enrollment target is 319 participants. The study started on 2013-04-05. Estimated completion is 2017-11-30.
Who is sponsoring clinical trial NCT01784679?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
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