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NCT00195637 · ClinicalTrials.gov registry record · Phase 1
Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
A Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT00195637 is a Phase 1 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 4 participants.
The verdict
NCT00195637, a Phase 1 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study Summary
This study will evaluate patients with Hereditary Inclusion Body Myopathy (HIBM) and examine the effects of immune globulin (IG) treatment on muscle and muscle function. HIBM is a progressive neuromuscular disease that begins in early adulthood, primarily affecting limb muscles. It results from mutations of the gene that is responsible for producing sialic acid, a sugar normally found on the surface of certain proteins, including alpha-dystroglycan, which is involved in muscle function. Some patients with HIBM have decreased sialic acid on the alpha-dystroglycan protein, which may be the cause of their muscle weakness. IG is a protein in the blood that carries a large amount of sialic acid. This study will administer IG to patients with HIBM and determine if the sialic acid in IG is taken up by muscle cells in these patients and if it can restore some of their muscle function. Four patients with HIBM will be admitted to this study at the NIH Clinical Center for evaluation and IG treatment. The evaluation lasts about 1 month. After completing baseline studies (see below), patients receive two intravenous doses of immune globulin (on days 6 and 7), followed by measurement of muscle strength 2 days later (day 9). They receive additional IG infusions on days 13, 20, and 27. A final set of tests is performed on day 29. Patients may leave the hospital on pass when no studies are being done. A patient's initial evaluation includes: * History and physical examination, neurological examination, eye examination * 24-hour urine collection * Blood tests on two separate days * Photographs showing the extent of muscle affected * Chest x-ray, electrocardiogram (EKG), and echocardiogram * Two muscle biopsies, one before and one after the IG treatments. For this procedure, a small sample of muscle tissue is surgically removed for examination under the microscope. * Muscle strength and endurance testing, including the following: The patient uses pulleys attached to machines that
Interventions
- DRUG Immune Globulin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2005-09-16 |
| Est. Completion | 2006-07-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00195637
The ClinicalTrials.gov registry entry for NCT00195637 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 4 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 Immune Globulin is the first listed.
NCT00195637 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00195637 about?
NCT00195637 is a clinical study titled "Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy". This study will evaluate patients with Hereditary Inclusion Body Myopathy (HIBM) and examine the effects of immune globulin (IG) treatment on muscle and muscle function. HIBM is a progressive neuromuscular disease that begins in early adulthood, primarily affecting limb muscles. It results from muta...
What is the current status of trial NCT00195637?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2005-09-16. Estimated completion is 2006-07-03.
What interventions are being tested in trial NCT00195637?
The interventions under investigation include: Immune Globulin (DRUG).
Who is sponsoring clinical trial NCT00195637?
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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