Status:
COMPLETED
Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
Lead Sponsor:
National Human Genome Research Institute (NHGRI)
Conditions:
Hereditary Inclusion Body Myopathy
Eligibility:
All Genders
18-70 years
Phase:
PHASE1
Brief 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 neuromus...
Detailed Description
Hereditary Inclusion Body Myopathy (HIBM) is an autosomal recessive neuromuscular disorder with onset in early adulthood and progressive muscle weakness leading to death within 2-3 decades. The causat...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Age 18-70 years, either gender
- Diagnosis of HIBM based upon a consistent clinical course plus either convincing muscle histology or identification of GNE gene mutations
- Ability to travel to the NIH Clinical Research Center for admissions
- EXCLUSION CRITERIA:
- Age less than18 or greater than 70, pregnancy
- Previous adverse reaction to IvIg that did not resolve with acetaminophen or benadryl treatment
- History of myocardial infarction, stroke, or kidney disease
- Psychiatric illness or neurological disease that interferes with compliance or communication with health care personnel
- Current malignancy
- Uncontrolled hypertension (blood pressure greater than180 systolic or greater than 95 diastolic)
- Electrocardiogram changes indicative of myocardial infarction, arrhythmia, tachycardia, bradycardia, left bundle branch block
- Chest radiographic abnormalities, including an infiltrate, mass, congestive heart failure, embolism, atelectasis
- Serum potassium less than 3.0 mEq/L
- Serum creatinine greater than 2.0 mg/dL
- SGPT or SGOT greater than 70 U/L
- Hemoglobin less than 10.0 g/dL
- Platelets less than100 k/mm(3)
- WBC less than 3.0 k/microliters
- ESR greater than100 mm/h
Exclusion
Key Trial Info
Start Date :
September 16 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 3 2006
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT00195637
Start Date
September 16 2005
End Date
July 3 2006
Last Update
December 4 2019
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892