Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
Information source: FDA Office of Orphan Products Development
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Osteopetrosis
Intervention: calcitriol (Drug); interferon gamma (Drug)
Phase: Phase 3
Status: Completed
Sponsored by: FDA Office of Orphan Products Development Official(s) and/or principal investigator(s):
L. Lyndon Key, Jr., Study Chair, Affiliation: Medical University of South Carolina
Summary
OBJECTIVES: I. Compare the rate of treatment failure in osteopetrosis patients receiving
interferon gamma in combination with calcitriol to the rate of treatment failure in patients
receiving calcitriol alone.
II. Compare the number of adverse events or clinical manifestations of disease progression
occurring in these patients.
III. Assess the effects of interferon gamma on hematopoiesis, cranial nerve function, and
rate of infection in these patients.
Clinical Details
Study design: Treatment
Detailed description:
PROTOCOL OUTLINE: This is a randomized, placebo controlled, open label study. Patients are
randomized to one of two arms (interferon gamma in combination with calcitriol or calcitriol
alone).
Arm I: Patients receive calcitriol once daily. Interferon gamma is administered by
subcutaneous injection three times a week.
Arm II: Patients receive calcitriol once daily. Patients may continue treatment in the
absence of toxicity and disease progression. If disease progression is diagnosed in the
control group, patients will then receive interferon gamma in combination with calcitriol.
Patients are followed every 4 weeks.
Eligibility
Minimum age: N/A.
Maximum age: 10 Years.
Gender(s): Both.
Criteria:
PROTOCOL ENTRY CRITERIA:
- -Disease Characteristics--
- Biopsy and x-ray confirmed primary osteopetrosis
- Presence of anemia and/or cranial nerve compression
- -Prior/Concurrent Therapy--
- Biologic therapy: No prior/concurrent bone marrow transplantation No prior interferon
gamma No other investigational biologic agents
- Chemotherapy: No prior/concurrent chemotherapeutic agents for bone marrow
transplantation
- Endocrine therapy: Prior/concurrent corticosteroid as supportive therapy allowed
- Radiotherapy: Not specified
- Surgery: At least 5 days since major surgery
- Other: Prior/concurrent calcitriol as supportive therapy allowed Prior/concurrent
transfusion as supportive therapy allowed Prior/concurrent dietary therapy allowed
- -Patient Characteristics--
- Age: 2 months to 10 years
- Performance status: Not specified
- Life expectancy: At least 6 months
- Hematopoietic: Not specified
- Hepatic: Bilirubin less than 2 mg/dL
- Renal: Creatinine less than 1. 5 mg/dL OR Creatinine clearance greater than 50 mL/min
- Pulmonary: No uncorrected airway obstruction
- Other: No active infection requiring intravenous antibiotics No known seizure disorder
not related to hypocalcemia No uncorrected hydrocephalus No MRI evidence of cerebral
atrophy Must maintain or gain body weight No sleep apnea No thrombocytopenia No
massive splenomegaly
Locations and Contacts
Additional Information
Starting date: November 1999
Ending date: June 2000
Last updated: June 23, 2005
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