Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis

• Status: Completed
• Trial end date: 2000-06-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: FDA Office of Orphan Products Development
• Collaborator: Medical University of South Carolina
• Treatments: Calcitriol; Interferon-gamma; Interferons

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