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

Sponsor

FDA Office of Orphan Products Development (U.S. Fed)

Overall Status

Completed

CT.gov ID

NCT00004402

Collaborator

Medical University of South Carolina (Other)

Enrollment

Duration (Months)

Study Details

Study Description

Brief 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.

Compare the number of adverse events or clinical manifestations of disease progression occurring in these patients.
Assess the effects of interferon gamma on hematopoiesis, cranial nerve function, and rate of infection in these patients.

Condition or Disease	Intervention/Treatment	Phase
Osteopetrosis	Drug: calcitriol Drug: interferon gamma	Phase 3

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.

Study Design

Study Type:

Interventional

Primary Purpose:

Treatment

Study Start Date :

Nov 1, 1999

Study Completion Date :

Jun 1, 2000

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 10 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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