Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
calcitriol
+ interferon gamma
Bone Diseases+9
+ Bone Diseases, Developmental
+ Musculoskeletal Diseases
Treatment Study
Summary
Study start date: November 1, 1999
Actual date on which the first participant was enrolled.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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.30 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Until 10 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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