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

NCT ID: NCT00004402

Last Updated: 2015-03-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1999-11-30
• Study Completion Date: 2000-06-30

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.

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.

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.

Conditions

Osteopetrosis

Study Design

• Primary Study Purpose: TREATMENT

Interventions

calcitriol

Intervention Type: DRUG

interferon gamma

Intervention Type: DRUG

Eligibility Criteria

Inclusion 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

• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Medical University of South Carolina

OTHER

Sponsor Role: collaborator

FDA Office of Orphan Products Development

FED

Sponsor Role: lead

Principal Investigators

L. Lyndon Key, Jr.

Role: STUDY_CHAIR

Medical University of South Carolina

Other Identifiers

MUSC-FDR000768

Identifier Type: -

Identifier Source: secondary_id

199/13284

Identifier Type: -

Identifier Source: org_study_id