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

Phase 3

Completed

• Conditions: Osteopetrosis

• Registration Number: NCT00004402
• Lead Sponsor: FDA Office of Orphan Products Development

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.

Study Timeline

• Study First Submitted: 1999-10-18
• Study First Submitted QC: 1999-10-18
• Study First Posted: 1999-10-19
• Study Start: 1999-11
• Study Completion: 2000-06
• Status Verified: 2001-01
• Last Update Submitted: 2015-03-24
• Last Update Posted: 2015-03-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified