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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title:

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 Summary:

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.

Sponsor: FDA Office of Orphan Products Development

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: FDA Office of Orphan Products Development

Dates:
Date Received: October 18, 1999
Date Started: November 1999
Date Completion: June 2000
Last Updated: March 24, 2015
Last Verified: January 2001