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

March 24, 2015 updated by: FDA Office of Orphan Products Development

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment

30

Phase

  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 10 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

FDA Office of Orphan Products Development

Collaborators

Medical University of South Carolina

Investigators

  • Study Chair: L. Lyndon Key, Jr., Medical University of South Carolina

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 1999

Study Completion

June 1, 2000

Study Registration Dates

First Submitted

October 18, 1999

First Submitted That Met QC Criteria

October 18, 1999

First Posted (Estimate)

October 19, 1999

Study Record Updates

Last Update Posted (Estimate)

March 25, 2015

Last Update Submitted That Met QC Criteria

March 24, 2015

Last Verified

January 1, 2001

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 199/13284
  • MUSC-FDR000768

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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