search
Back to results

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

Primary Purpose

Osteopetrosis

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
calcitriol
interferon gamma
Sponsored by
FDA Office of Orphan Products Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteopetrosis focused on measuring genetic diseases and dysmorphic syndromes, osteopetrosis, rare disease

Eligibility Criteria

undefined - 10 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    October 18, 1999
    Last Updated
    March 24, 2015
    Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Medical University of South Carolina
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT00004402
    Brief Title
    Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    January 2001
    Overall Recruitment Status
    Completed
    Study Start Date
    November 1999 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    June 2000 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Medical University of South Carolina

    4. Oversight

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Osteopetrosis
    Keywords
    genetic diseases and dysmorphic syndromes, osteopetrosis, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Enrollment
    30 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    calcitriol
    Intervention Type
    Drug
    Intervention Name(s)
    interferon gamma

    10. Eligibility

    Sex
    All
    Maximum Age & Unit of Time
    10 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    L. Lyndon Key, Jr.
    Organizational Affiliation
    Medical University of South Carolina
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

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

    We'll reach out to this number within 24 hrs