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

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Primary Purpose

Status

Completed

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

calcitriol

interferon gamma

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

NCT ID

NCT00004402

First Posted

October 18, 1999

Last Updated

March 24, 2015

Sponsor

FDA Office of Orphan Products Development

Collaborators

Medical University of South Carolina

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

Osteopetrosis

About this trial

Eligibility Criteria

Sites / Locations

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial