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

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.

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Key information

Conditions

Osteopetrosis Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias Osteosclerosis Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

Up to 10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

calcitriol

Drug

Interferon Gamma

Drug

Sponsors and collaborators

Lead sponsor

FDA Office of Orphan Products Development

Fed

Collaborators

Medical University of South Carolina

Registry information

Important dates

Study start: 1999
Study completion: 2000
First posted: Oct 19, 1999
Registry last updated: Mar 25, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed