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

NCT00004402 | Completed Phase 3

• 2 other identifiers: 199/13284MUSC-FDR000768
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Osteopetrosis

Keywords

genetic diseases and dysmorphic syndromes; osteopetrosis; rare disease

Interventions

calcitriol DRUG

interferon gamma DRUG

Eligibility Criteria

• Age: Up to 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• FDA Office of Orphan Products Development: lead
• Medical University of South Carolina: collaborator

MeSH Terms

Conditions

Osteopetrosis; Genetic Diseases, Inborn; Rare Diseases

Interventions

Calcitriol; Interferon-gamma

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Dihydroxycholecalciferols; Hydroxycholecalciferols; Cholecalciferol; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids; Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Macrophage-Activating Factors; Lymphokines; Proteins; Biological Factors

Study Officials

• L. Lyndon Key, Jr.

  Medical University of South Carolina

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Purpose: TREATMENT
• Sponsor Type: FED

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: November 1, 1999
• Study Completion: June 1, 2000
• Last Updated: March 25, 2015

Record last verified: 2001-01