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

This study has been completed.

First Received on October 18, 1999. Last Updated on June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	Medical University of South Carolina
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004402

Purpose

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.

Condition	Intervention	Phase
Osteopetrosis	Drug: calcitriol Drug: interferon gamma	Phase III

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: Melnick-Needles syndrome mucopolysaccharidosis type IV osteopetrosis

Drug Information available for: Calcitriol Interferon gamma-1b Gamma Rays Interferons

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	30
Study Start Date:	November 1999
Estimated Study Completion Date:	June 2000

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.

Eligibility

Ages Eligible for Study:	up to 10 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004402

Sponsors and Collaborators

FDA Office of Orphan Products Development

Medical University of South Carolina

Investigators

Study Chair:

L. Lyndon Key, Jr.

Medical University of South Carolina

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00004402 History of Changes
Other Study ID Numbers:	199/13284, MUSC-FDR000768
Study First Received:	October 18, 1999
Last Updated:	June 23, 2005
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

genetic diseases and dysmorphic syndromes
osteopetrosis
rare disease

Additional relevant MeSH terms:

Osteopetrosis
Osteosclerosis
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Calcitriol
Interferon-gamma
Interferon-gamma, Recombinant
Interferons
Vitamins
Micronutrients
Growth Substances

Physiological Effects of Drugs
Pharmacologic Actions
Bone Density Conservation Agents
Calcium Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Vasoconstrictor Agents
Cardiovascular Agents
Therapeutic Uses
Antiviral Agents
Anti-Infective Agents
Antineoplastic Agents

ClinicalTrials.gov processed this record on February 12, 2012