Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

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

This study has been completed.
Medical University of South Carolina
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: January 2001

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
Drug: calcitriol
Drug: interferon gamma
Phase 3

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

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.


Ages Eligible for Study:   up to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004402

Sponsors and Collaborators
FDA Office of Orphan Products Development
Medical University of South Carolina
Study Chair: L. Lyndon Key, Jr. Medical University of South Carolina
  More Information Identifier: NCT00004402     History of Changes
Other Study ID Numbers: 199/13284
Study First Received: October 18, 1999
Last Updated: March 24, 2015

Keywords provided by FDA Office of Orphan Products Development:
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Calcium Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Vasoconstrictor Agents
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents processed this record on April 27, 2017