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

Home

Study Topics

Glossary


	Full Text View


	Tabular View


	Contacts and Locations


	No Study Results Posted


	Related Studies

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

This study has been completed.

Study NCT00004402. Last updated on June 23, 2005. Information provided by FDA Office of Orphan Products Development

This Tabular View shows the required WHO registration data elements as marked by ^†

Descriptive Information Fields

Brief Title ^†

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

Official Title ^†

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.

Study Phase

Phase III

Study Type ^†

Interventional

Study Design ^†

Treatment

Primary Outcome Measure ^†

Secondary Outcome Measure ^†

Condition ^†

Osteopetrosis

Intervention ^†

Drug: calcitriol
Drug: interferon gamma

MEDLINE PMIDs

Links

Recruitment Information Fields

Recruitment Status ^†

Completed

Enrollment ^†

Start Date ^†

November 1999

Completion Date

June 2000

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

Gender

Both

Ages

up to 10 Years

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

Administrative Information Fields

NCT ID ^†

NCT00004402

Organization ID

199/13284

Secondary IDs ^††

MUSC-FDR000768

Study Sponsor ^†

FDA Office of Orphan Products Development

Collaborators ^††

Medical University of South Carolina

Investigators ^†

Study Chair:

L. Lyndon Key, Jr.

Medical University of South Carolina

Information Provided By

FDA Office of Orphan Products Development

Verification Date

January 2001

First Received Date ^†

October 18, 1999

Last Updated Date

June 23, 2005

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.