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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	7 April 2015
Main ID:	NCT00004402
Date of registration:	18/10/1999
Prospective Registration:	Yes
Primary sponsor:	FDA Office of Orphan Products Development
Public title:	Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
Scientific title:
Date of first enrolment:	November 1999
Target sample size:	30
Recruitment status:	Completed
URL:	http://clinicaltrials.gov/show/NCT00004402
Study type:	Interventional
Study design:	Primary Purpose: Treatment
Phase:	Phase 3

Countries of recruitment

Contacts

Name:	L. Lyndon Key, Jr.
Address:
Telephone:
Email:
Affiliation:	Medical University of South Carolina

Key inclusion & exclusion 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

Age minimum: N/A
Age maximum: 10 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Osteopetrosis

Intervention(s)

Drug: interferon gamma

Drug: calcitriol

Primary Outcome(s)

Secondary Outcome(s)

Secondary ID(s)

MUSC-FDR000768

199/13284

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Medical University of South Carolina

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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