Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis (NCT00004402) | Clinical Trial Compass
CompletedPhase 3
Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
30 participantsStarted 1999-11
Plain-language 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.
Who can participate
Age range
10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.