Stem Cell Transplantation for Children Affected With Osteopetrosis (NCT00145587) | Clinical Trial Compass
TerminatedNot Applicable
Stem Cell Transplantation for Children Affected With Osteopetrosis
Stopped: Due to the principal investigator having left the institution.
United States15 participantsStarted 2004-07
Plain-language summary
Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation. Stem cells provided from an HLA identical matched sibling donor is the standard of care, but not feasible for the majority of patients. In addition, due to the potentially rapid progression of this disease, the time to identify a suitable HLA matched unrelated donor is not optimal. Therefore this study is designed to test the hypothesis that children with osteopetrosis can properly engraft hematopoietic stem cells that are donated from a partially matched parental donor, or "haploidentical" stem cell donor that are processed on the investigational device, CliniMACS selection system.
Who can participate
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.
Inclusion Criteria:
* Clinical diagnosis of malignant osteopetrosis as documented by bone marrow biopsy and radiographic imaging
* A suitable hematopoietic stem cell donor is available
Exclusion Criteria:
* Participant has the Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant
* Symptomatic cardiac disease or evidence of significant cardiac dysfunction by ECHO (shortening fraction \<30%)
* Creatinine clearance ≤ 40ml/min/1.73m\^2
* Bilirubin ≥ 3mg/dL
* SGPT ≥ 500 U/L
* Evidence of current severe infection which would preclude ablative chemotherapy or a successful transplantation
* Karnofsky or Lansky score \< 70 noting expected abnormalities
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.