Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non… (NCT00744692) | Clinical Trial Compass
CompletedPhase 1
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
United States22 participantsStarted 2008-10
Plain-language summary
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
Who can participate
Age range
21 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.
Inclusion Criteria:
* 0-21 years of age with a diagnosis of a immunodeficiency, congenital marrow failure syndrome, inborn error of metabolism, or hereditary anemia
* Appropriately matched related or unrelated umbilical cord blood unit with a cell dose ≥ 3 x 10e7cells/kg
* Performance score (lansky or karnofsky) greater than or equal to 70
* Adequate organ function (Creatinine ≤ 2.0 mg/dl and creatinine clearance ≥ 50 ml/min/1.73 m2; Hepatic transaminases (ALT/AST) ≤ 4 x normal; Shortening fraction \>26% or ejection fraction \>40% or \> 80% of normal value for age; Pulmonary function tests demonstrating CVC or FEV1/FVC of \>60% of predicted for age.)
* Informed consent
* Not pregnant or breast feeding
* Minimum life expectancy of at least 6 months
* HIV negative
* No uncontrolled infections at the time of cytoreduction
* Disease specific inclusion criteria
Exclusion Criteria:
* Patients with hemoglobinopathies \> 3 years of age
* UCB unit with a total nucleated cell count \< 3 x 10e7/kg or \> 2 antigen mismatching
* Available HLA-matched related living donor able to donate without previous UCB donation
* Allogeneic hematopoietic stem cell transplant within the previous 6 months
* Any active malignancy, MDS, or any history of malignancy
* Severe acquired aplastic anemia
* DLCO \< 60% of normal value for age; requirement for supplemental oxygen
* Uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms)
* Pregnancy o…
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.
What they're measuring
1
Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.