Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bo… (NCT00295971) | Clinical Trial Compass
CompletedPhase 1
Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease
United States21 participantsStarted 2005-04
Plain-language summary
RATIONALE: Giving chemotherapy and total body irradiation before a donor bone marrow transplant or peripheral blood stem cell transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects and best dose of donor T cells and antithymocyte globulin when given together with chemotherapy and total-body irradiation in treating young patients who are undergoing T-cell depleted donor stem cell transplant for myelodysplastic syndrome, leukemia, bone marrow failure syndrome, or severe immunodeficiency disease.
Who can participate
Age range
1 Year – 17 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.
DISEASE CHARACTERISTICS:
* Diagnosis of one of the following:
* Acute lymphoblastic leukemia in ≥ 2nd remission or delayed remission induction
* High-risk myelodysplastic syndromes
* Refractory anemia with excess blasts (RAEB)
* RAEB in transformation
* Chronic myelogenous leukemia in second chronic phase
* No accelerated phase (\> 5% blasts in marrow)
* Juvenile myelomonocytic leukemia
* Acute nonlymphoblastic leukemia in \> 1st remission or induction failure and \< 30% blasts in marrow
* Severe aplastic anemia, defined as absolute neutrophil count \< 500/mm\^3 and platelet and/or red blood cell transfusion dependent
* Unresponsive to immunosuppressive therapy
* No Fanconi's anemia
* Congenital marrow aplasias unresponsive to cytokines and transfusion dependent
* Inherited immunodeficiency disease involving neutrophils or lymphocytes, including any of the following:
* Chediak-Higashi disease
* Wiskott-Aldrich syndrome
* Combined immunodeficiency disease (Nezelof's)
* Hyper IgM syndrome
* No relapsed disease
* Haplocompatible related donor, including parent, cousin, aunt, uncle, grandparent, half-sibling, or sibling (≥ 12 years of age), available
* 2 or 3 HLA antigen mismatch
* At least a 3 HLA antigen genotypic match
* No closely matched related or unrelated donor available in sufficient time to do the transplant
PATIENT CHARACTERISTICS:
* No active hepatitis or cytomegalovirus infection
* Cardiac ejection fr…
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
Engraftment at 4 weeks post bone marrow transplantation through 100 days