This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the time to neutrophil and platelet engraftment, to determine the time to immune reconstitution (including normalization of T, B and natural killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant disease.
Who can participate
Age range
40 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:
* General Eligibility (All Patients)
* Patient must be \< or = 40 years of age. Patients with sickle cell anemia must be at least 2 years of age.
* Patient or the patient's legally authorized guardian must be fully informed about their illness and the investigational nature of the study protocol (including foreseeable risks and possible side effects) and must sign an informed consent in accordance with the institutional policies approved by the U.S. Department of Health and Human Services.
* Approval for the use of this treatment protocol by the individual institution's Human Rights Committee must be obtained, in accordance with the institutional assurance policies of the U. S. Department of Health and Human Services.
* Human leukocyte antigen (HLA) typing will be performed by high-resolution molecular DNA typing for HLA Class I A, B, and C and HLA Class II DRB1 and DQB1 alleles.
* Unrelated donor: An 8/10, 9/10 or 10/10 matched unrelated adult donor (MUD) will be required for study entry.
* Related Donor: A 5/10, 6/10, 7/10, 8/10, 9/10 or 10/10 matched (or partially matched) family donor will be required for study entry.
* Non-malignant Disorders per protocol.
* Hemoglobinopathies per protocol.
* Requirement for CD34+ stem cell selection for a second infusion of stem cells following an allogeneic stem cell transplant from a related or unrelated adult donor.
* Additional eligibility for patients with non-malignant disorders receivi…
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.
1This trial is specifically trying to reduce acute graft versus host disease by selecting CD34+ stem cells — does my diagnosis of [condition] make me a good candidate to discuss this approach, and how does GVHD risk factor into my current treatment plan?
2Since this is a Phase 1/2 trial that is no longer actively enrolling, are there any published or preliminary results from this study that you've seen, and what do they suggest about how well the CD34+ selection approach controlled GVHD?
3CD34+ stem cell selection involves filtering the donor stem cells before transplant — can you explain what that process means for my situation, and are there any trade-offs in terms of immune recovery or engraftment that I should understand?
4Because the trial is no longer recruiting, is there a similar or follow-on study available that uses this same CD34+ selection method, or has this approach become part of standard transplant care at this center?
5How does this CD34+ selection strategy compare to other ways of preventing GVHD in allogeneic transplants — like using standard immunosuppressive drugs — and would those alternatives be a better first path given where I am right now?
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
Incidence of acute graft versus host disease (GVHD)