Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors fo… (NCT04806347) | Clinical Trial Compass
Not Yet RecruitingPhase 1
Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults
12 participantsStarted 2026-06
Plain-language summary
This study is being done to see if the investigators can take peripheral blood stem cells from either an adult family member or a closely matched unrelated donor, and run them through a special lab instrument to remove alpha/beta T cells and B cells and then give them to the patient to treat disease. This is an experimental way of doing a hematopoietic stem cell transplant (HSCT). The investigators want to see if the new stem cells will grow without bad graft vs. host disease (GVHD). This treatment approach is experimental in the United States.
Who can participate
Age range
3 Months – 25 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:
* No Human leukocyte antigen (HLA) identical sibling available AND
* NO HLA matched unrelated donor available OR urgent need of HSCT precludes time necessary to search for suitable HLA matched unrelated donor AND
* Haploidentical donor OR closely matched unrelated donor available and willing to undergo mobilization and apheresis
* If subject has genetically confirmed inherited bone marrow failure, related donor must be evaluated for this disorder and testing must be negative.
* If subject has sickle cell disease, donor must be unaffected or have only sickle cell trait
* Patient must be diagnosed with one of the following diseases or disorders:
* Hemoglobinopathies
* Sickle Cell Disease for patients ≤ 21 years of age for whom hydroxyurea has been trialed for at least six months, and failed
* Thalassemia Major for patients ≤ 21 years of age
* Acquired Bone Marrow Failure Syndromes
* Paroxysmal Nocturnal Hemoglobinuria with bone marrow failure
* Myelodysplastic Syndromes (lower risk)
* Severe acquired aplastic anemia
* Inherited Bone Marrow Failure Syndromes
* Fanconi Anemia
* Diamond Blackfan Anemia
* Dyskeratosis Congenita and related telomere disorders
* Congenital Thrombocytopenia Syndromes
* Severe Congenital Neutropenia
* Shwachman-Diamond Syndrome
* Inborn Errors of Immunity (IEI) or Primary Immune Regulatory Disorders (PIRD)
* Wiskott-Aldrich syndrome (WAS)
* Chronic granulomatous disea…
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
Incidence of grade III-IV acute graft-versus-host disease (GVHD)