Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Pati… (NCT03047746) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
United States50 participantsStarted 2017-02-01
Plain-language summary
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.
Who can participate
Age range
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
. Must meet criteria for severe or very severe aplastic anemia (AA), defined by:
. absolute neutrophil count (ANC) \< 500/µL (severe) or \< 200/µL (very severe). Current ANC or last ANC prior to start of Granulocyte-colony stimulating factor (G-CSF) may be used.
. platelets \< 30,000/µL or transfusion dependence
. absolute reticulocyte count \< 40,000/µL
. Patients meeting other eligibility criteria may receive study therapy as the initial treatment approach provided an eligible unrelated or mismatched related ("haplo") donor is available
. Patients who have received prior immune suppression therapy will be eligible if they have refractory or relapsed disease defined as per treating clinician's judgement, at least 12 weeks after initiation of immune suppression therapy. Relapsed patients who previously met hematologic criteria for severe aplastic anemia do not have to meet these hematologic criteria for severe aplastic anemia at time of relapse to be eligible for transplant.
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 uses a process called TCR αβ depletion to remove certain T cells from the donor stem cells before transplant — can you explain how that's different from a standard unrelated donor transplant, and whether that approach might lower my risk of graft-versus-host disease?
2The trial is measuring both graft failure rates and how quickly neutrophils recover after transplant — based on what's been reported so far, how do those outcomes compare to what I might expect from a more conventional bone marrow transplant for my specific condition?
3Since this trial is no longer enrolling new patients, does that mean there are already published or preliminary results I could review, and would those results influence whether you'd still recommend this type of TCR αβ depletion approach for me?
4This trial accepts patients with acquired aplastic anemia, paroxysmal nocturnal hemoglobinuria, or inherited bone marrow failure syndromes — given my specific diagnosis, is a partially matched related or unrelated donor transplant even the right path, or should I try immunosuppressive therapy or another treatment first?
5Chronic and acute GVHD are both primary things this trial is tracking — what would managing GVHD look like for me day-to-day if I pursued this kind of transplant, and how would that affect my quality of life during recovery?
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
Rate of graft failure
Timeframe: Up to three years post-transplantation
2
Time to neutrophil engraftment
Timeframe: Up to 60 days post-transplantation
3
Incidence of acute graft vs. host disease (GVHD)
Timeframe: Up to 100 days post-transplantation
4
Incidence of chronic graft vs. host disease (GVHD)
. Patients must have testing (eg. Flow Cytometry demonstrating cells with absent cluster of differentiation 55 (CD55) or cluster of differentiation 59 (CD59) expression demonstrating a PNH clone in greater than 10% of peripheral blood red blood cells and/or granulocytes, along with clinical or laboratory evidence of intravascular hemolysis, such as:
. Patients who have small paroxysmal nocturnal hemoglobinuria (PNH) clones, no evidence of hemolysis, and meet criteria for severe or very severe AA as defined above, will be classified as acquired AA for treatment stratification.