Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantati… (NCT07629947) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia
180 participantsStarted 2026-06-15
Plain-language summary
Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.
Traditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.
This multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.
Who can participate
Age range
18 Years – 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:
Patients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria. SAA is defined as bone marrow cellularity \<25% and at least two of the following peripheral blood criteria: absolute neutrophil count \<0.5 × 10⁹/L, platelet count \<20 × 10⁹/L, or reticulocyte count \<20 × 10⁹/L. vSAA is defined as SAA with an absolute neutrophil count \<0.2 × 10⁹/L.
Age 18 to 40 years. Availability of a haploidentical related donor. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
Adequate organ function, defined as:
Cardiac function: left ventricular ejection fraction ≥50% and no severe arrhythmia; Hepatic function: total bilirubin ≤2 times the upper limit of normal and alanine aminotransferase/aspartate aminotransferase ≤3 times the upper limit of normal; Renal function: creatinine clearance ≥60 mL/min. No active infection. For women of childbearing potential, a negative pregnancy test is required. All patients must agree to use effective contraception during the study period.
Written informed consent provided by the patient or legal representative.
Exclusion Criteria:
Presence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).
Prior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.
Availability of an HLA-matched sibling donor. Active uncontrolled infection, including u…
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
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation