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Haploidentical stem cell transplantation for patients with refractory aplastic anemia

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression: a Nationwide Phase II Study

Phase 2 Interventional Assistance Publique - Hôpitaux de Paris · NCT05126849

This study tests whether a new type of stem cell transplant from a half-matched donor can help people with severe aplastic anemia who haven't responded to other treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment31 (estimated)
Ages3 Years to 35 Years
SexAll
SponsorAssistance Publique - Hôpitaux de Paris Academic / other
Drugs / interventionscyclophosphamide
Locations35 sites (Amiens and 34 other locations)
Trial IDNCT05126849 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the use of haploidentical allogeneic hematopoietic stem cell transplantation combined with post-transplant cyclophosphamide in patients suffering from acquired refractory aplastic anemia. The study aims to improve outcomes for patients who have not responded to first-line immunosuppressive therapy and lack a matched unrelated donor. By utilizing a haploidentical donor, the trial seeks to enhance survival rates and reduce complications associated with severe aplastic anemia. Participants will be closely monitored for their response to the treatment and any potential side effects.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals aged 3 to 35 years with refractory acquired idiopathic aplastic anemia who lack a matched donor.

Not a fit: Patients with severe and uncontrolled infections or those who have a matched donor may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve survival rates and quality of life for patients with refractory aplastic anemia.

How similar studies have performed: Other studies have shown promising results with haploidentical stem cell transplantation in similar patient populations, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Aged from 3 to 35 years old
* Suffering from refractory acquired idiopathic aplastic anemia (at least one course of immunosuppression with anti-thymocyte globulin)
* Absence of geno-identical donor or 10/10 matched donor
* With identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
* Absence of donor specific antibody detected in the patient with a MFI ≥ 1500 (antibodies directed towards the distinct haplotype between donor and recipient)
* With usual criteria for HSCT :

  * ECOG(Eastern Cooperative Oncology Group) ≤ 2
  * No severe and uncontrolled infection
  * Cardiac function compatible with high dose of cyclophosphamide
* Adequate organ function: ASAT(aspartate transaminase) and ALAT (alanine aminotransferase) ≤ 2.5 N (the norm), total bilirubin ≤ 2N, creatinine \< 150 μmol/L
* With health insurance coverage
* Contraception methods must be prescribed during all the duration of the research. Women and men of childbearing age must use contraceptive methods within 12 months and 6 months after the last dose of cyclophosphamide, respectively.
* Having signed a written informed consent (2 parents for patients aged less than 18)

Exclusion Criteria :

* With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7 abnormalities and complex karyotype)
* With uncontrolled infection
* With seropositivity for HIV or HTLV-1 (Human T cell Leukemia) or active hepatitis B or C defined by a positive PCR (polymerase chain reaction) HBV (hepatitis B virus) or HCV (hepatitis C virus) and associated hepatic cytolysis
* Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
* Pregnant (βHCG positive) or breast-feeding.
* Who received live attenuated vaccine within 2 months before transplantation and during the research
* Uncontrolled coronary insufficiency, recent myocardial infarction \<6 month, current manifestations of heart failure, uncontrolled cardiac rhythm disorders, ventricular ejection fraction \<50%
* With heart failure according to NYHA (New York Heart Association) (II or more)
* Preexisting acute hemorrhagic cystitis
* Renal failure with creatinine clearance \<30ml / min
* With urinary tract obstruction
* Who receive the following treatments Phenytoin, Pentostatin, inhibitor of adenoside)
* Who have any debilitating medical or psychiatric illness, which preclude understanding the inform consent as well as optimal treatment and follow-up
* Under tutorship or curatorship

Where this trial is running

Amiens and 34 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Refractory Idiopathic Aplastic AnemiaHaploidentical Allogeneic Hematopoietic Stem Cell TransplantationAcquired refractory idiopathic aplastic anemiaHaplo identical hematopoietic stem cell transplantationPost-transplantation cyclophosphamide
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.