Haploidentical stem cell transplantation for patients with refractory aplastic anemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• CHU Amiens
  Amiens, France Recruiting
• CHU Angers
  Angers, France Recruiting
• CHU Besancon
  Besançon, France Recruiting
• CHU Bordeaux
  Bordeaux, France Recruiting
• Hôpital du Haut-Lévêque
  Bordeaux, France Recruiting
• CHU Caen
  Caen, France Recruiting

Study contacts

• Study coordinator: Regis Peffault de Latour, Pr
• Email: regis.peffaultdelatour@aphp.fr
• Phone: +33142385073

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.