Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression

Recruiting Phase 2 Interventional Study
Refractory Idiopathic Aplastic Anemia Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 – 35
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Other
Participants needed
31 (estimated)
Sponsor
Assistance Publique - Hôpitaux de Paris · Other
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About This Trial
Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor. Recently, the use of eltrombopag (ELT) has shown blood count improvements in 40% of these patients. However, most refractory patients do not respond to ELT or other second-line treatment and are therefore exposed to life-…
Trial Locations
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Eligibility 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…
Contacts

Regis Peffault de Latour, Pr

+33142385073

regis.peffaultdelatour@aphp.fr

CONTACT

Jérôme Lambert, Pr

+33142499742

jerome.lambert@u-paris.fr

CONTACT