NCT05126849: HAPLO-EMPTY

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

Recruiting now Phase 2 Last updated 24 November 2023

What this trial tests

Phase 2 trial testing Allogenic transplantation in Refractory Idiopathic Aplastic Anemia in 31 participants. Currently enrolling.

Timeline

6 January 2022

Primary endpoint
6 January 2027

6 January 2027

Quick facts

Lead sponsor	Assistance Publique - Hôpitaux de Paris
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	other
Enrollment	31
Start date	6 January 2022
Primary completion	6 January 2027
Estimated completion	6 January 2027
Sites	35 locations across France

Drugs / interventions tested

• Allogenic transplantation

Conditions studied

• Refractory Idiopathic Aplastic Anemia — all drugs for Refractory Idiopathic Aplastic Anemia →
• Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation — all drugs for Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation →

Sponsor

Assistance Publique - Hôpitaux de Paris — full company profile →

Who can join

Adults 3 to 35, any sex, with Refractory Idiopathic Aplastic Anemia or Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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-threatening infections, and bleeding. During the past 2 decades, there has been a significant decrease in infection-related mortality in patients with SAA unresponsive to initial IST but clonal evolution including paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome (MDS), and acute myeloid leukemia (AML) still occur in the long-term with a grim prognosis. Overall, the overall survival of such patients with acquired refractory SAA to ELT is about 60-70% at 2 years.

Hematopoietic stem cell transplantation (HSCT) using alternative donor sources (i.e., mismatched unrelated donors, cord blood (CBT), and haplo-identical family donors) may be curative in patients with refractory SAA, despite carrying much higher rates of complications than in transplantations from matched related or unrelated donors. Recently, our group showed that CBT is a valuable curative option for young adults with refractory SAA. However, not all patients have available CB and CBT treatment related mortality is high in adult patients. Haploidentical (haplo) related donor Stem Cell Transplantation (haplo-SCT) have improved dramatically outcomes using T-cell replete grafts with administration of post-transplantation cyclophosphamide (PTCy). Preliminary results in a little number of patients with refractory SAA at Kings college (London, UK) and John Hopkins (Baltimore, USA) seem promising. The investigators retrospectively analyzed data from 36 patients (median age 42 years) transplanted between 2010 and 2017 in Europe on behalf of the SAA working party of the European Blood and Marrow Transplantation group. The 1-year overall survival was about 80% suggesting that this approach might be a valid option in this particular poor clinical situation.

The main objective of this study is to demonstrate a benefit in term of the 2-year overall survival rate from 60% (historical rates in patients with acquired refractory idiopathic aplastic anemia) up to 80% using haplo-SCT with PTCy.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.