Actively Recruiting
Evaluation of an Optimized Allogeneic Hematopoietic Stem Cell Transplantation Protocol With Post-transplant Cyclophosphamide in Patients Aged 40 to 60 Years Old With Acquired Aplastic Anemia Refractory or in Relapse After Immunosuppression
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2026-04-28
52
Participants Needed
26
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating an optimized allogeneic hematopoietic stem cell transplantation (HSCT) protocol using post-transplant cyclophosphamide (PTCy) in adults aged 40 to 60 years with acquired severe aplastic anemia (SAA) that is refractory or has relapsed after first-line immunosuppressive therapy. The study addresses the poor outcomes of HSCT in this population, focusing on preventing graft versus host disease (GvHD), a major cause of death following transplantation. This Phase 2 trial is sponsored by Assistance Publique - Hôpitaux de Paris and aims to improve survival and reduce complications using new strategies in HSCT. The treatment involves a conditioning regimen including thymoglobulin, fludarabine, cyclophosphamide, and total body irradiation, followed by transplantation of bone marrow stem cells. Post-transplant GvHD prevention includes cyclophosphamide given on days 3 and 4, along with tacrolimus and mycophenolate starting day 5. Additional measures include rituximab to prevent Epstein Barr Virus reactivation. The stem cell source is exclusively bone marrow, with specific cell count targets and granulocyte colony-stimulating factor administered post-transplant until neutrophil recovery. Participants will undergo extensive monitoring including blood counts, graft survival, GvHD incidence, infections, organ function, immune reconstitution, and quality of life assessments at various time points up to 24 months after transplantation. The primary outcome is survival free from GvHD and relapse two years post-transplant. This comprehensive follow-up aims to assess safety, effectiveness, and long-term outcomes of the optimized HSCT protocol in this patient group.
CONDITIONS
Brief Title
Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged from 40 to 60 years old
- Diagnosed with acquired refractory severe idiopathic aplastic anemia after at least 6 months of treatment with anti-thymocyte globulin, cyclosporine with Eltrombopag or in relapse
- Allograft validated in the National Multidisciplinary expertise meetings of the French reference centre for aplastic anemia
- Availability of a geno-identical donor, 10/10 matched donor, or haploidentical donor
- Absence of donor specific antibody with a mean fluorescence intensity (MFI) less than 1500
- ECOG performance status of 2 or less
- No severe or uncontrolled infection
- Cardiac function compatible with high dose cyclophosphamide
- Adequate organ function: ASAT and ALAT less than or equal to 3 times normal, conjugated bilirubin less than or equal to 2 times normal (or total bilirubin if conjugated not available), creatinine clearance at least 50 ml/min
- Health insurance coverage
- Use of effective contraceptive methods during the study and for specified periods after last cyclophosphamide dose for women of childbearing potential and men
- Signed informed consent
You will not qualify if you...
- Evidence of clonal evolution such as chromosome 7 abnormalities or complex karyotype
- Positive for HIV, HTLV-1-2, active hepatitis B or C with liver damage
- History of cancer in the last 5 years except certain skin or cervical cancers
- Pregnant or breastfeeding
- Received live virus vaccines within 2 months before transplantation or during the study
- Uncontrolled coronary insufficiency, recent heart attack within 6 months, heart failure with NYHA class II or higher, or ejection fraction below 50%
- Renal failure with creatinine clearance below 50 ml/min
- Contraindications or allergy to any study medications including conditioning regimen, GVHD prophylaxis, or infection prevention
- Debilitating medical or psychiatric illness preventing informed consent or follow-up
- Under legal protection such as tutorship or curatorship
- Receiving state medical aid
- Participation in another interventional trial involving medicinal products or cell therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 year
Participants undergo an allogeneic hematopoietic stem cell transplantation with a conditioning regimen followed by graft-versus-host disease prophylaxis and infection prevention.
Multiple visits including daily care during hospitalization and weekly visits for up to 12 months
Duration - Up to 2 years
Participants are monitored for graft survival, immune reconstitution, infections, and quality of life for up to 2 years after transplantation.
Regular visits at 1, 3, 6, 12, and 24 months post-transplant
Trial Site Locations
Total: 26 locations
1
Saint Louis hospital
Paris, France, France, 75010
Actively Recruiting
2
CHU Amiens
Amiens, France
Actively Recruiting
3
CHU Angers
Angers, France
Actively Recruiting
4
CHU Besançon
Besançon, France
Actively Recruiting
5
CHU Bordeaux
Bordeaux, France
Actively Recruiting
6
CHU Caen
Caen, France
Not Yet Recruiting
7
HNIA Percy
Clamart, France
Actively Recruiting
8
Hôpital d'Estaing
Clermont-Ferrand, France
Actively Recruiting
9
Hôpital Henri Mondor AP-HP
Créteil, France
Not Yet Recruiting
10
CHU Grenoble Alpes
Grenoble, France
Actively Recruiting
11
CHU Lille
Lille, France
Not Yet Recruiting
12
CHU Limoges
Limoges, France
Actively Recruiting
13
CHU Lyon Sud
Lyon, France
Actively Recruiting
14
Institut Paoli Calmettes
Marseille, France
Not Yet Recruiting
15
CHU Montpellier
Montpellier, France
Actively Recruiting
16
CHRU Nancy
Nancy, France
Actively Recruiting
17
CHU Nantes
Nantes, France
Actively Recruiting
18
CHU Nice
Nice, France
Actively Recruiting
19
Hopital Necker - APHP
Paris, France
Actively Recruiting
20
Hôpital La Pitié Salpêtrière AP-HP
Paris, France
Not Yet Recruiting
21
CHU Poitiers
Poitiers, France
Actively Recruiting
22
CHU Rennes
Rennes, France
Not Yet Recruiting
23
Henri Becquerel
Rouen, France
Not Yet Recruiting
24
CHU Saint Etienne
Saint-Etienne, France
Actively Recruiting
25
CHU Strasbourg
Strasbourg, France
Actively Recruiting
26
CHU Toulouse
Toulouse, France
Actively Recruiting
Research Team
R
Régis Peffault de Latour, MD PhD
J
Jérôme Lambert, MD PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here