Actively Recruiting
Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2026-04-28
52
Participants Needed
26
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Outcomes for adult patients with Severe Aplastic Anemia (SAA) aged more than 40 years who are refractory or in relapse after first-line IST remain poor. Hematopoietic stem cell transplantation (HSCT) is the unic valid therapeutic option but results have always been disappointing in patients aged 40 years or older. The first cause of death after HSCT in those refractory/relapse SAA patients is still graft versus host disease (GvHD). Recently, new strategies to prevent GvHD, including T-cell replete grafts with administration of post-transplantation cyclophosphamide (PTCy), have revolutionized the field, notably in haplo-identical donor setting. Using marrow as source of stem cells and a PTCy strategy not only in haplo-identical donor setting but also in case of an available matched sibling or unrelated donor might prevent drastically GvHD and eventually be practice changing. Evaluating this new strategy is the main objectives of "APARR".
CONDITIONS
Official 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 approved by the National Multidisciplinary expertise meetings of the French reference centre for aplastic anemia
- Has an available geno-identical donor, 10/10 matched donor, or haploidentical donor
- No donor-specific antibodies detected with a mean fluorescence intensity less than 1500
- Meets usual criteria for hematopoietic stem cell transplantation including ECOG performance status ≤ 2, no severe uncontrolled infection, cardiac function compatible with high-dose cyclophosphamide, adequate organ function (ASAT and ALAT ≤ 3 times normal, bilirubin ≤ 2 times normal, creatinine clearance ≥ 50 ml/min)
- Has health insurance coverage
- Women of childbearing potential and men agree to use contraceptive methods during the study and for 12 months and 6 months after last cyclophosphamide dose, respectively
- Has signed written informed consent
You will not qualify if you...
- Evidence of clonal evolution excluding isolated bone marrow cytogenetic abnormalities except chromosome 7 abnormalities and complex karyotypes
- Seropositive for HIV, HTLV-1-2, or active hepatitis B or C with liver damage
- Cancer diagnosis within the last 5 years except basal cell carcinoma of the skin or in situ cervical carcinoma
- Pregnant or breastfeeding
- Received yellow fever vaccine or other live virus vaccines within 2 months before transplantation or during the study
- Uncontrolled coronary insufficiency, recent myocardial infarction within 6 months, heart failure NYHA class II or higher, or ventricular ejection fraction below 50%
- Renal failure with creatinine clearance less than 50 ml/min
- Any contraindication to trial medications including conditioning regimen, GVHD prophylaxis, EBV prevention, or infection prophylaxis
- Known allergy or intolerance to trial medications or excipients
- Any debilitating medical or psychiatric illness preventing informed consent or proper treatment and follow-up
- Under legal protection (tutorship or curatorship)
- Receiving state medical aid
- Participating 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
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
CONTACT
J
Jérôme Lambert, MD PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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