Aplastic AnemiaPhase 2APARRNCT06646497
Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years
A Phase 2 study testing Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow in people with Aplastic Anemia, run by Assistance Publique - Hôpitaux de Paris at 26 sites in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 40 to 60 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 26 sitesFrance
- Study dates
- 24 Jan 2025to 24 Jan 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Aged from 40 to 60 years old
- Suffering from acquired refractory severe idiopathic aplastic anemia after at least 6 months 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
- With an available geno-identical donor or 10/10 matched donor or haploidentical donor
- With the absence of donor specific antibody detected in the patient with a MFI < 1500 (antibodies to the distinct haplotype between donor and recipient)
- Usual criteria for HSCT:
Show all 20Show fewer
- ECOG ≤ 2
- No severe and uncontrolled infection
- Cardiac function compatible with high dose of cyclophosphamide
- With an adequate organ function ASAT and ALAT ≤ 3N, conjugated bilirubin ≤ 2N (or total bilirubin ≤ 2N if not available), clearance creatinine ≥ 50ml / min
- With health insurance coverage
- Women of childbearing potential and men must use contraceptive methods during their participation to the research and for 12 months and 6 months after the last dose of cyclophosphamide, respectively.
- Having signed a written informed consent
- NB: The authorized contraceptive methods are: For women of childbearing age and in absence of permanent sterilization:
- oral, intravaginal or transdermal combined hormonal contraception,
- oral, injectable or transdermal progestogen-only hormonal contraception,
- intrauterine hormonal-releasing system (IUS),
- sexual abstinence (need to be evaluated in relation to the duration of clinical trial and the preferred and usual lifestyle of the participants).
- For men in absence of permanent sterilization: sexual abstinence, condoms.
- Individuals must meet all of the inclusion criteria as verified at the screening / inclusion visit to be eligible to participate at the study.
Who cannot join
- Patients:
- With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7 abnormalities and complex karyotype).
- With seropositivity for HIV or HTLV-1-2 or active hepatitis B or C 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
- Yellow fever vaccine and all others live virus vaccines within 2 months before transplantation and during the research
Show all 15Show fewer
- With uncontrolled coronary insufficiency, recent myocardial infarction < 6-month, current manifestations of heart failure according to NYHA (II or more), ventricular ejection fraction <50%
- With renal failure with creatinine clearance <50ml /min
- Any contraindication mentioned in the SmPC and the Investigator's brochure of all medicinal products planned to be used in the trial including conditioning regimen, GVHD prophylaxis, prevention of EBV reactivation, infection prophylaxis
- Known allergy or intolerance to all medicinal products and/or excipients planned to be used in the trial including conditioning regimen, GVHD prophylaxis, prevention of EBV reactivation, infection prophylaxis, according to Investigator's brochure and SmPC.
- Who have any debilitating medical or psychiatric illness, which precludes understanding the inform consent as well as optimal treatment and follow-up
- Under legal protection (tutorship or curatorship)
- Under state medical aid
- Participation to another interventional trial on a medicinal product or cell therapy
- Individuals meeting any of the exclusion criteria as verified at the screening / inclusion visit will be ineligible to participate at the study.
Trial locations and contacts
26 sites in 1 country. Contact the study team or a site near you to ask about taking part.
26 sites in 1 country
France26 sites, 26 recruiting
- Recruiting
CHU Amiens
Amiens
Magalie Joris, +33 3 22 45 54 23 ext. +33, joris.magalie@chu-amiens.fr
- Recruiting
CHU Angers
Angers
Sylvie François, +33 2 41 35 44 82 ext. +33, syfrancois@chu-angers.fr
- Recruiting
CHU Besançon
Besançon
Etienne Daguindau, +33370632285 ext. +33, edaguindau@chu-besancon.fr
- Recruiting
CHU Bordeaux
Bordeaux
Edouard Forcade, +33 5 57 65 65 11 ext. +33, edouard.forcade@chu-bordeaux.fr
- Not yet recruiting
CHU Caen
Caen
Sylvain Chantepie, +33231272073, Chantepie-s@chu-caen.fr
- Recruiting
HNIA Percy
Clamart
Johanna Konopacki, +33 1 41 46 63 03 ext. +33, johanna.konopacki@intradef.gouv.fr
- Recruiting
Hôpital d'Estaing
Clermont-Ferrand
Aurélie Ravinet, +33 4 73 75 00 65 ext. +33, aravinet@chu-clermontferrant.fr
- Not yet recruiting
Hôpital Henri Mondor AP-HP
Créteil
Sébastien Maury, +33 1 49 81 45 26 ext. +33, sebastien.maury@aphp.fr
- Recruiting
CHU Grenoble Alpes
Grenoble
Claude-Eric Bulabois, +33 4 76 76 92 09 ext. +33, cebulabois@chu-grenoble.fr
- Not yet recruiting
CHU Lille
Lille
Micha Srour, Micha.SROUR@chu-lille.fr
Micha Srour, +33 3 20 44 42 85 ext. +33, Micha.SROUR@chu-lille.fr
- Recruiting
CHU Limoges
Limoges
Pedro de Lima Prata, +33 5 55 05 88 67 ext. +33, Pedro.PRATA@chu-limoges.fr
- Recruiting
CHU Lyon Sud
Lyon
Vincent Alcazer, +33 4 78 86 22 59 ext. +33, vincent.alcazer@chu-lyon.fr
- Not yet recruiting
Institut Paoli Calmettes
Marseille
Raynier Devillier, +33 4 91 22 33 33, DEVILLIERR@ipc.unicancer.fr
- Recruiting
CHU Montpellier
Montpellier
Patrice Ceballos, +33 4 67 33 32 68 ext. +33, p-ceballos@chu-montpellier.fr
- Recruiting
CHRU Nancy
Nancy
Simona Pagliuca, +33383155259 ext. +33, s.pagliuca@chru-nancy.fr
- Recruiting
CHU Nantes
Nantes
Alice Garnier, +33 2 40 08 42 28 ext. +33, alice.garnier@chu-nantes.fr
- Recruiting
CHU Nice
Nice
Michael Loschi, +33 4 92 03 52 11 ext. +33, loschi.m@chu-nice.fr
- Recruiting
Saint Louis hospital
Paris, France, 75010
Régis Peffault de La Tour, MD PhD, +33142385073
- Recruiting
Hopital Necker - APHP
Paris
Felipe Suarez, +331 44 49 51 88 ext. +33, felipe.suarez@aphp.fr
- Not yet recruiting
Hôpital La Pitié Salpêtrière AP-HP
Paris
Stéphanie Nguyen-Quoc, +33 1 42 17 61 84 ext. +33, stephanie.nguyen-quoc@aphp.fr
- Recruiting
CHU Poitiers
Poitiers
Natacha Maillard, +33 5 16 60 43 29, natacha.maillard@chu-poiters.fr
- Not yet recruiting
CHU Rennes
Rennes
Jean-Baptiste Mear, +33299284321 ext. +33, jean-baptiste.mear@chu-rennes.fr
- Not yet recruiting
Henri Becquerel
Rouen
Anne-Lise Menard, +33 2.32.08.24.98 ext. +33, anne-lise.menard@chb-unicancer.fr
- Recruiting
CHU Saint Etienne
Saint-Etienne
Jérôme Cornillon, +334 77 82 24 55 ext. +33, Jerome.Cornillon@chu-st-etienne.fr
- Recruiting
CHU Strasbourg
Strasbourg
Bruno Lioure, +33 3 68 76 74 03 ext. +33, bruno.lioure@chru-strasbourg.fr
- Recruiting
CHU Toulouse
Toulouse
Anne Huynh, +33 5 31 15 63 31 ext. +33, huynh.anne@iuct-oncopole.fr
About this study
Official title: 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
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
- Aplastic Anemia
- Being tested
- Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow
- Keywords
- Refractory/relapse idiopathic anemia, Idiopathic aplastic anemia, Hematopoietic stem cell transplantation, Post-transplantation cyclophosphamide
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone MarrowBiological
- Conditioning regimen Thymoglobulin (0.5/mg/kg à D-9, 2 mg /kg at D-8 and 2.5 mg/kg à D-7), Fludarabine (30mg/m2/day i.v: day -6 to day -2), pre-transplant, Cyclophosphamide (14.5 mg/kg/day i.v: day -6 and day -5), and Total Body Irradiation (2 Gray on day -1).
- Stem cell source Bone Marrow only. Target of 4 × 10^8 nucleated cells/kg recipient body weight. Granulocyte colony stimulating factor is given subcutaneously starting on day +5 at 5 mg/ kg/day until the absolute neutrophil count is greater than 1.5 × 10^9/L for 3 days.
- GVHD prophylaxis Cyclophosphamide 50 mg/Kg/day at D+3 and D+4. Tacrolimus (0,2 à 0,3 mg/kg/day per os divided into 2 doses or 0.05 to 0.1 mg/kg/d IVSE) and mycophenolate (MMF) will begin from D+5. In absence of GvHD, MMF will be stopped between D35 and D45 and Tacrolimus at day 365.
- Prevention of EBV reactivation Rituximab 150mg/m2 intravenously at Day+5 post HSCT (except patients and their donor with EBV serology and EBV PCR negative).
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow | Experimental | Not described Receives: Biological: Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow |
What is being measured
The primary outcome is the main question the study is designed to answer.
GRFS (Graft Versus Host Disease (GvHD) and Relapse/rejection-Free Survival)
GRFS is a composite right-censored endpoint, defined as the time from HSCT to the first of the following events:
- primary graft failure, defined as the absence of engraftment from aplasia at day 60 after graft (D0) (i.e., persistence of neutrophils< 500 AND platelets < 20 Giga/L)
- secondary graft failure, defined as the reoccurrence of aplasia after engraftment (defined as both occurrence of neutrophils< 500 for 3 days and platelets < 20 Giga/L for 7 consecutive days)
- grade 3-4 acute GVHD, according to the MAGIC CONSORTIUM 2016
- severe chronic GVHD, according to the NIH classification
- death, whatever the cause
Measured: 2 years after transplantation
Secondary outcomes (45)
Neutrophil engraftment
Neutrophils engraftment will be defined as first day of 3 consecutive days with neutrophils >0.5 G/L.
With donor chimerism> 85% on the total blood.
Measured: At day 100
Platelets engraftment
Platelets engraftment will be defined as first day of 7 consecutive days with platelets >20 G/L.
With donor chimerism> 85% on the total blood.
Measured: At day 100
Absolute number of neutrophils
Measured: At 1 month
Absolute number of neutrophils
Measured: At 3 months
Absolute number of neutrophils
Measured: At 6 months
Absolute number of neutrophils
Measured: At 12 months
Absolute number of neutrophils
Measured: At 24 months
Absolute number of neutrophils
Measured: At day of last platelet and red blood cell transfusions (up to 24 months)
Absolute number of platelets
Measured: At 1 month
Absolute number of platelets
Measured: At 3 months
Absolute number of platelets
Measured: At 6 months
Absolute number of platelets
Measured: At 12 months
Absolute number of platelets
Measured: At 24 months
Absolute number of platelets
Measured: At day of last platelet and red blood cell transfusions (up to 24 months)
Acute GvHD incidence grade 2-4
Measured: At 3 months
Chronic GvHD incidence
Measured: At 24 months
Severe chronic GvHD
Measured: At 24 months
Secondary graft failure
Measured: At 12 months
Secondary graft failure
Measured: At 24 months
Severe infections
CTCAE grade 3-4
Measured: At 1 month
Severe infections
CTCAE grade 3-4
Measured: At 3 months
Severe infections
CTCAE grade 3-4
Measured: At 6 months
Severe infections
CTCAE grade 3-4
Measured: At 12 months
Severe infections
CTCAE grade 3-4
Measured: At 24 months
Incidence of cardiac toxicities
Measured: At 12 months
Incidence of Epstein Barr Virus (EBV) infection
Measured: At 12 months
Incidence of CytoMegaloVirus (CMV) infection
Measured: At 12 months
Mortality
Measured: At 12 months
Mortality
Measured: At 24 months
Overall survival
Measured: At 12 months
Overall survival
Measured: At 24 months
Quality Of Life questionnaire
Quality of life will be evaluated using PedsQL questionnaire. Scores varies from 0 to100, with higher scores associated with better health-related quality of life
Measured: Before transplantation - at baseline day 0
Quality Of Life questionnaire
Quality of life will be evaluated using PedsQL questionnaire. Scores varies from 0 to100, with higher scores associated with better health-related quality of life
Measured: At 6 months
Quality Of Life questionnaire
Quality of life will be evaluated using PedsQL questionnaire. Scores varies from 0 to100, with higher scores associated with better health-related quality of life
Measured: At 12 months
Quality Of Life questionnaire
Quality of life will be evaluated using PedsQL questionnaire. Scores varies from 0 to100, with higher scores associated with better health-related quality of life
Measured: At 24 months
Chimerism
Proportion of patients with a donor chimerism of 85% or more
Measured: At 1 month
Chimerism
Proportion of patients with a donor chimerism of 85% or more
Measured: At 3 months
Chimerism
Proportion of patients with a donor chimerism of 85% or more
Measured: At 6 months
Chimerism
Proportion of patients with a donor chimerism of 85% or more
Measured: At 12 months
Chimerism
Proportion of patients with a donor chimerism of 85% or more
Measured: At 24 months
Immune reconstitution
Immune reconstitution will be done by analyzing T, B, NK, regulatory T cell levels in the peripheral blood. All have the same unit measure namely absolute numbers/microL.
Measured: At 1 month
Immune reconstitution
Immune reconstitution will be done by analyzing T, B, NK, regulatory T cell levels in the peripheral blood. All have the same unit measure namely absolute numbers/microL.
Measured: At 3 months
Immune reconstitution
Immune reconstitution will be done by analyzing T, B, NK, regulatory T cell levels in the peripheral blood. All have the same unit measure namely absolute numbers/microL.
Measured: At 6 months
Immune reconstitution
Immune reconstitution will be done by analyzing T, B, NK, regulatory T cell levels in the peripheral blood. All have the same unit measure namely absolute numbers/microL.
Measured: At 12 months
Immune reconstitution
Immune reconstitution will be done by analyzing T, B, NK, regulatory T cell levels in the peripheral blood. All have the same unit measure namely absolute numbers/microL.
Measured: At 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2024
- Study start24 Jan 2025
- Primary completion24 Jan 2030 (estimated)
- Study completion24 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A phase II multicenter, national, prospective, single-arm trial
- Masking
- None (open label)
- Enrolment
- 52 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06646497
- Sponsor's protocol ID
- APHP230832
- Acronym
- APARR
- Status last verified
- Apr 2026
- First submitted
- 13 Sep 2024
- Last updated
- 28 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06646497
- Acronym / sponsor ID
- APARR / APHP230832
- Conditions
- Aplastic Anemia
- Keywords
- Refractory/relapse idiopathic anemia, Idiopathic aplastic anemia, Hematopoietic stem cell transplantation, Post-transplantation cyclophosphamide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 52 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 24 Jan 2025 (actual)
- Primary completion
- 24 Jan 2030 (estimated)
- Study completion
- 24 Jan 2030 (estimated)
- First posted
- 17 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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".
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A phase II multicenter, national, prospective, single-arm trial
| Arm | Type | Receives |
|---|---|---|
| Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow | Experimental | Biological: Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow |
Who may qualify
- Ages
- 40 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (20 listed)
- Aged from 40 to 60 years old
- Suffering from acquired refractory severe idiopathic aplastic anemia after at least 6 months 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
- With an available geno-identical donor or 10/10 matched donor or haploidentical donor
- With the absence of donor specific antibody detected in the patient with a MFI < 1500 (antibodies to the distinct haplotype between donor and recipient)
Key exclusion criteria (15 listed)
- With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7 abnormalities and complex karyotype).
- With seropositivity for HIV or HTLV-1-2 or active hepatitis B or C 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
- Yellow fever vaccine and all others live virus vaccines within 2 months before transplantation and during the research
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow1. Conditioning regimen Thymoglobulin (0.5/mg/kg à D-9, 2 mg /kg at D-8 and 2.5 mg/kg à D-7), Fludarabine (30mg/m2/day i.v: day -6 to day -2), pre-transplant, Cyclophosphamide (14.5 mg/kg/day i.v: day -6 and day -5), and Total Body Irradiation (2 Gray on day -1). 2. Stem cell source Bone Marrow only. Target of 4 × 10^8 nucleated cells/kg recipient body weight. Granulocyte colony stimulating factor is given subcutaneously starting on day +5 at 5 mg/ kg/day until the absolute neutrophil count is greater than 1.5 × 10^9/L for 3 days. 3. GVHD prophylaxis Cyclophosphamide 50 mg/Kg/day at D+3 and D+4. Tacrolimus (0,2 à 0,3 mg/kg/day per os divided into 2 doses or 0.05 to 0.1 mg/kg/d IVSE) and mycophenolate (MMF) will begin from D+5. In absence of GvHD, MMF will be stopped between D35 and D45 and Tacrolimus at day 365. 4. Prevention of EBV reactivation Rituximab 150mg/m2 intravenously at Day+5 post HSCT (except patients and their donor with EBV serology and EBV PCR negative). | Biological | Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow |
Primary outcome
GRFS (Graft Versus Host Disease (GvHD) and Relapse/rejection-Free Survival)
Time frame: 2 years after transplantation
GRFS is a composite right-censored endpoint, defined as the time from HSCT to the first of the following events: * primary graft failure, defined as the absence of engraftment from aplasia at day 60 after graft (D0) (i.e., persistence of neutrophils< 500 AND platelets < 20 Giga/L) * secondary graft failure, defined as the reoccurrence of aplasia after engraftment (defined as both occurrence of neutrophils< 500 for 3 days and platelets < 20 Giga/L for 7 consecutive days) * grade 3-4 acute GVHD, according to the MAGIC CONSORTIUM 2016 * severe chronic GVHD, according to the NIH classification * death, whatever the cause
45 secondary outcomes
Neutrophil engraftment
Time frame: At day 100
Platelets engraftment
Time frame: At day 100
Absolute number of neutrophils
Time frame: At 1 month
Absolute number of neutrophils
Time frame: At 3 months
Absolute number of neutrophils
Time frame: At 6 months
Absolute number of neutrophils
Time frame: At 12 months
Absolute number of neutrophils
Time frame: At 24 months
Absolute number of neutrophils
Time frame: At day of last platelet and red blood cell transfusions (up to 24 months)
Absolute number of platelets
Time frame: At 1 month
Absolute number of platelets
Time frame: At 3 months
Absolute number of platelets
Time frame: At 6 months
Absolute number of platelets
Time frame: At 12 months
Absolute number of platelets
Time frame: At 24 months
Absolute number of platelets
Time frame: At day of last platelet and red blood cell transfusions (up to 24 months)
Acute GvHD incidence grade 2-4
Time frame: At 3 months
Chronic GvHD incidence
Time frame: At 24 months
Severe chronic GvHD
Time frame: At 24 months
Secondary graft failure
Time frame: At 12 months
Secondary graft failure
Time frame: At 24 months
Severe infections
Time frame: At 1 month
Severe infections
Time frame: At 3 months
Severe infections
Time frame: At 6 months
Severe infections
Time frame: At 12 months
Severe infections
Time frame: At 24 months
Incidence of cardiac toxicities
Time frame: At 12 months
Incidence of Epstein Barr Virus (EBV) infection
Time frame: At 12 months
Incidence of CytoMegaloVirus (CMV) infection
Time frame: At 12 months
Mortality
Time frame: At 12 months
Mortality
Time frame: At 24 months
Overall survival
Time frame: At 12 months
Overall survival
Time frame: At 24 months
Quality Of Life questionnaire
Time frame: Before transplantation - at baseline day 0
Quality Of Life questionnaire
Time frame: At 6 months
Quality Of Life questionnaire
Time frame: At 12 months
Quality Of Life questionnaire
Time frame: At 24 months
Chimerism
Time frame: At 1 month
Chimerism
Time frame: At 3 months
Chimerism
Time frame: At 6 months
Chimerism
Time frame: At 12 months
Chimerism
Time frame: At 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2026.
Related trials for Aplastic Anemia
RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
26 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow in people with Aplastic Anemia, run by Assistance Publique - Hôpitaux de Paris at 26 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 28 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Régis Peffault de Latour, MD PhD (142385073 ext. +33, regis.peffaultdelatour@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GRFS (Graft Versus Host Disease (GvHD) and Relapse/rejection-Free Survival) (time frame: 2 years after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2026; AzTrials last refreshed its copy on 7 Nov 2026.