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New treatment option for Aplastic Anemia
Official title Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years
ClinicalTrials.gov ID: NCT06646497
What this study is testing
What is Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow?
Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for aplastic anemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 40 to 60
You may be able to join if
- 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...
- 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...
You likely can't join if
- Patients:
- With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7...
- 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
See the full eligibility criteria
- 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:
- 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...
- 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
- 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...
- 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...
- 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 treatment 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...
The study team makes the final eligibility decision.
Where it's taking place
- Paris, France, France
- Amiens, France
- Angers, France
- Besançon, France
- Bordeaux, France
- Caen, France
- Clamart, France
- Clermont-Ferrand, France
- Créteil, France
- Grenoble, France
- Lille, France
- Limoges, France
- Lyon, France
- Marseille, France
- Montpellier, France
- Nancy, France
- Nantes, France
- Nice, France
- Paris, France
- Poitiers, France
+ 5 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 40 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Paris, France, France; Amiens, France; Angers, France; Besançon, France; Bordeaux, France; Caen, France and 19 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.