Status:
NOT_YET_RECRUITING
Up-front Matched Unrelated Donor Transplantation in Pediatric Patients With Idiopathic Aplastic Anemia
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Conditions:
Idiopathic Aplastic Anemia
Severe Aplastic Anemia
Eligibility:
All Genders
Up to 18 years
Phase:
PHASE2
Brief Summary
Pediatric patients with idiopathic aplastic anemia (AA) respond better than adults to immunosuppressive therapy (IST) but the long-term risks of relapse, ciclosporine dependence, and clonal evolution ...
Eligibility Criteria
Inclusion
- age\<18years old
- Pediatric patients aged less than 18 years with idiopathic aplastic anemia and an indication for treatment (severe aplastic anemia or moderate aplastic anemia requiring transfusions)
- With a good probability to have a HLA-10/10 matched unrelated donor available (the patient needs to have at least 3 MUD identified within the book BMDW (Bone Marrow Donors Worldwide) or using the easy match software to be included)
- With usual criteria for allo-SCT:
- Lansky \>70% for those below 16 years and Karnofsky \> 70% for those above 16 years
- No severe and uncontrolled infection
- Adequate organ function: ASAT and ALAT ≤ 5N\*, total bilirubin ≤ 2N, creatinine clearance \> 70% of higher normal values for age.
- With health insurance coverage
- Contraception methods\*\* for young girl and men of childbearing age must be prescribed during all the duration of the research.
- Parents having read and understand the information note and signed a written informed consent (the patient's agreement depending on his age will be sought)
- \*because typical presentation of aplastic anemia post-hepatitis
- \*\* 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).
- For man in absence of permanent sterilization: condoms
Exclusion
- Patients :
- With a matched related donor available
- With uncontrolled infection
- With seropositivity for HIV or HTLV-1 or active hepatitis B or C defined by a positive PCR HBV or HCV and associated hepatic cytolysis
- Renal failure with creatinine clearance below 70% of higher normal values for age
- Pregnant (βHCG positive) or breast-feeding
- With Heart failure according to NYHA (II or more)
- Preexisting acute hemorrhagic cystitis
- Urinary tract obstruction
- Yellow fever vaccine within 2 months before transplantation
- Who have any debilitating medical or psychiatric illness, which preclude understanding the inform consent as well as optimal treatment and follow-up (depending of his age and understanding).
- With Contraindication to treatments used during the research
Key Trial Info
Start Date :
June 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2027
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT05419843
Start Date
June 1 2022
End Date
June 1 2027
Last Update
June 15 2022
Active Locations (0)
Enter a location and click search to find clinical trials sorted by distance.
No Results Found
We couldn’t find results for the location/zipcode entered or within the selected range. Please check your input or adjust your search.