Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Jun 6, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a type of treatment called haploidentical transplant for patients with Fanconi disease, a condition that affects blood production and makes it difficult for the body to fight infections. The goal of the study is to see if this transplant can help improve the survival rates of patients who don't have a fully matched donor available. This is especially important for patients who are facing severe blood-related issues, such as very low blood cell counts or have experienced a worsening of their condition.

To be eligible for the study, participants need to be between 6 months and 60 years old and have a confirmed diagnosis of Fanconi disease. They should also have severe blood count problems and a haploidentical donor who is unaffected by the disease. Participants will need to agree to take part in the study after understanding all the information provided. This trial is not yet recruiting, so no one is enrolled at this time. If you or a loved one meets the criteria, this could be an important opportunity to help advance treatment options for Fanconi disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosis of Fanconi disease confirmed by chromosome breakage test and/or genetic analysis
• • aged between 6 months and 60 years
• • with severe pancytopenia (2 of the following criteria: reticulocytes \< 60 G/L, PNN \< 0.5 G/L and/or platelets \< 20 G/L or patients with more than 6 transfusions in the last 12 months)
• • with clonal progression (poor prognostic cytogenetics, myelodysplastic syndrome or acute leukaemia)
• • with an unaffected haploidentical donor
• • having signed the consent after having read the information note, consent of both parents for minors, of the guardian for patients under guardianship
• • having a social security scheme (beneficiary or entitled person)
• Exclusion Criteria:
• • with an unaffected matched related or HLA 10/10 matched unrelated donnor
• • under guardianship