Ignite Creation Date:
2024-05-06 @ 7:08 PM
Last Modification Date:
2024-10-26 @ 3:01 PM
Study NCT ID:
NCT05903365
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-06-15
First Post:
2023-06-06
Brief Title:
Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease
Sponsor:
Assistance Publique - Hôpitaux de Paris
Organization:
Assistance Publique - Hôpitaux de Paris
Study Overview
Official Title:
Etude Observationnelle de Suivi Des Greffes Haplo-identique Dans la Maladie de Fanconi
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HAPLO-FANCONI
Brief Summary:
This observational protocol will allow for an independent prospective evaluation of the improvement in survival of patients with Fanconi disease in hematological deadlock due to the absence of an HLA-identical donor and having received a haploidentical transplant
Detailed Description:
Fanconis disease is characterised by a constitutional defect in DNA repair which results in the occurrence of bone marrow failure and haematological malignancies mainly myeloid at the age of 40 the cumulative incidence of these two types of pathology reaches almost 100 The only curative treatment for haemtalogocial diseases is allogenic hematopoietic stem cell transplant Transplantation modalities must be adapted to the particular susceptibility of these patients to DNA bridging agents and radiotherapy HSC transplantation is indicated with an unaffected matched related or matched unrelated donor when the patient has severe bone marrow failure or a poor prognostic clonal evolution cytogenetic evolution or proven haemopathy Alternative transplants 910 pheno-identical haplo-identical and placental blood donors were no longer proposed in most cases due to the frequency of severe complications graft-versus-host disease viral infections and the catastrophic medium-term survival of around 40 Dufort Bone Marrow Transplant 2012 Gluckman Biol Blood Marrow Transplant 2007 The development over the last decade of new haploidentical or phenoidentical 910 transplant protocols with unmodified grafts and GVH prophylaxis with post-transplant cyclosphosphamide or ex vivo T-depletion adapted to the particular susceptibility of patients with Fanconi disease has reduced the incidence of these severe complications
This observational protocol will allow for an independent prospective evaluation of the improvement in survival of patients with Fanconi disease in hematological deadlock due to the absence of an HLA-identical donor and having received a haploidentical transplant
This observational protocol will allow for an independent prospective evaluation of the improvement in survival of patients with Fanconi disease in hematological deadlock due to the absence of an HLA-identical donor and having received a haploidentical transplant
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None