Clinical trial NCT01340404: Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
Official title: A Multicenter Study Comparing the Results of Allogeneic Stem Cell Genoidentical in Children With Sickle Cell Anemia and Cerebral Vascular Disease Detected by Transcranial Doppler
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sickle Cell Anemia
Cerebrovascular Accident
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2011-04-22
Enrollment: 63
Sex: ALL
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients.

Detailed description: The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD)are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients. Sixty-three patients will be enrolled, namely 21 sickle-cell patients in the "SCT" group and 42 in the "Transfusion Program (TP)" group

Eligibility criteria: Inclusion Criteria:

* Sickle cell anemia patients (SS/Sb0)
* \< 15 years old
* History of abnormal transcranial doppler (TCD) (≥ 200 cm/sec)
* Siblings from the same parental couple
* Parents amenable to Human Leucocyte Antigen (HLA) typing, SCT if an HLA-identical sibling is available or to long-term transfusion program

Exclusion Criteria:

* Sickle cell patients older than 15 years
* no history of abnormal transcranial doppler (TCD) (≥ 200 cm/sec)
* no sibling from the same parental couple
* parents averse to HLA typing, SCT if an HLA-identical sibling is available or to long-term transfusion program

Primary outcomes:
measure: Cerebral vasculopathy
timeFrame: 1 year
Secondary outcomes:
measure: ischemic lesions on magnetic resonance imaging (MRI)
timeFrame: 1 year
measure: stenoses on magnetic resonance imaging (MRI)
timeFrame: 1 year
measure: normalisation of arterial velocities
timeFrame: 12 months
Other outcomes:
measure: cognitive dysfunction
timeFrame: 12 months
Locations:
city: Créteil
country: France
facility: CHIC
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Françoise Bernaudin, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Bernaudin F, Verlhac S, Peffault de Latour R, Dalle JH, Brousse V, Petras E, Thuret I, Paillard C, Neven B, Galambrun C, Divialle-Doumdo L, Pondarre C, Guitton C, Missud F, Runel C, Jubert C, Elana G, Ducros-Miralles E, Drain E, Taieb O, Arnaud C, Kamdem A, Malric A, Elmaleh-Berges M, Vasile M, Leveille E, Socie G, Chevret S; DREPAGREFFE Trial Investigators. Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia. JAMA. 2019 Jan 22;321(3):266-276. doi: 10.1001/jama.2018.20059.
citation: Chevret S, Verlhac S, Ducros-Miralles E, Dalle JH, de Latour RP, de Montalembert M, Benkerrou M, Pondarre C, Thuret I, Guitton C, Lesprit E, Etienne-Julan M, Elana G, Vannier JP, Lutz P, Neven B, Galambrun C, Paillard C, Runel C, Jubert C, Arnaud C, Kamdem A, Brousse V, Missud F, Petras M, Doumdo-Divialle L, Berger C, Freard F, Taieb O, Drain E, Elmaleh M, Vasile M, Khelif Y, Bernaudin M, Chadebech P, Pirenne F, Socie G, Bernaudin F. Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404). Contemp Clin Trials. 2017 Nov;62:91-104. doi: 10.1016/j.cct.2017.08.008. Epub 2017 Aug 15.