NCT01340404

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
63

participants targeted

Target at P50-P75 for not_applicable

Timeline
Completed

Started Dec 2010

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2010

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

April 21, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 22, 2011

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2013

Completed
2.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2015

Completed
Last Updated

December 5, 2016

Status Verified

November 1, 2016

Enrollment Period

2.3 years

First QC Date

April 21, 2011

Last Update Submit

December 2, 2016

Conditions

Sickle Cell AnemiaCerebrovascular Accident

Outcome Measures

Primary Outcomes (1)

  • Cerebral vasculopathy

    velocity in the artery with highest velocity

    1 year

Secondary Outcomes (3)

  • ischemic lesions on magnetic resonance imaging (MRI)

    1 year

  • stenoses on magnetic resonance imaging (MRI)

    1 year

  • normalisation of arterial velocities

    12 months

Other Outcomes (1)

  • cognitive dysfunction

    12 months

Study Arms (2)

Stem Cell Transplantation

EXPERIMENTAL
Procedure: Stem cell transplantation

Transfusion program

ACTIVE COMPARATOR
Procedure: Transfusion program

Interventions

Stem cell transplantationPROCEDURE

Stem cell transplantation

Stem Cell Transplantation
Transfusion programPROCEDURE

Transfusion program

Transfusion program

Eligibility Criteria

AgeUp to 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CHIC

Créteil, France

Location

Related Publications (2)

  • 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.

    PMID: 30667500DERIVED

  • 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.

    PMID: 28821470DERIVED

MeSH Terms

Conditions

Anemia, Sickle CellStroke

Interventions

Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCerebrovascular DisordersBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesVascular DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

Cell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Officials

  • Françoise Bernaudin, MD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 21, 2011

First Posted

April 22, 2011

Study Start

December 1, 2010

Primary Completion

April 1, 2013

Study Completion

October 1, 2015

Last Updated

December 5, 2016

Record last verified: 2016-11

Locations

FR(1)