Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy (DREPAGREFFE)

December 2, 2016 updated by: Assistance Publique - Hôpitaux de Paris

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Actual)

63

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Créteil, France
        • CHIC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Stem Cell Transplantation
Procedure: Stem cell transplantation
Stem cell transplantation
Active Comparator: Transfusion program
Procedure: Transfusion program
Transfusion program

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cerebral vasculopathy
Time Frame: 1 year
velocity in the artery with highest velocity
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ischemic lesions on magnetic resonance imaging (MRI)
Time Frame: 1 year
percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)
1 year
stenoses on magnetic resonance imaging (MRI)
Time Frame: 1 year
percentage of patients with stenoses on magnetic resonance imaging (MRI)
1 year
normalisation of arterial velocities
Time Frame: 12 months
percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec)
12 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
cognitive dysfunction
Time Frame: 12 months
the scale of cognitive dyfunction depends on the child's age : definition of dysfunction uses the WISC-4, WPPSI-4 or WAIS-3 according to age
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Investigators

  • Principal Investigator: Françoise Bernaudin, MD, Assistance Publique - Hôpitaux de Paris

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2010

Primary Completion (Actual)

April 1, 2013

Study Completion (Actual)

October 1, 2015

Study Registration Dates

First Submitted

April 21, 2011

First Submitted That Met QC Criteria

April 21, 2011

First Posted (Estimate)

April 22, 2011

Study Record Updates

Last Update Posted (Estimate)

December 5, 2016

Last Update Submitted That Met QC Criteria

December 2, 2016

Last Verified

November 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P071247

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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