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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01340404
First Posted: April 22, 2011
Last Update Posted: December 5, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
  Purpose
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.

Condition Intervention
Sickle Cell Anemia Cerebrovascular Accident Procedure: Stem cell transplantation Procedure: Transfusion program

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • Cerebral vasculopathy [ Time Frame: 1 year ]
    velocity in the artery with highest velocity


Secondary Outcome Measures:
  • ischemic lesions on magnetic resonance imaging (MRI) [ Time Frame: 1 year ]
    percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)

  • stenoses on magnetic resonance imaging (MRI) [ Time Frame: 1 year ]
    percentage of patients with stenoses on magnetic resonance imaging (MRI)

  • normalisation of arterial velocities [ Time Frame: 12 months ]
    percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec)


Other Outcome Measures:
  • 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


Enrollment: 63
Study Start Date: December 2010
Study Completion Date: October 2015
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Stem Cell Transplantation Procedure: Stem cell transplantation
Stem cell transplantation
Active Comparator: Transfusion program Procedure: Transfusion program
Transfusion program

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

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Ages Eligible for Study:   up to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01340404


Locations
France
CHIC
Créteil, France
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Françoise Bernaudin, MD Assistance Publique - Hôpitaux de Paris
  More Information

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01340404     History of Changes
Other Study ID Numbers: P071247
First Submitted: April 21, 2011
First Posted: April 22, 2011
Last Update Posted: December 5, 2016
Last Verified: November 2016

Additional relevant MeSH terms:
Anemia
Stroke
Anemia, Sickle Cell
Hematologic Diseases
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn