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

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ClinicalTrials.gov Identifier: NCT01340404
Recruitment Status : Completed
First Posted : April 22, 2011
Last Update Posted : December 5, 2016
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

April 21, 2011
April 22, 2011
December 5, 2016
December 2010
April 2013   (Final data collection date for primary outcome measure)
Cerebral vasculopathy [ Time Frame: 1 year ]
velocity in the artery with highest velocity
Cerebral vasculopathy [ Time Frame: 1 year ]
percentage of patients at 1 year with velocities normalization on TCD (< 170 cm/sec)
Complete list of historical versions of study NCT01340404 on ClinicalTrials.gov Archive Site
  • 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)
  • ischemic lesions on MRI [ Time Frame: 1 year ]
    percentage of patients with ischemic lesions on MRI
  • stenoses on MRA [ Time Frame: 1 year ]
    percentage of patients with stenoses on MRA
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
Not Provided
 
Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
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.
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
Interventional
Not Applicable
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Sickle Cell Anemia
  • Cerebrovascular Accident
  • Procedure: Stem cell transplantation
    Stem cell transplantation
  • Procedure: Transfusion program
    Transfusion program
  • Experimental: Stem Cell Transplantation
    Intervention: Procedure: Stem cell transplantation
  • Active Comparator: Transfusion program
    Intervention: Procedure: Transfusion program
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
63
Same as current
October 2015
April 2013   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
up to 15 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
France
 
 
NCT01340404
P071247
Yes
Not Provided
Not Provided
Assistance Publique - Hôpitaux de Paris
Assistance Publique - Hôpitaux de Paris
Not Provided
Principal Investigator: Françoise Bernaudin, MD Assistance Publique - Hôpitaux de Paris
Assistance Publique - Hôpitaux de Paris
November 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP