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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

Tracking Information
First Submitted Date  ICMJE April 21, 2011
First Posted Date  ICMJE April 22, 2011
Last Update Posted Date December 5, 2016
Study Start Date  ICMJE December 2010
Actual Primary Completion Date April 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 2, 2016)
Cerebral vasculopathy [ Time Frame: 1 year ]
velocity in the artery with highest velocity
Original Primary Outcome Measures  ICMJE
 (submitted: April 21, 2011)
Cerebral vasculopathy [ Time Frame: 1 year ]
percentage of patients at 1 year with velocities normalization on TCD (< 170 cm/sec)
Change History Complete list of historical versions of study NCT01340404 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 2, 2016)
  • 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)
Original Secondary Outcome Measures  ICMJE
 (submitted: April 21, 2011)
  • 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
Current Other Pre-specified Outcome Measures
 (submitted: December 2, 2016)
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
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
Official Title  ICMJE 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
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Anemia
  • Cerebrovascular Accident
Intervention  ICMJE
  • Procedure: Stem cell transplantation
    Stem cell transplantation
  • Procedure: Transfusion program
    Transfusion program
Study Arms  ICMJE
  • Experimental: Stem Cell Transplantation
    Intervention: Procedure: Stem cell transplantation
  • Active Comparator: Transfusion program
    Intervention: Procedure: Transfusion program
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 21, 2011)
63
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 2015
Actual Primary Completion Date April 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 15 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01340404
Other Study ID Numbers  ICMJE P071247
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Assistance Publique - Hôpitaux de Paris
Study Sponsor  ICMJE Assistance Publique - Hôpitaux de Paris
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Françoise Bernaudin, MD Assistance Publique - Hôpitaux de Paris
PRS Account Assistance Publique - Hôpitaux de Paris
Verification Date November 2016

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