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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01340404
First received: April 21, 2011
Last updated: April 22, 2014
Last verified: April 2014
  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
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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 ] [ Designated as safety issue: No ]
    percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec)


Secondary Outcome Measures:
  • ischemic lesions on magnetic resonance imaging (MRI) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)

  • stenoses on magnetic resonance imaging (MRI) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    percentage of patients with stenoses on magnetic resonance imaging (MRI)


Enrollment: 63
Study Start Date: December 2010
Estimated Study Completion Date: October 2014
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
Other Name: Stem cell transplantation
Active Comparator: Transfusion program Procedure: Transfusion program
Transfusion program
Other Name: 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

Ages Eligible for Study:   up to 15 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01340404     History of Changes
Other Study ID Numbers: P071247
Study First Received: April 21, 2011
Last Updated: April 22, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

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

ClinicalTrials.gov processed this record on October 23, 2014