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Completed

NCT Number: NCT01340404

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

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.

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Key information

Conditions

Sickle Cell Anemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Brain Diseases Cardiovascular Diseases Central Nervous System Diseases Cerebrovascular Accident Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Nervous System Diseases Stroke Vascular Diseases

Age range

Up to 15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

CHIC

Créteil, France

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

stem cell transplantation

Procedure

Stem cell transplantation

Transfusion program

Procedure

Transfusion program

Primary outcomes

  1. Cerebral vasculopathy

    Time frame: 1 year

    velocity in the artery with highest velocity

Secondary outcomes

  1. ischemic lesions on magnetic resonance imaging (MRI)

    Time frame: 1 year

    percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)

  2. stenoses on magnetic resonance imaging (MRI)

    Time frame: 1 year

    percentage of patients with stenoses on magnetic resonance imaging (MRI)

  3. 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 outcomes

  1. 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

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Official study 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

Acronym: DREPAGREFFE

Important dates

Study start
2010
Primary completion
2013
Study completion
2015
First posted
Apr 22, 2011
Registry last updated
Dec 5, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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