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

Cerebrovascular Accident Clinical Trial

— DREPAGREFFE  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01340404
• Other study ID #: P071247
• Status: Completed
• Phase: N/A
• First received: April 21, 2011
• Last updated: December 2, 2016
• Start date: December 2010
• Est. completion date: October 2015

Study information

• Verified date: November 2016
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. completion date: October 2015
• Est. primary completion date: April 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 15 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Sickle Cell
• Cerebrovascular Accident
• Sickle Cell Anemia
• Stroke

Intervention

Procedure:
• Stem cell transplantation
Stem cell transplantation
• Transfusion program
Transfusion program

Locations

Country	Name	City	State
France	CHIC	Créteil

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	cognitive dysfunction	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	12 months	No
Primary	Cerebral vasculopathy	velocity in the artery with highest velocity	1 year	No
Secondary	ischemic lesions on magnetic resonance imaging (MRI)	percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)	1 year	No
Secondary	stenoses on magnetic resonance imaging (MRI)	percentage of patients with stenoses on magnetic resonance imaging (MRI)	1 year	No
Secondary	normalisation of arterial velocities	percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec)	12 months	No