Sickle Cell AnemiaDREPAGREFFENCT01340404
Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
A study testing Stem cell transplantation and Transfusion program in people with Sickle Cell Anemia and Cerebrovascular Accident, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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… Read more about this study
- Who is it for?
- Ages Up to 15 years
- Conditions the study is for
- Sickle Cell Anemia, Cerebrovascular Accident
- What is being tested?
- Stem cell transplantation and Transfusion program
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 1 siteFrance
- Study dates
- Dec 2010to Oct 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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
- Conditions
- Sickle Cell Anemia, Cerebrovascular Accident
- Being tested
- Stem cell transplantation, Transfusion program
- Medical subject headings
- Anemia, Sickle Cell, Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem cell transplantationProcedure or surgery
Stem cell transplantation
Transfusion programProcedure or surgery
Transfusion program
| Study group | Role | What they receive |
|---|---|---|
| Stem Cell Transplantation | Experimental | Not described Receives: Procedure: Stem cell transplantation |
| Transfusion program | Active comparator | Not described Receives: Procedure: Transfusion program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cerebral vasculopathy
velocity in the artery with highest velocity
Measured: 1 year
Secondary outcomes (3)
ischemic lesions on magnetic resonance imaging (MRI)
percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)
Measured: 1 year
stenoses on magnetic resonance imaging (MRI)
percentage of patients with stenoses on magnetic resonance imaging (MRI)
Measured: 1 year
normalisation of arterial velocities
percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2011 (estimated)
- Study startDec 2010
- Primary completionApr 2013
- Study completionOct 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Françoise Bernaudin, MD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01340404
- Sponsor's protocol ID
- P071247
- Acronym
- DREPAGREFFE
- Status last verified
- Nov 2016
- First submitted
- 21 Apr 2011
- Last updated
- 5 Dec 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Bernaudin F, Arnaud C, Kamdem A, Youn J, Vasile M, Hau I, Madhi F, Malterre A, Delestrain C, Epaud R, Jung C, Verlhac S. Early detection and management of extracranial arteriopathy reduces the incidence of silent cerebral infarcts in sickle cell anemia: a long-term prospective cohort study. Haematologica. 2026 Jan 1;111(1):314-328. doi: 10.3324/haematol.2025.287720. Epub 2025 Jun 26.
PubMed 40568713Bernaudin F, Verlhac S, Peffault de Latour R, Dalle JH, Brousse V, Petras E, Thuret I, Paillard C, Neven B, Galambrun C, Divialle-Doumdo L, Pondarre C, Guitton C, Missud F, Runel C, Jubert C, Elana G, Ducros-Miralles E, Drain E, Taieb O, Arnaud C, Kamdem A, Malric A, Elmaleh-Berges M, Vasile M, Leveille E, Socie G, Chevret S; DREPAGREFFE Trial Investigators. Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia. JAMA. 2019 Jan 22;321(3):266-276. doi: 10.1001/jama.2018.20059.
PubMed 30667500Chevret S, Verlhac S, Ducros-Miralles E, Dalle JH, de Latour RP, de Montalembert M, Benkerrou M, Pondarre C, Thuret I, Guitton C, Lesprit E, Etienne-Julan M, Elana G, Vannier JP, Lutz P, Neven B, Galambrun C, Paillard C, Runel C, Jubert C, Arnaud C, Kamdem A, Brousse V, Missud F, Petras M, Doumdo-Divialle L, Berger C, Freard F, Taieb O, Drain E, Elmaleh M, Vasile M, Khelif Y, Bernaudin M, Chadebech P, Pirenne F, Socie G, Bernaudin F. Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404). Contemp Clin Trials. 2017 Nov;62:91-104. doi: 10.1016/j.cct.2017.08.008. Epub 2017 Aug 15.
PubMed 28821470
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01340404
- Acronym / sponsor ID
- DREPAGREFFE / P071247
- Conditions
- Sickle Cell Anemia, Cerebrovascular Accident
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Dec 2010
- Primary completion
- Apr 2013 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 22 Apr 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stem Cell Transplantation | Experimental | Procedure: Stem cell transplantation |
| Transfusion program | Active comparator | Procedure: Transfusion program |
Who may qualify
- Ages
- Up to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Stem cell transplantationStem cell transplantation | Procedure or surgery | Stem Cell Transplantation |
| Transfusion programTransfusion program | Procedure or surgery | Transfusion program |
Primary outcome
Cerebral vasculopathy
Time frame: 1 year
velocity in the artery with highest velocity
3 secondary outcomes
ischemic lesions on magnetic resonance imaging (MRI)
Time frame: 1 year
stenoses on magnetic resonance imaging (MRI)
Time frame: 1 year
normalisation of arterial velocities
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2016.
- Derived from this study: PubMed 40568713Bernaudin F, Arnaud C, Kamdem A, Youn J, Vasile M, Hau I, Madhi F, Malterre A, Delestrain C, Epaud R, Jung C, Verlhac S. Early detection and management of extracranial arteriopathy reduces the incidence of silent cerebral infarcts in sickle cell anemia: a long-term prospective cohort study. Haematologica. 2026 Jan 1;111(1):314-328. doi: 10.3324/haematol.2025.287720. Epub 2025 Jun 26.
- Derived from this study: PubMed 30667500Bernaudin F, Verlhac S, Peffault de Latour R, Dalle JH, Brousse V, Petras E, Thuret I, Paillard C, Neven B, Galambrun C, Divialle-Doumdo L, Pondarre C, Guitton C, Missud F, Runel C, Jubert C, Elana G, Ducros-Miralles E, Drain E, Taieb O, Arnaud C, Kamdem A, Malric A, Elmaleh-Berges M, Vasile M, Leveille E, Socie G, Chevret S; DREPAGREFFE Trial Investigators. Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia. JAMA. 2019 Jan 22;321(3):266-276. doi: 10.1001/jama.2018.20059.
- Derived from this study: PubMed 28821470Chevret S, Verlhac S, Ducros-Miralles E, Dalle JH, de Latour RP, de Montalembert M, Benkerrou M, Pondarre C, Thuret I, Guitton C, Lesprit E, Etienne-Julan M, Elana G, Vannier JP, Lutz P, Neven B, Galambrun C, Paillard C, Runel C, Jubert C, Arnaud C, Kamdem A, Brousse V, Missud F, Petras M, Doumdo-Divialle L, Berger C, Freard F, Taieb O, Drain E, Elmaleh M, Vasile M, Khelif Y, Bernaudin M, Chadebech P, Pirenne F, Socie G, Bernaudin F. Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404). Contemp Clin Trials. 2017 Nov;62:91-104. doi: 10.1016/j.cct.2017.08.008. Epub 2017 Aug 15.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Françoise Bernaudin, MD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Stem cell transplantation and Transfusion program in people with Sickle Cell Anemia and Cerebrovascular Accident, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cerebral vasculopathy (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01340404, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2016.