Sickle Cell DiseaseDREPAGREFFE2NCT05053932
Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia
An observational study of Sickle Cell Disease and Cerebral Ischemia, run by Centre Hospitalier Intercommunal Creteil at 14 sites in France.
Trial summary
The purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol and whose 1- and 3-year results were published in JAMA (Journal of the American Medical… Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Sickle Cell Disease, Cerebral Ischemia, Stenosis
- What is being tested?
- blood collection
- How many people
- 67 participants
- Where
- 14 sitesFrance
- Study dates
- 7 Oct 2022to 31 Jan 2025
- Phase
- Observational
- 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 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Population concerned 67 patients included in 2011-2013 in the DREPAGREFFE protocol [NCT 01340404]
Who may be able to join
- Patient of legal age or minor who participated in the DREPAGREFFE research protocol [NCT 01340404] between December 2010 and June 2013,
- Having read and understood the information letter
Who cannot join
- Refusal to participate
- Patient deceased
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
France14 sites
Groupe hospitalier Pellegrin
Bordeaux, 33000
Centre Hospitalier Intercommunal de Créteil
Créteil, 94000
Hôpital Henri Monr - APHP
Créteil, 94000
CHU de la Guadeloupe
La Guadeloupe, 97110
Hôpital Bicêtre AP-HP
Le Kremlin-Bicêtre, 94270
IHOPe
Lyon, 69008
CHU de Lyon
Lyon, 69737
Hôpital de la Timone
Marseille, 13005
CHU de Montpellier
Montpellier, 34295
Hôpital Robert Debré AP-HP
Paris, IDF, 75019
Hôpital Necker - AP-HP
Paris, 75015
CH de Pau
Pau, 64000
CHU de Rennes
Rennes, 35033
Hôpital Hautepierre
Strasbourg, 67200
About this study
Official title: Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Children With Sickle Cell Anemia ( DREPAGREFFE-2)
The purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol and whose 1- and 3-year results were published in JAMA (Journal of the American Medical Association) in 2019 and in BHJ in 2020.
Read the detailed description
The present observational study has the objective to reevaluate at distance the cohort of 67 children with sickle cell anemia enrolled in the "Drepagreffe"trial because of cerebral vasculopathy detected by transcranial Doppler. Results at 1 and 3 years were reported in JAMA in 2019 in BHJ in 2020. This trial was the first worldwide prospective study comparing transplantation to standard care in sickle cell disease. Velocities were highly significantly more reduced with a higher proportion of patients with normalized velocities and better quality of life after transplantation than on standard care. Despite a trend to a better ischemic lesions outcome at 3 years, the difference was not significant and cognitive performances were not different between both groups. The biologic study only assessed at enrollment and 1-year showed lower levels of Ang-2 and HGF (hepatocyte growth factor) after transplant and a significant and independent association between Doppler normalization probability with low Ang-2 and BDNF (brain-derived neurotrophic factor) levels.The aim of the present study is to reassess at 9-10 years this cohort with grants allowing to reevaluate cognitive functioning and hypoxia/angiogenic factors not realized in the systematic cohort follow-up
- Conditions
- Sickle Cell Disease, Cerebral Ischemia, Stenosis
- Being tested
- blood collection
- Keywords
- transplantation
- Medical subject headings
- Anemia, Sickle Cell, Brain Ischemia, Constriction, Pathologic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood collectionOther
blood collection
| Study group | Role | What they receive |
|---|---|---|
| Patients included in Drepagreffe 1 study (NCT01340404) | biological collection Receives: Other: blood collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long term evolution (at 9-10 years) of Ischemic lesion on brain magnetic resonance imaging 10-year measurement of ischemic lesion on magnetic resonance imaging
The MRI-scores, ranging from 0 (best outcome) to 10 (worst outcome), are obtained by adding up the ischemic lesion scores from the left and right sides, i.e., 3 for territorial or 2 for border zone (cortical and subcortical), 1 for white matter and 1 for basal ganglia infarcts and 0 if absent on each side.
Measured: within 6 months of inclusion
Long term evolution (at 9-10 years) of arterial stenosis on cerebral and cervical magnetic resonance angiography
The MRA stenosis-scores, ranging from 0 (best outcome) to 32 (worst outcome) are defined as the weighted sums over the 8 assessed cerebral arteries, as 0 if no stenosis, 1 if mild stenosis (25-49%), 2 if moderate stenosis (50-74%), 3 if severe stenosis (75-99%), and 4 if occlusion.
Measured: within 6 months of inclusion
Secondary outcomes (3)
Long term evolution (at 9-10 years) of cognitive performance
Full Scale Intelligence Quotient (40= worst outcome, 160= best outcome) is measured by Wechsler Intelligence Scale for Children -Fourth Edition (WISC-4) for children 7-16 years of age and by WAIS-3 (Weschler Adult Intelligence Scale-3) for patients older than 16 years
Measured: within 6 months of inclusion
Long term evolution (at 9-10 years) of quality of life
Quality of life assessment is collected using the French version of the Pediatric Quality of Life Inventory Generic Core Scale (PedsQLTM 4.0 generic core scales) (physical, emotional, social, school items) (0= worst outcome, 100= best outcome) via self-report and parent proxy-report
Measured: within 6 months of inclusion
Evolution at 9-10 years of factors of hypoxia and oxidative stress
Assessment on plasma Phosphatidyl-serine expression VEGF, Angiopoietin-1 (Ang-1) and Angiopoietin-2 (Ang-2),EPO, HIF-1, BDNF, PDGF-AA
Measured: within 6 months of inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2021
- Study start7 Oct 2022
- Primary completion5 Jan 2025
- Study completion31 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Intercommunal Creteil
- Sponsor type
- University, hospital or other
- Collaborators
- Centre National de la Recherche Scientifique, France, Etablissement Français du Sang
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05053932
- Sponsor's protocol ID
- DREPAGREFFE2
- Acronym
- DREPAGREFFE2
- Status last verified
- Jun 2026
- First submitted
- 12 May 2021
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Children With Sickle Cell Anemia ( DREPAGREFFE-2)
- ClinicalTrials.gov ID
- NCT05053932
- Acronym / sponsor ID
- DREPAGREFFE2 / DREPAGREFFE2
- Conditions
- Sickle Cell Disease, Cerebral Ischemia, Stenosis
- Keywords
- transplantation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Centre Hospitalier Intercommunal CreteilUniversity, hospital or other
- Collaborators
- Centre National de la Recherche Scientifique, France, Etablissement Français du Sang
- Study start
- 7 Oct 2022 (actual)
- Primary completion
- 5 Jan 2025 (actual)
- Study completion
- 31 Jan 2025 (actual)
- First posted
- 23 Sep 2021 (actual)
- 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 purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol and whose 1- and 3-year results were published in JAMA (Journal of the American Medical Association) in 2019 and in BHJ in 2020.
Detailed description
The present observational study has the objective to reevaluate at distance the cohort of 67 children with sickle cell anemia enrolled in the "Drepagreffe"trial because of cerebral vasculopathy detected by transcranial Doppler. Results at 1 and 3 years were reported in JAMA in 2019 in BHJ in 2020. This trial was the first worldwide prospective study comparing transplantation to standard care in sickle cell disease. Velocities were highly significantly more reduced with a higher proportion of patients with normalized velocities and better quality of life after transplantation than on standard care. Despite a trend to a better ischemic lesions outcome at 3 years, the difference was not significant and cognitive performances were not different between both groups. The biologic study only assessed at enrollment and 1-year showed lower levels of Ang-2 and HGF (hepatocyte growth factor) after transplant and a significant and independent association between Doppler normalization probability with low Ang-2 and BDNF (brain-derived neurotrophic factor) levels.The aim of the present study is to reassess at 9-10 years this cohort with grants allowing to reevaluate cognitive functioning and hypoxia/angiogenic factors not realized in the systematic cohort follow-up
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients included in Drepagreffe 1 study (NCT01340404) | – | Other: blood collectionbiological collection |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Population concerned 67 patients included in 2011-2013 in the DREPAGREFFE protocol [NCT 01340404]
Key inclusion criteria (2 listed)
- Patient of legal age or minor who participated in the DREPAGREFFE research protocol [NCT 01340404] between December 2010 and June 2013,
- Having read and understood the information letter
Key exclusion criteria (2 listed)
- Refusal to participate
- Patient deceased
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 |
|---|---|---|
| blood collectionblood collection | Other | Patients included in Drepagreffe 1 study (NCT01340404) |
Primary outcome
Long term evolution (at 9-10 years) of Ischemic lesion on brain magnetic resonance imaging 10-year measurement of ischemic lesion on magnetic resonance imaging
Time frame: within 6 months of inclusion
The MRI-scores, ranging from 0 (best outcome) to 10 (worst outcome), are obtained by adding up the ischemic lesion scores from the left and right sides, i.e., 3 for territorial or 2 for border zone (cortical and subcortical), 1 for white matter and 1 for basal ganglia infarcts and 0 if absent on each side.
Long term evolution (at 9-10 years) of arterial stenosis on cerebral and cervical magnetic resonance angiography
Time frame: within 6 months of inclusion
The MRA stenosis-scores, ranging from 0 (best outcome) to 32 (worst outcome) are defined as the weighted sums over the 8 assessed cerebral arteries, as 0 if no stenosis, 1 if mild stenosis (25-49%), 2 if moderate stenosis (50-74%), 3 if severe stenosis (75-99%), and 4 if occlusion.
3 secondary outcomes
Long term evolution (at 9-10 years) of cognitive performance
Time frame: within 6 months of inclusion
Long term evolution (at 9-10 years) of quality of life
Time frame: within 6 months of inclusion
Evolution at 9-10 years of factors of hypoxia and oxidative stress
Time frame: within 6 months of inclusion
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 15 Jun 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Cerebral Ischemia, run by Centre Hospitalier Intercommunal Creteil at 14 sites 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 2 inclusion and 2 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 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites 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?
Long term evolution (at 9-10 years) of Ischemic lesion on brain magnetic resonance imaging 10-year measurement of ischemic lesion on magnetic resonance imaging (time frame: within 6 months of inclusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Jun 2026.