Sickle-cell Disease (SCD)DREPAtientNCT04413539
Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
An observational study of Sickle-cell Disease (SCD), run by Argo Sante at 1 site in France.
Trial summary
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve patients care according to their specific needs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle-cell Disease (SCD)
- How many people
- 1,088 participants
- Where
- 1 siteFrance
- Study dates
- 2 Jun 2020to 27 Apr 2021
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults or parents of minor children with SCD living in France.
Who may be able to join
- 18 years or older
- Diagnosed with SCD or parent of a minor child diagnosed with SCD
- Able to speak and understand French
- Willing to participate to the study
Who cannot join
- Person having serious difficulties to read or speak French, unable to answer the questions
- Person suffering from other chronic disease which could bias the representation they have about SCD
- Patient cured with a bone marrow transplant
- Patient with mental disorder preventing the patient to understand the study
Trial locations and contacts
1 site in 1 country.
France1 site
Centre de références syndromes drépanocytaires majeurs thalassémie et autres maladies rares du globule rouge et de l'érythropoïése
Créteil
About this study
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve patients care according to their specific needs.
The aim of the study is to accurately describe the impact of SCD on quality of life of patients living in France, or their family (for minor patients). The consequences of the disease on professional life, education and material condition of patients or their parents will be described by the patients themselves.
Read the detailed description
This is a retrospective study. Patients or parents of minor patients living in France and suffering from SCD will be informed about the study and enrolled during their usual follow-up, or by patients' associations. They will be asked to answer an anonymous survey online relating to their whole life-course with SCD and exploring different aspects of the consequences of the disease on their life. It contains 4 different sections:
- socio-demographic questions to establish a respondent profile
- health status of the respondent to establish the severity of the disease
- education, professional life, material and economic situation
- quality of life through MOS SF-36 questionnaire This is a one-shot survey, which will take approximatively 20 to 30 minutes to answer by the respondent.
- Conditions
- Sickle-cell Disease (SCD)
- Keywords
- Sickle-cell disease, quality of life, education, socio-professional integration, france
- Medical subject headings
- Anemia, Sickle Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
Describe the disease impact on quality of life through description of professional life, education and material condition of patients, or their parents (for minor patients), suffering from sickle-cell disease and living in France.
Measure of mutual impact of health status on patients educational and socio-professional life course.
Measured: Once at enrollment
Secondary outcomes (1)
- Study the factors of heterogeneity of patients' profiles - Explore the causal relation between health status and employment conditions, education, material and economic situation - Assess the patients' needs in care and socio-economic support
The perceived impact of disease on education will be confirmed by:
- Type of education adjustment introduced
- The level and the cause of leaving full-time education
- The intensity of school absences
- The feeling of discrimination on school time
The impact of disease on professional life will be measured by:
- The professional status
- The sick leaves
- The feeling of discrimination at work
- The compatibility of work conditions with the disease
The impact of disease on material and economic situation will be assessed by:
- The income level
- The patient cost sharing
- The remaining balance
- The recognition of handicap
- The recognition of disability
The quality of life score of respondents:
- Medical outcome study short form 36 item health survey (MOS SF-36)
Measured: Once at enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2020
- Study start2 Jun 2020
- Primary completion27 Apr 2021
- Study completion27 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for Sickle-cell Disease (SCD)Studies 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 1,088 participants (actual)
Sponsor and investigators
- Lead sponsor
- Argo Sante
- Sponsor type
- University, hospital or other
- Collaborators
- Filière de santé maladies rares MCGRE, Fédération des malades drépanocytaires et thalassémiques SOS Globi, Genetix Biotherapeutics Inc.
- Responsible party
- Sponsor
- Investigators
- Frederic Galacteros, MD,PhD,Prof., Principal investigator, Red Cell genetic diseases unity, Henri Mondor Hospital, Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04413539
- Sponsor's protocol ID
- 2020-A00729-30
- Acronym
- DREPAtient
- Status last verified
- Jul 2021
- First submitted
- 29 May 2020
- Last updated
- 29 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Crosby LE, Joffe NE, Irwin MK, Strong H, Peugh J, Shook L, Kalinyak KA, Mitchell MJ. School Performance and Disease Interference in Adolescents with Sickle Cell Disease. Phys Disabil. 2015;34(1):14-30. doi: 10.14434/pders.v34i1.13918.
PubMed 27547816Dos Santos JP, Gomes Neto M. Sociodemographic aspects and quality of life of patients with sickle cell anemia. Rev Bras Hematol Hemoter. 2013;35(4):242-5. doi: 10.5581/1516-8484.20130093.
PubMed 24106440Gardner K, Douiri A, Drasar E, Allman M, Mwirigi A, Awogbade M, Thein SL. Survival in adults with sickle cell disease in a high-income setting. Blood. 2016 Sep 8;128(10):1436-8. doi: 10.1182/blood-2016-05-716910. Epub 2016 Jul 20. No abstract available.
PubMed 27439910Habibi A, Arlet JB, Stankovic K, Gellen-Dautremer J, Ribeil JA, Bartolucci P, Lionnet F; centre de reference maladies rares << syndromes drepanocytaires majeurs >>. [French guidelines for the management of adult sickle cell disease: 2015 update]. Rev Med Interne. 2015 May 11;36(5 Suppl 1):5S3-84. doi: 10.1016/S0248-8663(15)60002-9. French.
PubMed 26007619Chapelon E, Garabedian M, Brousse V, Souberbielle JC, Bresson JL, de Montalembert M. Osteopenia and vitamin D deficiency in children with sickle cell disease. Eur J Haematol. 2009 Dec 1;83(6):572-8. doi: 10.1111/j.1600-0609.2009.01333.x. Epub 2009 Aug 13.
PubMed 19682065Godeau B, Noel V, Habibi A, Schaeffer A, Bachir D, Galacteros F. [Sickle cell disease in adults: which emergency care by the internists?]. Rev Med Interne. 2001 May;22(5):440-51. doi: 10.1016/s0248-8663(01)00369-1. French.
PubMed 11402515LINDENBAUM J, KLIPSTEIN FA. FOLIC ACID DEFICIENCY IN SICKLE-CELL ANEMIA. N Engl J Med. 1963 Oct 24;269:875-82. doi: 10.1056/NEJM196310242691701. No abstract available.
PubMed 14050985Noll RB, Vannatta K, Koontz K, Kalinyak K, Bukowski WM, Davies WH. Peer relationships and emotional well-being of youngsters with sickle cell disease. Child Dev. 1996 Apr;67(2):423-36. doi: 10.1111/j.1467-8624.1996.tb01743.x.
PubMed 8625722Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.
PubMed 21131035Salih KMA. The impact of sickle cell anemia on the quality of life of sicklers at school age. J Family Med Prim Care. 2019 Feb;8(2):468-471. doi: 10.4103/jfmpc.jfmpc_444_18.
PubMed 30984656Sehlo MG, Kamfar HZ. Depression and quality of life in children with sickle cell disease: the effect of social support. BMC Psychiatry. 2015 Apr 11;15:78. doi: 10.1186/s12888-015-0461-6.
PubMed 25880537
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
- ClinicalTrials.gov ID
- NCT04413539
- Acronym / sponsor ID
- DREPAtient / 2020-A00729-30
- Conditions
- Sickle-cell Disease (SCD)
- Keywords
- Sickle-cell disease, quality of life, education, socio-professional integration, france
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,088 participants (actual)
- Lead sponsor
- Argo SanteUniversity, hospital or other
- Collaborators
- Filière de santé maladies rares MCGRE, Fédération des malades drépanocytaires et thalassémiques SOS Globi, Genetix Biotherapeutics Inc.
- Study start
- 2 Jun 2020 (actual)
- Primary completion
- 27 Apr 2021 (actual)
- Study completion
- 27 Apr 2021 (actual)
- First posted
- 4 Jun 2020 (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.
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve patients care according to their specific needs.
The aim of the study is to accurately describe the impact of SCD on quality of life of patients living in France, or their family (for minor patients). The consequences of the disease on professional life, education and material condition of patients or their parents will be described by the patients themselves.
Detailed description
This is a retrospective study. Patients or parents of minor patients living in France and suffering from SCD will be informed about the study and enrolled during their usual follow-up, or by patients' associations. They will be asked to answer an anonymous survey online relating to their whole life-course with SCD and exploring different aspects of the consequences of the disease on their life. It contains 4 different sections:
- socio-demographic questions to establish a respondent profile
- health status of the respondent to establish the severity of the disease
- education, professional life, material and economic situation
- quality of life through MOS SF-36 questionnaire This is a one-shot survey, which will take approximatively 20 to 30 minutes to answer by the respondent.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults or parents of minor children with SCD living in France.
Key inclusion criteria (4 listed)
- 18 years or older
- Diagnosed with SCD or parent of a minor child diagnosed with SCD
- Able to speak and understand French
- Willing to participate to the study
Key exclusion criteria (4 listed)
- Person having serious difficulties to read or speak French, unable to answer the questions
- Person suffering from other chronic disease which could bias the representation they have about SCD
- Patient cured with a bone marrow transplant
- Patient with mental disorder preventing the patient to understand the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Describe the disease impact on quality of life through description of professional life, education and material condition of patients, or their parents (for minor patients), suffering from sickle-cell disease and living in France.
Time frame: Once at enrollment
Measure of mutual impact of health status on patients educational and socio-professional life course.
1 secondary outcome
- Study the factors of heterogeneity of patients' profiles - Explore the causal relation between health status and employment conditions, education, material and economic situation - Assess the patients' needs in care and socio-economic support
Time frame: Once at enrollment
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 29 Jan 2025.
- Background: PubMed 27547816Crosby LE, Joffe NE, Irwin MK, Strong H, Peugh J, Shook L, Kalinyak KA, Mitchell MJ. School Performance and Disease Interference in Adolescents with Sickle Cell Disease. Phys Disabil. 2015;34(1):14-30. doi: 10.14434/pders.v34i1.13918.
- Background: PubMed 24106440Dos Santos JP, Gomes Neto M. Sociodemographic aspects and quality of life of patients with sickle cell anemia. Rev Bras Hematol Hemoter. 2013;35(4):242-5. doi: 10.5581/1516-8484.20130093.
- Background: PubMed 27439910Gardner K, Douiri A, Drasar E, Allman M, Mwirigi A, Awogbade M, Thein SL. Survival in adults with sickle cell disease in a high-income setting. Blood. 2016 Sep 8;128(10):1436-8. doi: 10.1182/blood-2016-05-716910. Epub 2016 Jul 20. No abstract available.
- Background: PubMed 26007619Habibi A, Arlet JB, Stankovic K, Gellen-Dautremer J, Ribeil JA, Bartolucci P, Lionnet F; centre de reference maladies rares << syndromes drepanocytaires majeurs >>. [French guidelines for the management of adult sickle cell disease: 2015 update]. Rev Med Interne. 2015 May 11;36(5 Suppl 1):5S3-84. doi: 10.1016/S0248-8663(15)60002-9. French.
- Background: PubMed 19682065Chapelon E, Garabedian M, Brousse V, Souberbielle JC, Bresson JL, de Montalembert M. Osteopenia and vitamin D deficiency in children with sickle cell disease. Eur J Haematol. 2009 Dec 1;83(6):572-8. doi: 10.1111/j.1600-0609.2009.01333.x. Epub 2009 Aug 13.
- Background: PubMed 11402515Godeau B, Noel V, Habibi A, Schaeffer A, Bachir D, Galacteros F. [Sickle cell disease in adults: which emergency care by the internists?]. Rev Med Interne. 2001 May;22(5):440-51. doi: 10.1016/s0248-8663(01)00369-1. French.
- Background: PubMed 14050985LINDENBAUM J, KLIPSTEIN FA. FOLIC ACID DEFICIENCY IN SICKLE-CELL ANEMIA. N Engl J Med. 1963 Oct 24;269:875-82. doi: 10.1056/NEJM196310242691701. No abstract available.
- Background: PubMed 8625722Noll RB, Vannatta K, Koontz K, Kalinyak K, Bukowski WM, Davies WH. Peer relationships and emotional well-being of youngsters with sickle cell disease. Child Dev. 1996 Apr;67(2):423-36. doi: 10.1111/j.1467-8624.1996.tb01743.x.
- Background: PubMed 21131035Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.
- Background: PubMed 30984656Salih KMA. The impact of sickle cell anemia on the quality of life of sicklers at school age. J Family Med Prim Care. 2019 Feb;8(2):468-471. doi: 10.4103/jfmpc.jfmpc_444_18.
- Background: PubMed 25880537Sehlo MG, Kamfar HZ. Depression and quality of life in children with sickle cell disease: the effect of social support. BMC Psychiatry. 2015 Apr 11;15:78. doi: 10.1186/s12888-015-0461-6.
- Related InfoLink provided by the sponsor.
- 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.
Frederic Galacteros, MD,PhD,Prof. principal investigator
Red Cell genetic diseases unity, Henri Mondor Hospital, Paris
1 site 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 (SCD), run by Argo Sante 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 4 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 29 Jan 2025. 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?
Describe the disease impact on quality of life through description of professional life, education and material condition of patients, or their parents (for minor patients), suffering from sickle-cell disease and living in France. (time frame: Once at enrollment). 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 NCT04413539, which also lists 11 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 29 Jan 2025.