Sickle Cell DiseaseDREPADOMNCT05649280
Safety Evaluation of DREPADOM - Home Care Services and Hospitalizations for Sickle Cell Disease Patients
An observational study of Sickle Cell Disease, run by Soutien aux Actions contre les Maladies du Globule Rouge at 1 site in France.
Trial summary
The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2) Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 100 participants
- Where
- 1 siteFrance
- Study dates
- 1 Feb 2021to 1 Jun 2022
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Sickle Cell Disease (SCD) is the most widespread genetic disease in the world. With an incidence rate of approximately 1/3000 newborns each year in France, International Standards consider SCD a rare disease. However, in the Ile-de-France region, that incidence drops to 1/435 newborns, rendering SCD a major territorial issue. It is estimated that more than 10 000 SCD patients live in this region, which represents approximately 30% of the French SCD population and 10% of the European SCD population. Vaso-occlusive crisis (VOC) or acute bone pain vaso-occlusion is a characteristic manifestation of SCD and represents the first cause of patients 'hospitalizations. Moreover, 16% of VOCs can develop into a potentially severe complication: the acute chest syndrome (ACS).
Who may be able to join
- SS or Sβ0
- Adult patients with a low PRESEV score
Who cannot join
- SC/Sβ+ patients
- SCD adult patients with an intermediate or high PRESEV score
- Pregnant or breastfeeding women
- Homeless patient
- Patients without Social Security
- Patient deprived of liberty by judicial or administrative decision or patient under guardianship
Trial locations and contacts
1 site in 1 country.
About this study
The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2)
Read the detailed description
This prospective, observational study will take place, as follows:
Calculation of the PRESEV score in the ER will determine or not eligibility of the patient in the DREPADOM structure. With a low score, a patient is eligible for home monitoring and after duly provided information, can sign a patient consent for the use of personal medical data.
The coordinating nurse from DREPADOM can then set up a whole network, with the delivery of oxygen supply at the patient's house, dispatch of a medical prescription to a neighboring pharmacy and daily visits from homecare service providers. Both opioids and parenteral hydration and treatments can be prescribed and daily visits, up to three, are programmed. As such, the patient receives a minimum of 1 visit per day from a nurse for monitoring and is provided with a medical oxygen concentrator for home use (3L/min continuously or for at least 15h/day) and parenteral hydration. In addition, patients are called daily by general practitioners or SCD experts, for teleconsultations.
The DREPADOM coordinating nurse is in constant interaction with nurses/providers of homecare services/medical doctors and status and evolution of the patient's global condition is reported both through the constant contact with the coordinating nurse from DREPADOM and in a dedicated software (Link4Life), with monitoring of blood pressure, temperature, respiratory rate, pain, etc… Link4Life is a software tool developed especially for home monitoring. It is adaptable to specific disorders, fully complies with the French Law for Health Data management and allows data extraction. Link4Life has been adapted to SCD in full collaboration with SCD medical experts of the Henri Mondor hospital. It is to be noted that all medical doctors (general practitioners or SCD experts), as well as coordinating and visiting nurses have all access to this application. As such, Link4Life will gather and capture all patient data.
When reporting patients' clinical parameters daily in the dedicated app (controlled by the DREPADOM team of experts), alerts are generated in the software when these are above or below determined thresholds. The medical team of experts from DREPADOM then takes over for daily teleconsultations and medical decisions for pursuing home monitoring or hospital admittance.
Of important note, a patient can be included twice in the DREPADOM study, for two distinct VOCs. Indeed, the PRESEV1 study highlighted that each crisis is unique and should be treated accordingly.
Data capture will be done through the Link4Life software, as well as extraction for data analysis.
- Open-label
- Blind
- Number of treatment arms:
Single arm
Multi arms specify the number of arms:
- Not Controlled
- Controlled, specify the comparator:
Versus placebo Versus reference product Versus other (specify) -Randomized: Parallel group Cross over Other (specify)
-Intervention (gas and/or drug, and/or device, and/or service /study arm):
- Conditions
- Sickle Cell Disease
- Medical subject headings
- Anemia, Sickle Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of acute chest pain
The primary endpoint is the incidence of Acute chest pain complications developed by patients in the DREPADOM system ( Home Care Services and Hospitalizations for Sickle Cell Disease Patients) and comparison with the historical cohorts of( PRESEV1 and PRESEV2 studies).
Measured: 30 months
Secondary outcomes (1)
Incidence of clinical complications associated to Sickle cell disease
- Transfusions and comparison with historical cohorts of PRESEV1 and PRESEV2 studies
- Patient admission in intensive care unit and comparison with historical cohorts of PRESEV1 and PRESEV2 studies
- Mortality and comparison with historical cohorts of PRESEV1 and PRESEV2 studies
Measured: 30 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2022
- Study start1 Feb 2021
- Primary completion1 Feb 2022
- Study completion1 Jun 2022
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
- Retrospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Soutien aux Actions contre les Maladies du Globule Rouge
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Pablo BARTOLUCCI, PROFESSOR, Principal investigator, Soutien aux Actions contre les Maladies du Globule Rouge
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05649280
- Sponsor's protocol ID
- AIR LIQUIDE HEALTH CARE
- Acronym
- DREPADOM
- Status last verified
- Dec 2022
- First submitted
- 1 Jun 2022
- Last updated
- 14 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety Evaluation of DREPADOM - Home Care Services and Hospitalizations for Sickle Cell Disease Patients
- ClinicalTrials.gov ID
- NCT05649280
- Acronym / sponsor ID
- DREPADOM / AIR LIQUIDE HEALTH CARE
- Conditions
- Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Soutien aux Actions contre les Maladies du Globule RougeUniversity, hospital or other
- Study start
- 1 Feb 2021 (actual)
- Primary completion
- 1 Feb 2022 (actual)
- Study completion
- 1 Jun 2022 (actual)
- First posted
- 14 Dec 2022 (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 objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2)
Detailed description
This prospective, observational study will take place, as follows:
Calculation of the PRESEV score in the ER will determine or not eligibility of the patient in the DREPADOM structure. With a low score, a patient is eligible for home monitoring and after duly provided information, can sign a patient consent for the use of personal medical data.
The coordinating nurse from DREPADOM can then set up a whole network, with the delivery of oxygen supply at the patient's house, dispatch of a medical prescription to a neighboring pharmacy and daily visits from homecare service providers. Both opioids and parenteral hydration and treatments can be prescribed and daily visits, up to three, are programmed. As such, the patient receives a minimum of 1 visit per day from a nurse for monitoring and is provided with a medical oxygen concentrator for home use (3L/min continuously or for at least 15h/day) and parenteral hydration. In addition, patients are called daily by general practitioners or SCD experts, for teleconsultations.
The DREPADOM coordinating nurse is in constant interaction with nurses/providers of homecare services/medical doctors and status and evolution of the patient's global condition is reported both through the constant contact with the coordinating nurse from DREPADOM and in a dedicated software (Link4Life), with monitoring of blood pressure, temperature, respiratory rate, pain, etc… Link4Life is a software tool developed especially for home monitoring. It is adaptable to specific disorders, fully complies with the French Law for Health Data management and allows data extraction. Link4Life has been adapted to SCD in full collaboration with SCD medical experts of the Henri Mondor hospital. It is to be noted that all medical doctors (general practitioners or SCD experts), as well as coordinating and visiting nurses have all access to this application. As such, Link4Life will gather and capture all patient data.
When reporting patients' clinical parameters daily in the dedicated app (controlled by the DREPADOM team of experts), alerts are generated in the software when these are above or below determined thresholds. The medical team of experts from DREPADOM then takes over for daily teleconsultations and medical decisions for pursuing home monitoring or hospital admittance.
Of important note, a patient can be included twice in the DREPADOM study, for two distinct VOCs. Indeed, the PRESEV1 study highlighted that each crisis is unique and should be treated accordingly.
Data capture will be done through the Link4Life software, as well as extraction for data analysis.
- Open-label
- Blind
- Number of treatment arms:
Single arm
Multi arms specify the number of arms:
- Not Controlled
- Controlled, specify the comparator:
Versus placebo Versus reference product Versus other (specify) -Randomized: Parallel group Cross over Other (specify)
-Intervention (gas and/or drug, and/or device, and/or service /study arm):
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Sickle Cell Disease (SCD) is the most widespread genetic disease in the world. With an incidence rate of approximately 1/3000 newborns each year in France, International Standards consider SCD a rare disease. However, in the Ile-de-France region, that incidence drops to 1/435 newborns, rendering SCD a major territorial issue. It is estimated that more than 10 000 SCD patients live in this region, which represents approximately 30% of the French SCD population and 10% of the European SCD population. Vaso-occlusive crisis (VOC) or acute bone pain vaso-occlusion is a characteristic manifestation of SCD and represents the first cause of patients 'hospitalizations. Moreover, 16% of VOCs can develop into a potentially severe complication: the acute chest syndrome (ACS).
Key inclusion criteria (2 listed)
- SS or Sβ0
- Adult patients with a low PRESEV score
Key exclusion criteria (6 listed)
- SC/Sβ+ patients
- SCD adult patients with an intermediate or high PRESEV score
- Pregnant or breastfeeding women
- Homeless patient
- Patients without Social Security
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
Incidence of acute chest pain
Time frame: 30 months
The primary endpoint is the incidence of Acute chest pain complications developed by patients in the DREPADOM system ( Home Care Services and Hospitalizations for Sickle Cell Disease Patients) and comparison with the historical cohorts of( PRESEV1 and PRESEV2 studies).
1 secondary outcome
Incidence of clinical complications associated to Sickle cell disease
Time frame: 30 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 14 Dec 2022.
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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.
Pablo BARTOLUCCI, PROFESSOR principal investigator
Soutien aux Actions contre les Maladies du Globule Rouge
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, run by Soutien aux Actions contre les Maladies du Globule Rouge 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 2 inclusion and 6 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 14 Dec 2022. 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?
Incidence of acute chest pain (time frame: 30 months). 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 14 Dec 2022.