Sickle Cell DiseaseDrepaformeNCT03977532
Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease
A study testing Micro-blood sampling in people with Sickle Cell Disease, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial summary
Sickle cell disease is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells (RBCs). They become more rigid and less deformable. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Micro-blood sampling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteFrance
- Study dates
- 14 Sep 2018to 3 Aug 2023
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Homozygous SS or SB° sickle cell patients
- Person without regular blood transfusion
Who cannot join
- Other general diseases
- Psychiatric disorder
- Alcoholics
- Minors
- Absence of informed consent
- Pregnant or lactating woman
Show all 13Show fewer
- Person under guardianship
- Person living in a health or social institution
- Person in emergency
- Person out of healthcare system
- Person deprived of liberty
- Person who has received a blood transfusion in the previous 3 months
- Person with regular blood transfusion or in a blood transfusion program
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease, at Basal State and During Vaso-occlusive Crisis
Sickle cell disease is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells (RBCs). They become more rigid and less deformable. The fragility of sickle-cell RBCs leads to their massive destruction, leading to chronic anemia (i.e. low hemoglobin in the blood) and to low tissue oxygenation. More rigid and less deformable, sickle-shaped RBCs tend to obstruct microvessels, leading to particularly painful vaso-occlusive crisis (VOC), which can cause organ failure (spleen, kidneys, brain, lungs, heart, liver, bone ...) and patient's lifethreatning. A preliminary work on red blood cells of sickle cell patients showed alteration of a parameter measuring the overall deformability of RBCs by assessing the nature of their movement in a shear flow. This parameter is altered sickle cell patients at basal state compared to a population of healthy individuals. This alteration is increased when sickle cell patients are in crisis.
The main objective of this project is to study the evolution of this parameter in sickle cell patients according to their health status (basal state vs vaso-occlusive crisis). The investigators hypothesize that the alteration of the RBC deformability parameter is significant before symptoms of vaso-occlusive crisis (several hours to several days). The main objective is a weekly analysis of the evolution of the parameter in 30 sickle cell patients (SS or SB°) in the basal state and daily in at least 6 patients at the beginning, during and just after a vaso-occlusive crisis. The comparison between the parameter measured in a subject in the basal state and in the same subject in crisis will be performed. The criteria for the presence of a vaso-occlusive crisis were: the appearance of a attacks of pain affecting at least two territories +/- fever> 38.3 +/- dyspnea and / or sputum. The investigators will differentiate the moderate VOC managed at home with low-level analgesics and VOC requiring hospitalization. The number of days of hospitalization, the occurrence of thrombotic complications, the degree of anemia and hemolysis will be noted. The measurement of the parameter will be performed on a capillary sampling of 40 microliters performed at the fingertip, weekly outside crises and daily when a crisis occurs.
- Conditions
- Sickle Cell Disease
- Being tested
- Micro-blood sampling
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Micro-blood samplingOther
capillary sampling of 40 microliters performed at the fingertip
| Study group | Role | What they receive |
|---|---|---|
| patients with sickle cell disease | Experimental | Not described Receives: Other: Micro-blood sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
cytoplasmic viscosity measurement of red blood cells
Determination of the cytoplasmic viscosity parameter variations by measuring percentage of tanktreading (nature of movement of cells in a shear flow) from a micro-blood sample
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2019
- Study start14 Sep 2018
- Primary completion11 Dec 2019
- Study completion3 Aug 2023
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.
- More trials run by Assistance Publique Hopitaux De MarseilleThe 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Emilie Garrido Pradalié, Study director, Assistance Publique Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03977532
- Sponsor's protocol ID
- 2018-36
- Acronym
- Drepaforme
- Status last verified
- Aug 2023
- First submitted
- 3 Jun 2019
- Last updated
- 4 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease, at Basal State and During Vaso-occlusive Crisis
- ClinicalTrials.gov ID
- NCT03977532
- Acronym / sponsor ID
- Drepaforme / 2018-36
- Conditions
- Sickle Cell Disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- 14 Sep 2018 (actual)
- Primary completion
- 11 Dec 2019 (actual)
- Study completion
- 3 Aug 2023 (actual)
- First posted
- 6 Jun 2019 (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 is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells (RBCs). They become more rigid and less deformable. The fragility of sickle-cell RBCs leads to their massive destruction, leading to chronic anemia (i.e. low hemoglobin in the blood) and to low tissue oxygenation. More rigid and less deformable, sickle-shaped RBCs tend to obstruct microvessels, leading to particularly painful vaso-occlusive crisis (VOC), which can cause organ failure (spleen, kidneys, brain, lungs, heart, liver, bone ...) and patient's lifethreatning. A preliminary work on red blood cells of sickle cell patients showed alteration of a parameter measuring the overall deformability of RBCs by assessing the nature of their movement in a shear flow. This parameter is altered sickle cell patients at basal state compared to a population of healthy individuals. This alteration is increased when sickle cell patients are in crisis.
The main objective of this project is to study the evolution of this parameter in sickle cell patients according to their health status (basal state vs vaso-occlusive crisis). The investigators hypothesize that the alteration of the RBC deformability parameter is significant before symptoms of vaso-occlusive crisis (several hours to several days). The main objective is a weekly analysis of the evolution of the parameter in 30 sickle cell patients (SS or SB°) in the basal state and daily in at least 6 patients at the beginning, during and just after a vaso-occlusive crisis. The comparison between the parameter measured in a subject in the basal state and in the same subject in crisis will be performed. The criteria for the presence of a vaso-occlusive crisis were: the appearance of a attacks of pain affecting at least two territories +/- fever> 38.3 +/- dyspnea and / or sputum. The investigators will differentiate the moderate VOC managed at home with low-level analgesics and VOC requiring hospitalization. The number of days of hospitalization, the occurrence of thrombotic complications, the degree of anemia and hemolysis will be noted. The measurement of the parameter will be performed on a capillary sampling of 40 microliters performed at the fingertip, weekly outside crises and daily when a crisis occurs.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with sickle cell disease | Experimental | Other: Micro-blood sampling |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Homozygous SS or SB° sickle cell patients
- Person without regular blood transfusion
Key exclusion criteria (13 listed)
- Other general diseases
- Psychiatric disorder
- Alcoholics
- Minors
- Absence of informed consent
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 |
|---|---|---|
| Micro-blood samplingcapillary sampling of 40 microliters performed at the fingertip | Other | patients with sickle cell disease |
Primary outcome
cytoplasmic viscosity measurement of red blood cells
Time frame: 6 months
Determination of the cytoplasmic viscosity parameter variations by measuring percentage of tanktreading (nature of movement of cells in a shear flow) from a micro-blood sample
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 4 Aug 2023.
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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.
Emilie Garrido Pradalié study director
Assistance Publique Hôpitaux de Marseille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Micro-blood sampling in people with Sickle Cell Disease, run by Assistance Publique Hopitaux De Marseille 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 13 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 4 Aug 2023. 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?
cytoplasmic viscosity measurement of red blood cells (time frame: 6 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 4 Aug 2023.