Vaso Occlusive CrisisPresev2NCT03032055
Validation of a Predictive Score of Acute Chest Syndrome
An observational study of Vaso Occlusive Crisis and Acute Chest Syndrome, run by Soutien aux Actions contre les Maladies du Globule Rouge at 1 site in France.
Trial summary
Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is the first cause of death, particularly when complicated by an acute chest syndrome (ACS). The PRESEV score could help the physicians to better manage VOC and could be used for future therapeutic trials. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Vaso Occlusive Crisis, Acute Chest Syndrome
- How many people
- 800 participants
- Where
- 1 siteFrance
- Study dates
- 1 Jan 2016to 31 Dec 2023
- 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: Children (>2 years) and adults * Male and Female * Homozygous SCD patients * VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes
Who may be able to join
- Children (>2 years) and adults
- Male and Female
- Homozygous SCD patients
- VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes.
- Patient has health care insurance (in Europe)
- Written consent given after being informed of the purpose, progress and potential risks
Who cannot join
- No inaugural Acute Chest Syndrome
- Homeless patients
- Deprived of their liberty by a court or administrative order or under guardianship
- Unable to understand the purpose and conditions of carrying out the study, unable to give consent
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Henri Mondor Hospital
Créteil, 94000
Pablo BARTOLUCCI, Professor, 33 (1) 49817406, pablo.bartolucci@aphp.fr
About this study
Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is the first cause of death, particularly when complicated by an acute chest syndrome (ACS).
The PRESEV score could help the physicians to better manage VOC and could be used for future therapeutic trials. This predictive score of secondary ACS has to be validated in a multicenter international study.
Read the detailed description
International multicentre prospective observational study.
Prediction of an ACS within 15 days after admission by the PRESEV2 score at arrival.
Validation of a Predictive Score of Acute Chest Syndrome (Presev2) associates a categorical pain score of the spine /or pelvis and 3 biological parameters: Reticulocytes, Leucocytes and Hemoglobin.
ACS is defined by crepitant or bronchial breathing or the association of new radiologic infiltrate and chest pain
Inclusion criteria:
- Children (>2 years) and adults
- Male and Female
- Homozygous SCD patients
- The patient can only be included once
- VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes.
- Patient has health care insurance (in Europe)
- Written consent given after being informed of the purpose, progress and potential risks
Exclusion criteria:
- No inaugural Acute Chest Syndrome
- Homeless patients
- Deprived of their liberty by a court or administrative order or under guardianship
- Unable to understand the purpose and conditions of carrying out the study, unable to give consent
STUDY SCHEDULE Screening and inclusion once patients are admitted at the accident and emergency department or medical day unit.
Inclusion visit (day 1) Once admitted at the accident and emergency department, the patient will be informed about the protocol and asked to participate in the study. Informed consent will be obtained according to local regulations by signing the informed consent form. The inclusion and non-inclusion criteria will be verified.
Demographic data, current and previous treatments taken within one month, and medical history will be recorded.
The following parameters of the score will be collected:
Reticulocytes and/or leucocytes counts, urea (mmol/L) and Categorical pain score.
The Score is adjusted with Hydroxyurea treatment (Yes/No) and Hb level (g/dL). Plasmodium falciparum test will be performed only in Africa. Hemoculture if fever (>38°C) is recorded within the 2 first days after admission.
Temperature, Blood pressure, Oxygen saturation, Respiratory rate, Visual analogue score will be recorded.
STUDY DURATION Inclusion period: 2 years Per patient: 15 days Total duration of the study: 2 years
- Conditions
- Vaso Occlusive Crisis, Acute Chest Syndrome
- Keywords
- sickle cell desease, vaso occlusive crisis, acute chest syndrome, Validation of predictif score of acute chest syndrome
- Medical subject headings
- Vaso-Occlusive Crises, Acute Chest Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| VOC | Patients who won't develop a secondary Acute chest syndrome during a vaso occlusive crisis within 15 days after admission. Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate and chest pain or decreased breath sounds . A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries. | |
| 2°ACS | Patients who will develop a secondary acute chest syndrome during a vaso occlusive crisis within 15 days after admission. Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate AND chest pain or decreased breath sounds . A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults.It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome
The primary outcome mesure is the occurrence of an ACS defined by crepitant or bronchial breathing or the association of new radiologic infiltrats and chest pain during the first 15 days of hospitalization for Vaso Occlusive Crisis
Measured: 2 years
Secondary outcomes (1)
Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome
Association of the PRESEV 2 score with the following parameters collected for each patient:
- The number of days of hospitalization
- Blood transfusion requirement
- The number of hospitalization in intensive care unit
- Death
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2017 (estimated)
- Study start1 Jan 2016
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Vaso Occlusive CrisisStudies 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
- 800 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Soutien aux Actions contre les Maladies du Globule Rouge
- Sponsor type
- University, hospital or other
- Collaborators
- Theravia, Pierre Fabre Laboratories
- Responsible party
- Sponsor
- Investigators
- BARTOLUCCI Pablo, MD PhD, Pr, Principal investigator, APHP, UPEC, INSERM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03032055
- Sponsor's protocol ID
- Presev 2 IRB 00003835
- Acronym
- Presev2
- Status last verified
- Dec 2022
- First submitted
- 18 Nov 2015
- Last updated
- 13 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Kassasseya C, Kene S, Besse-Hammer T, Nzouakou R, Magnang H, Telfer P, Arlet JB, de Luna G, Affo L, Dautheville S, Ngo S, Pelinski Y, Mescam C, Djoumad S, Pham Hung d'Alexandry d'Orengiani AL, Mekontso Dessap A, Michel M, Galacteros F, Audureau E, Guindo A, Habibi A, Khellaf M, Diallo D, Bartolucci P. Validation and Application of a Predictive Score of Acute Chest Syndrome. NEJM Evid. 2026 Jan;5(1):EVIDoa2500074. doi: 10.1056/EVIDoa2500074. Epub 2025 Dec 18.
PubMed 41410575
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of a Predictive Score of Acute Chest Syndrome
- ClinicalTrials.gov ID
- NCT03032055
- Acronym / sponsor ID
- Presev2 / Presev 2 IRB 00003835
- Conditions
- Vaso Occlusive Crisis, Acute Chest Syndrome
- Keywords
- sickle cell desease, vaso occlusive crisis, acute chest syndrome, Validation of predictif score of acute chest syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 800 participants (estimated)
- Lead sponsor
- Soutien aux Actions contre les Maladies du Globule RougeUniversity, hospital or other
- Collaborators
- Theravia, Pierre Fabre Laboratories
- Study start
- 1 Jan 2016 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 26 Jan 2017 (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.
Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is the first cause of death, particularly when complicated by an acute chest syndrome (ACS).
The PRESEV score could help the physicians to better manage VOC and could be used for future therapeutic trials. This predictive score of secondary ACS has to be validated in a multicenter international study.
Detailed description
International multicentre prospective observational study.
Prediction of an ACS within 15 days after admission by the PRESEV2 score at arrival.
Validation of a Predictive Score of Acute Chest Syndrome (Presev2) associates a categorical pain score of the spine /or pelvis and 3 biological parameters: Reticulocytes, Leucocytes and Hemoglobin.
ACS is defined by crepitant or bronchial breathing or the association of new radiologic infiltrate and chest pain
Inclusion criteria:
- Children (>2 years) and adults
- Male and Female
- Homozygous SCD patients
- The patient can only be included once
- VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes.
- Patient has health care insurance (in Europe)
- Written consent given after being informed of the purpose, progress and potential risks
Exclusion criteria:
- No inaugural Acute Chest Syndrome
- Homeless patients
- Deprived of their liberty by a court or administrative order or under guardianship
- Unable to understand the purpose and conditions of carrying out the study, unable to give consent
STUDY SCHEDULE Screening and inclusion once patients are admitted at the accident and emergency department or medical day unit.
Inclusion visit (day 1) Once admitted at the accident and emergency department, the patient will be informed about the protocol and asked to participate in the study. Informed consent will be obtained according to local regulations by signing the informed consent form. The inclusion and non-inclusion criteria will be verified.
Demographic data, current and previous treatments taken within one month, and medical history will be recorded.
The following parameters of the score will be collected:
Reticulocytes and/or leucocytes counts, urea (mmol/L) and Categorical pain score.
The Score is adjusted with Hydroxyurea treatment (Yes/No) and Hb level (g/dL). Plasmodium falciparum test will be performed only in Africa. Hemoculture if fever (>38°C) is recorded within the 2 first days after admission.
Temperature, Blood pressure, Oxygen saturation, Respiratory rate, Visual analogue score will be recorded.
STUDY DURATION Inclusion period: 2 years Per patient: 15 days Total duration of the study: 2 years
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VOC | – | –Patients who won't develop a secondary Acute chest syndrome during a vaso occlusive crisis within 15 days after admission. Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate and chest pain or decreased breath sounds . A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries. |
| 2°ACS | – | –Patients who will develop a secondary acute chest syndrome during a vaso occlusive crisis within 15 days after admission. Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate AND chest pain or decreased breath sounds . A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults.It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children (>2 years) and adults * Male and Female * Homozygous SCD patients * VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes
Key inclusion criteria (6 listed)
- Children (>2 years) and adults
- Male and Female
- Homozygous SCD patients
- VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes.
- Patient has health care insurance (in Europe)
Key exclusion criteria (4 listed)
- No inaugural Acute Chest Syndrome
- Homeless patients
- Deprived of their liberty by a court or administrative order or under guardianship
- Unable to understand the purpose and conditions of carrying out the study, unable to give consent
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
Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome
Time frame: 2 years
The primary outcome mesure is the occurrence of an ACS defined by crepitant or bronchial breathing or the association of new radiologic infiltrats and chest pain during the first 15 days of hospitalization for Vaso Occlusive Crisis
1 secondary outcome
Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2022.
- Derived from this study: PubMed 41410575Kassasseya C, Kene S, Besse-Hammer T, Nzouakou R, Magnang H, Telfer P, Arlet JB, de Luna G, Affo L, Dautheville S, Ngo S, Pelinski Y, Mescam C, Djoumad S, Pham Hung d'Alexandry d'Orengiani AL, Mekontso Dessap A, Michel M, Galacteros F, Audureau E, Guindo A, Habibi A, Khellaf M, Diallo D, Bartolucci P. Validation and Application of a Predictive Score of Acute Chest Syndrome. NEJM Evid. 2026 Jan;5(1):EVIDoa2500074. doi: 10.1056/EVIDoa2500074. Epub 2025 Dec 18.
- Individual participant dataSharing planned: No
Related trials for Vaso Occlusive Crisis
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
RecruitingPhase 2
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
Sickle Cell Disease
Not yet recruiting
Hypoxic Red Blood Cells in Sickle Cell Anemia
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Recruiting
Virtual Reality for SCD VOC
Sickle Cell Disease
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
Sickle Cell Disease
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.
BARTOLUCCI Pablo, MD PhD, Pr principal investigator
APHP, UPEC, INSERM
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 Vaso Occlusive Crisis and Acute Chest Syndrome, 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 6 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: status not confirmed, as of the sponsor's last update on 13 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact DJOUMAD Sabrina, Master's (33 (1) 49812441, sabrina.djoumad@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome (time frame: 2 years). 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 NCT03032055, which also lists 1 linked publication.
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 13 Dec 2022.