Sickle Cell DiseaseSAPOTILLENCT03368261
Epidemiology and Pathophysiological Mechanisms of HTAP in SS and SC Children in Martinique and Guadeloupe.
A study testing HTAP/ clinical complications in the sickle cell disease in people with Sickle Cell Disease, run by Centre Hospitalier Universitaire de la Guadeloupe at 1 site in Martinique.
Trial summary
pulmonary arterial hypertension (PAH) has been reported with a prevalence of approximately 30% in adult sickle cell disease (SCD) patients, with an increased mortality in SCD patients with PAH, compared with those without PAH. Read more about this study
- Who is it for?
- Ages 8 to 16 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- HTAP/ clinical complications in the sickle cell disease
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 185 participants
- Where
- 1 siteMartinique
- Study dates
- 11 Jan 2010to 17 Oct 2016
- 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 8 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- children aged between 8 to 16 years old,
- SS homozygote or SC compound heterozygote, followed by the sickle cell centers of Guadeloupe and Martinique or by the pediatric department of the university hospital of Fort-de-France, identified by the systematic neonatal screening programs performed in Guadeloupe and Martinique or by other labeled centers, registered in the French medical social security national program, and for which the parental and the old children consent has been obtained.
Who cannot join
- other hemoglobinopathies, chronic transfusion therapy programs or treatments which affected the expression of the biomarkers studied (unless hydroxyurea treatment),
- recent blood transfusion or phlebotomies (less than 3 months);
- patients not at steady state,
- pregnancy or breast feeding,
- refusal of parental and old children consent.
Trial locations and contacts
1 site in 1 country.
Martinique1 site
Hospital University Center of Martinique
Fort-de-France, 97261
About this study
Official title: Epidemiology and Pathophysiological Mechanisms of Pulmonary Arterial Hypertension in SS and SC Children in Martinique and Guadeloupe.Three Years Follow up of a Cohort of Children Aged From 8 to 16 Years Old
pulmonary arterial hypertension (PAH) has been reported with a prevalence of approximately 30% in adult sickle cell disease (SCD) patients, with an increased mortality in SCD patients with PAH, compared with those without PAH. The identification of several hemolysis biomarkers such as lactate dehydrogenase, bilirubin, reticulocytes or hemoglobin level, has clearly documented a link between hemolysis and PAH. However, other physiopathological mechanisms may be involved to explain PAH in these patients, such as pulmonary thromboembolism, pulmonary fibrosis or left heart diastolic and / or systolic dysfunction.
The investigators suggest studying HTAP in patient's presenting the most frequent both drepanocytic syndromes, SS and SC and homogeneous in their medical coverage and the association between HTAP risk and specific SCD complications.
Read the detailed description
The primary aim of this study is to estimate the prevalence and the incidence of PAH in a population of SCD children (SS, SC) with similar medical caring, aged from 8 to 16 years old.
Unlike the important number of studies in SCD adults, very few SCD children studies were performed. None of these studies reported the mortality rate associated with PAH in children although the literature reported a decrease of this morbid-mortality comparing different medical caring of the patients. The investigators hypothesized that physiopathological mechanisms responsible for PAH had to be different in SCD children compared with adults, as most degenerative processes had no time enough to appear during children's lives. At the inclusion in our study the diagnosis of PAH will be performed by transthoracic Doppler-echocardiograms in a group of 306 children (aged between 8 to 16 years old) with either SS or SC genotype with similar medical caring, to avoid a known selection bias. These patients will be followed during a 3 years longitudinal period. The occurrence of the clinical specific complications associated with SCD (acute chest syndrome, painful vaso occlusive crisis, septicemia and stroke) and the observed mortality rate of our children group, will be compared in patients groups stratified according to the occurrence of PAH. The expression of several molecular and cellular genetic biomarkers potentially associated with this complication will also be studied.
- Conditions
- Sickle Cell Disease
- Being tested
- HTAP/ clinical complications in the sickle cell disease
- Keywords
- Pulmonary arterial hypertension, SCD, Epidemiology
- Medical subject headings
- Anemia, Sickle Cell, Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HTAP/ clinical complications in the sickle cell diseaseOther
At the inclusion in our study the diagnosis of PAH will be performed by transthoracic Doppler-echocardiograms in a group of 306 children (aged between 8 to 16 years old) with either SS or SC genotype with similar medical caring, to avoid a known selection bias. These patients will be followed during a 3 years longitudinal period. The occurrence of the clinical specific complications associated with SCD (acute chest syndrome, painful vaso occlusive crisis, septicemia and stroke) and the observed mortality rate of our children group, will be compared in patients groups stratified according to the occurrence of PAH. The expression of several molecular and cellular genetic biomarkers potentially associated with this complication will also be studied.
| Study group | Role | What they receive |
|---|---|---|
| HTAP/ clinical complications in the sickle cell disease | Other | Supply epidemiological data on this detected HTAP, and allow the characterization of the clinico-biological paintings and the mortality which are associated to them. Receives: Other: HTAP/ clinical complications in the sickle cell disease |
What is being measured
The primary outcome is the main question the study is designed to answer.
incidence of pulmonary arterial hypertension
The primary outcome of the present study is to estimate the incidence of pulmonary arterial hypertension documented by the presence of tricuspid regurgitation jet velocity of at least 2.5 ms-1 by Doppler echocardiographic assessment.
Measured: Through study completion, an average of 5 years
Secondary outcomes (1)
The association between PAH risk and specific SCD complications
To determine the association between PAH risk and specific SCD complications (painful crisis, acute chest syndrome, severe infectious events, stroke, cerebral vasculopathy), expression of molecular (pro-PBN, nitrite/ nitrate compounds, sVCAM-1, sICAM-1, S- and P-selectine, plasmatic hemoglobin, ET-1, CD40L), cellular (microparticles, hemorheological parameters) biomarkers, and genetic markers (alpha-globin, type 3 NOS, endothélin-1, ACVRL1, BMPR2, BMP6).
To determine if PAH is a risk factor of the clinical complications cited previously above and of mortality.
Measured: Through study completion, an average of 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2017
- Study start11 Jan 2010
- Primary completion2 Oct 2013
- Study completion17 Oct 2016
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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 185 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maryse ETIENNE-JULAN, Doctor specializing in SCD, Study director, Hospital University Center of Pointe-à-Pitre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03368261
- Sponsor's protocol ID
- RBM-PAP-2009/69
- Acronym
- SAPOTILLE
- Status last verified
- Dec 2017
- First submitted
- 1 Dec 2017
- Last updated
- 11 Dec 2017
- 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 11 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Epidemiology and Pathophysiological Mechanisms of Pulmonary Arterial Hypertension in SS and SC Children in Martinique and Guadeloupe.Three Years Follow up of a Cohort of Children Aged From 8 to 16 Years Old
- ClinicalTrials.gov ID
- NCT03368261
- Acronym / sponsor ID
- SAPOTILLE / RBM-PAP-2009/69
- Conditions
- Sickle Cell Disease
- Keywords
- Pulmonary arterial hypertension, SCD, Epidemiology
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 185 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire de la GuadeloupeUniversity, hospital or other
- Study start
- 11 Jan 2010 (actual)
- Primary completion
- 2 Oct 2013 (actual)
- Study completion
- 17 Oct 2016 (actual)
- First posted
- 11 Dec 2017 (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.
pulmonary arterial hypertension (PAH) has been reported with a prevalence of approximately 30% in adult sickle cell disease (SCD) patients, with an increased mortality in SCD patients with PAH, compared with those without PAH. The identification of several hemolysis biomarkers such as lactate dehydrogenase, bilirubin, reticulocytes or hemoglobin level, has clearly documented a link between hemolysis and PAH. However, other physiopathological mechanisms may be involved to explain PAH in these patients, such as pulmonary thromboembolism, pulmonary fibrosis or left heart diastolic and / or systolic dysfunction.
The investigators suggest studying HTAP in patient's presenting the most frequent both drepanocytic syndromes, SS and SC and homogeneous in their medical coverage and the association between HTAP risk and specific SCD complications.
Detailed description
The primary aim of this study is to estimate the prevalence and the incidence of PAH in a population of SCD children (SS, SC) with similar medical caring, aged from 8 to 16 years old.
Unlike the important number of studies in SCD adults, very few SCD children studies were performed. None of these studies reported the mortality rate associated with PAH in children although the literature reported a decrease of this morbid-mortality comparing different medical caring of the patients. The investigators hypothesized that physiopathological mechanisms responsible for PAH had to be different in SCD children compared with adults, as most degenerative processes had no time enough to appear during children's lives. At the inclusion in our study the diagnosis of PAH will be performed by transthoracic Doppler-echocardiograms in a group of 306 children (aged between 8 to 16 years old) with either SS or SC genotype with similar medical caring, to avoid a known selection bias. These patients will be followed during a 3 years longitudinal period. The occurrence of the clinical specific complications associated with SCD (acute chest syndrome, painful vaso occlusive crisis, septicemia and stroke) and the observed mortality rate of our children group, will be compared in patients groups stratified according to the occurrence of PAH. The expression of several molecular and cellular genetic biomarkers potentially associated with this complication will also be studied.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HTAP/ clinical complications in the sickle cell disease | Other | Other: HTAP/ clinical complications in the sickle cell diseaseSupply epidemiological data on this detected HTAP, and allow the characterization of the clinico-biological paintings and the mortality which are associated to them. |
Who may qualify
- Ages
- 8 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- children aged between 8 to 16 years old,
- SS homozygote or SC compound heterozygote, followed by the sickle cell centers of Guadeloupe and Martinique or by the pediatric department of the university hospital of Fort-de-France, identified by the systematic neonatal screening programs performed in Guadeloupe and Martinique or by other labeled centers, registered in the French medical social security national program, and for which the parental and the old children consent has been obtained.
Key exclusion criteria (5 listed)
- other hemoglobinopathies, chronic transfusion therapy programs or treatments which affected the expression of the biomarkers studied (unless hydroxyurea treatment),
- recent blood transfusion or phlebotomies (less than 3 months);
- patients not at steady state,
- pregnancy or breast feeding,
- refusal of parental and old children 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 |
|---|---|---|
| HTAP/ clinical complications in the sickle cell diseaseAt the inclusion in our study the diagnosis of PAH will be performed by transthoracic Doppler-echocardiograms in a group of 306 children (aged between 8 to 16 years old) with either SS or SC genotype with similar medical caring, to avoid a known selection bias. These patients will be followed during a 3 years longitudinal period. The occurrence of the clinical specific complications associated with SCD (acute chest syndrome, painful vaso occlusive crisis, septicemia and stroke) and the observed mortality rate of our children group, will be compared in patients groups stratified according to the occurrence of PAH. The expression of several molecular and cellular genetic biomarkers potentially associated with this complication will also be studied. | Other | HTAP/ clinical complications in the sickle cell disease |
Primary outcome
incidence of pulmonary arterial hypertension
Time frame: Through study completion, an average of 5 years
The primary outcome of the present study is to estimate the incidence of pulmonary arterial hypertension documented by the presence of tricuspid regurgitation jet velocity of at least 2.5 ms-1 by Doppler echocardiographic assessment.
1 secondary outcome
The association between PAH risk and specific SCD complications
Time frame: Through study completion, an average of 5 years
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 11 Dec 2017.
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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.
Maryse ETIENNE-JULAN, Doctor specializing in SCD study director
Hospital University Center of Pointe-à-Pitre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing HTAP/ clinical complications in the sickle cell disease in people with Sickle Cell Disease, run by Centre Hospitalier Universitaire de la Guadeloupe at 1 site in Martinique.
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 5 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 11 Dec 2017. 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 Martinique. 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 pulmonary arterial hypertension (time frame: Through study completion, an average of 5 years). 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 11 Dec 2017.