Sickle Cell DiseaseBIOCADRENCT03352986
Substudy of CADRE: for People With Extreme Phenotype: BIOCADRE
An observational study of Sickle Cell Disease, run by Cardiologie et Développement at 2 sites in 2 countries.
Trial summary
BIOCADRE is a CADRE substudy and aims to characterize more precisely the sickle cell patients with extreme phenotype. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- biological analysis and peripheral arterial tonometry
- How many people
- 300 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 15 May 2017to Dec 2019
- 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 10 years and older
- All sexes
- Child, Adult, Older adult
Study population: The study population is sickle cell patients with extreme phenotypes (SS-hemoglobin) with a steady state.
Who may be able to join
- sickle cell patients with extreme phenotypes: SS-hemoglobin
Who cannot join
- transfusion in the previous 2 months
- vaso-occlusive crisis in the previous 15 days
- infection in the previous 8 days
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
Mali1 site, 1 recruiting
- Recruiting
Centre de Recherches er de Lutte contre la Drépanocytose
Bamako
Dapa Diallo, MD, 0022320223898, da.diallo@laposte.net
Dapa Diallo, MD
Senegal1 site, 1 recruiting
- Recruiting
Centre National de Transfusion Sanguine
Dakar
Saliou Diop, MD, (221) 33 869 86 60, saliou.diop@ucad.edu.sn
Saliou Diop, MD
About this study
Official title: A Substudy of the CADRE Study: Determination of Clinical Markers in Patients With Extreme Sickle Cell Disease Phenotype
BIOCADRE is a CADRE substudy and aims to characterize more precisely the sickle cell patients with extreme phenotype.
Read the detailed description
Sickle cell disease (SCD) is the most frequent monogenic disease in the world, due to a unique mutation on the β-globin gene. Most affected individuals live in sub-Saharan Africa, yet, the natural history of the disease in Africa remains largely unknown. SCD usually presents in childhood and is characterized by the association of a chronic hemolytic anemia with episodes of acute vaso-occlusive events and progressive vascular organ damage. SCD is now widely recognized as a vascular disease with marked endothelial dysfunction. Hemolysis probably plays a key role by reducing NO bioavailability, but other involved mechanisms are not fully understood.
The project aims at better understanding SCD chronic vascular complications and in particular to explore extensively the different mechanisms associated with hemolysis. This will be addressed through both an epidemiological approach and a hypotheses-driven pathophysiological approach. On the one hand, a descriptive and analytic epidemiological study will isolate clusters of clinical, functional and usual biological phenotypes in SCD patients and look for new mechanistic and biological markers predictive of chronic vascular complications in SCD with SS phenotype. Investigators will specifically investigate i) microcirculation functions using peripheral arterial tonometry, ii) blood and plasma viscosities, iii) level of plasma blood cell derived microparticles, free hemoglobin, and the free heme content of erythrocytes-derived microparticles and expression of Duffy erythrocyte antigen, the unique erythroid receptor for chemokines. One the other hand, investigators will test novel markers and modifiers of hemolysis and heme metabolism and assess their relationship with inflammation and vascular phenotypes.These different biomarkers will be compared between the selected subgroups of patients with extreme vascular phenotype.
Methods: The project involves a transversal case control study, nested in the CADRE cohort, recently built up by Partner 1 and African collaborators. CADRE is the largest ongoing epidemiological cohort, including 4,300 SCD patients and 1,000 controls in five west and central African countries, in which various chronic complications of SCD have already been registered. The present second phase study will be conducted in the centres of Dakar and Bamako. Patients' selection will be performed in the existing database to obtain 6 subgroups of 40 SS patients with one of the main vascular chronic complications or none of them, for a total of 240 patients. Selected patients will be recalled during one year in parallel in the 2 recruiting centres. Clinical phenotyping, usual biology, functional microvascular functions (peripheral arterial tonometry), and blood/plasma viscosities analyses will be performed in the African recruiting centres after training of technicians, PhD and MD students by the French partners. Plasma samples, microparticles and extracellular DNA, will be prepared and frozen for further analyses in the partner's laboratories. DNA will be collected for each subject. A principal component analysis will isolate clusters of clinical complications, functional and biological markers and a multivariate logistic regression will quantify the effect of these markers on the risk of vasculopathy, with adjustment on all known SCD modifying factors.
Expected results: 1) The identification of high risk SCD patients for chronic vascular complications using new biomarkers, 2) A better understanding of chronic vascular disease process at the mechanistic and biological (viscosity, hemolysis, erythrocyte derived microparticles, inflammatory cytokines and receptors) levels, 3) The identification of endophenotypes and constitution of DNA bank for further genetic studies.
- Conditions
- Sickle Cell Disease
- Being tested
- biological analysis, peripheral arterial tonometry
- Keywords
- Sickle cell disease, anemia, Africa
- Medical subject headings
- Anemia, Sickle Cell, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
biological analysisBiological
biological analysis will be performed in the 6 groups of patients
peripheral arterial tonometryOther
peripheral arterial tonometry will be performed in the 6 groups of patients
| Study group | Role | What they receive |
|---|---|---|
| osteonecrosis | Sickle cell patients with osteonecrosis as a vascular main complication Receives: Biological: biological analysis; Other: peripheral arterial tonometry | |
| leg ulcer | Sickle cell patients with leg ulcer as a vascular main complication Receives: Biological: biological analysis; Other: peripheral arterial tonometry | |
| microalbuminuria | Sickle cell patients with microalbuminuria as a vascular main complication Receives: Biological: biological analysis; Other: peripheral arterial tonometry | |
| pulmonary hypertension | Sickle cell patients with pulmonary hypertension as a vascular main complication Receives: Biological: biological analysis; Other: peripheral arterial tonometry | |
| stroke | Sickle cell patients with strocke as a vascular main complication Receives: Biological: biological analysis; Other: peripheral arterial tonometry | |
| priapism | Sickle cell patients with priapism as a vascular main complication Receives: Biological: biological analysis; Other: peripheral arterial tonometry |
What is being measured
The primary outcome is the main question the study is designed to answer.
Description of microcirculation by peripheral arterial tonometry
EndoPAT2000®, Itamar Medical Ltd,
Measured: 1 year
Secondary outcomes (7)
Measure of blood viscosities
In whole blood by viscosimeter
Measured: 1 year
Dosage of microparticles
In plasma: microparticles of erythrocytic, platelet, monocytic, neutrophilic and endothelial origin by flow cytometry
Measured: 1 year
Determination of free hemoglobin
In plasma
Measured: 1 year
Measurement of neutrophil extracellular trap (NET)
In plasma
Measured: 1 year
Measurement of proinflammatory cytokines by ELISA
In plasma by ELISA
Measured: 1 year
Genotyping of the alpha-globin gene associated with persistence of fetal hemoglobin
In saliva DNA
Measured: 1 year
Genotyping of the polymorphisms associated with persistence of fetal hemoglobin
In saliva DNA
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2017
- Study start15 May 2017
- Primary completionJun 2019 (estimated)
- Study completionDec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (25)Papers the sponsor has linked to this study.
- 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
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cardiologie et Développement
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Brigitte Ranque, MD, Principal investigator, Institut National de la Santé Et de la Recherche Médicale, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03352986
- Sponsor's protocol ID
- 003
- Acronym
- BIOCADRE
- Status last verified
- Nov 2017
- First submitted
- 10 Oct 2017
- Last updated
- 24 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ranque B, Menet A, Diop IB, Thiam MM, Diallo D, Diop S, Diagne I, Sanogo I, Kingue S, Chelo D, Wamba G, Diarra M, Anzouan JB, N'Guetta R, Diakite CO, Traore Y, Legueun G, Deme-Ly I, Belinga S, Boidy K, Kamara I, Tharaux PL, Jouven X. Early renal damage in patients with sickle cell disease in sub-Saharan Africa: a multinational, prospective, cross-sectional study. Lancet Haematol. 2014 Nov;1(2):e64-73. doi: 10.1016/S2352-3026(14)00007-6. Epub 2014 Oct 28.
PubMed 27030156Ranque B, Menet A, Boutouyrie P, Diop IB, Kingue S, Diarra M, N'Guetta R, Diallo D, Diop S, Diagne I, Sanogo I, Tolo A, Chelo D, Wamba G, Gonzalez JP, Abough'elie C, Diakite CO, Traore Y, Legueun G, Deme-Ly I, Faye BF, Seck M, Kouakou B, Kamara I, Le Jeune S, Jouven X. Arterial Stiffness Impairment in Sickle Cell Disease Associated With Chronic Vascular Complications: The Multinational African CADRE Study. Circulation. 2016 Sep 27;134(13):923-33. doi: 10.1161/CIRCULATIONAHA.115.021015. Epub 2016 Aug 31.
PubMed 27582423Kato GJ, Gladwin MT, Steinberg MH. Deconstructing sickle cell disease: reappraisal of the role of hemolysis in the development of clinical subphenotypes. Blood Rev. 2007 Jan;21(1):37-47. doi: 10.1016/j.blre.2006.07.001. Epub 2006 Nov 7.
PubMed 17084951Hebbel RP. Reconstructing sickle cell disease: a data-based analysis of the "hyperhemolysis paradigm" for pulmonary hypertension from the perspective of evidence-based medicine. Am J Hematol. 2011 Feb;86(2):123-54. doi: 10.1002/ajh.21952.
PubMed 21264896Tharaux PL. Endothelin in renal injury due to sickle cell disease. Contrib Nephrol. 2011;172:185-199. doi: 10.1159/000328699. Epub 2011 Aug 30.
PubMed 21893999Connes P, Lamarre Y, Hardy-Dessources MD, Lemonne N, Waltz X, Mougenel D, Mukisi-Mukaza M, Lalanne-Mistrih ML, Tarer V, Tressieres B, Etienne-Julan M, Romana M. Decreased hematocrit-to-viscosity ratio and increased lactate dehydrogenase level in patients with sickle cell anemia and recurrent leg ulcers. PLoS One. 2013 Nov 4;8(11):e79680. doi: 10.1371/journal.pone.0079680. eCollection 2013.
PubMed 24223994Bunn HF, Nathan DG, Dover GJ, Hebbel RP, Platt OS, Rosse WF, Ware RE. Pulmonary hypertension and nitric oxide depletion in sickle cell disease. Blood. 2010 Aug 5;116(5):687-92. doi: 10.1182/blood-2010-02-268193. Epub 2010 Apr 15.
PubMed 20395414Kato GJ, McGowan V, Machado RF, Little JA, Taylor J 6th, Morris CR, Nichols JS, Wang X, Poljakovic M, Morris SM Jr, Gladwin MT. Lactate dehydrogenase as a biomarker of hemolysis-associated nitric oxide resistance, priapism, leg ulceration, pulmonary hypertension, and death in patients with sickle cell disease. Blood. 2006 Mar 15;107(6):2279-85. doi: 10.1182/blood-2005-06-2373. Epub 2005 Nov 15.
PubMed 16291595Kato GJ, Hebbel RP, Steinberg MH, Gladwin MT. Vasculopathy in sickle cell disease: Biology, pathophysiology, genetics, translational medicine, and new research directions. Am J Hematol. 2009 Sep;84(9):618-25. doi: 10.1002/ajh.21475.
PubMed 19610078Nouraie M, Lee JS, Zhang Y, Kanias T, Zhao X, Xiong Z, Oriss TB, Zeng Q, Kato GJ, Gibbs JS, Hildesheim ME, Sachdev V, Barst RJ, Machado RF, Hassell KL, Little JA, Schraufnagel DE, Krishnamurti L, Novelli E, Girgis RE, Morris CR, Rosenzweig EB, Badesch DB, Lanzkron S, Castro OL, Goldsmith JC, Gordeuk VR, Gladwin MT; Walk-PHASST Investigators and Patients. The relationship between the severity of hemolysis, clinical manifestations and risk of death in 415 patients with sickle cell anemia in the US and Europe. Haematologica. 2013 Mar;98(3):464-72. doi: 10.3324/haematol.2012.068965. Epub 2012 Sep 14.
PubMed 22983573Aessopos A, Farmakis D, Tsironi M, Diamanti-Kandarakis E, Matzourani M, Fragodimiri C, Hatziliami A, Karagiorga M. Endothelial function and arterial stiffness in sickle-thalassemia patients. Atherosclerosis. 2007 Apr;191(2):427-32. doi: 10.1016/j.atherosclerosis.2006.04.015. Epub 2006 May 18.
PubMed 16712855Tharaux PL, Girot R, Kanfer A, Dussaule JC, Gaitz JP, Tribout L, Baudot N, Vayssairat M. Cutaneous microvascular blood flow and reactivity in patients with homozygous sickle cell anaemia. Eur J Haematol. 2002 Jun;68(6):327-31. doi: 10.1034/j.1600-0609.2002.02701.x.
PubMed 12225389van Beers EJ, Schaap MC, Berckmans RJ, Nieuwland R, Sturk A, van Doormaal FF, Meijers JC, Biemond BJ; CURAMA study group. Circulating erythrocyte-derived microparticles are associated with coagulation activation in sickle cell disease. Haematologica. 2009 Nov;94(11):1513-9. doi: 10.3324/haematol.2009.008938. Epub 2009 Oct 8.
PubMed 19815831Donadee C, Raat NJ, Kanias T, Tejero J, Lee JS, Kelley EE, Zhao X, Liu C, Reynolds H, Azarov I, Frizzell S, Meyer EM, Donnenberg AD, Qu L, Triulzi D, Kim-Shapiro DB, Gladwin MT. Nitric oxide scavenging by red blood cell microparticles and cell-free hemoglobin as a mechanism for the red cell storage lesion. Circulation. 2011 Jul 26;124(4):465-76. doi: 10.1161/CIRCULATIONAHA.110.008698. Epub 2011 Jul 11.
PubMed 21747051Nebor D, Bowers A, Connes P, Hardy-Dessources MD, Knight-Madden J, Cumming V, Reid M, Romana M. Plasma concentration of platelet-derived microparticles is related to painful vaso-occlusive phenotype severity in sickle cell anemia. PLoS One. 2014 Jan 24;9(1):e87243. doi: 10.1371/journal.pone.0087243. eCollection 2014.
PubMed 24475257
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Substudy of the CADRE Study: Determination of Clinical Markers in Patients With Extreme Sickle Cell Disease Phenotype
- ClinicalTrials.gov ID
- NCT03352986
- Acronym / sponsor ID
- BIOCADRE / 003
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell disease, anemia, Africa
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Cardiologie et DéveloppementUniversity, hospital or other
- Collaborators
- Institut National de la Santé Et de la Recherche Médicale, France
- Study start
- 15 May 2017 (actual)
- Primary completion
- Jun 2019 (estimated)
- Study completion
- Dec 2019 (estimated)
- First posted
- 24 Nov 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.
BIOCADRE is a CADRE substudy and aims to characterize more precisely the sickle cell patients with extreme phenotype.
Detailed description
Sickle cell disease (SCD) is the most frequent monogenic disease in the world, due to a unique mutation on the β-globin gene. Most affected individuals live in sub-Saharan Africa, yet, the natural history of the disease in Africa remains largely unknown. SCD usually presents in childhood and is characterized by the association of a chronic hemolytic anemia with episodes of acute vaso-occlusive events and progressive vascular organ damage. SCD is now widely recognized as a vascular disease with marked endothelial dysfunction. Hemolysis probably plays a key role by reducing NO bioavailability, but other involved mechanisms are not fully understood.
The project aims at better understanding SCD chronic vascular complications and in particular to explore extensively the different mechanisms associated with hemolysis. This will be addressed through both an epidemiological approach and a hypotheses-driven pathophysiological approach. On the one hand, a descriptive and analytic epidemiological study will isolate clusters of clinical, functional and usual biological phenotypes in SCD patients and look for new mechanistic and biological markers predictive of chronic vascular complications in SCD with SS phenotype. Investigators will specifically investigate i) microcirculation functions using peripheral arterial tonometry, ii) blood and plasma viscosities, iii) level of plasma blood cell derived microparticles, free hemoglobin, and the free heme content of erythrocytes-derived microparticles and expression of Duffy erythrocyte antigen, the unique erythroid receptor for chemokines. One the other hand, investigators will test novel markers and modifiers of hemolysis and heme metabolism and assess their relationship with inflammation and vascular phenotypes.These different biomarkers will be compared between the selected subgroups of patients with extreme vascular phenotype.
Methods: The project involves a transversal case control study, nested in the CADRE cohort, recently built up by Partner 1 and African collaborators. CADRE is the largest ongoing epidemiological cohort, including 4,300 SCD patients and 1,000 controls in five west and central African countries, in which various chronic complications of SCD have already been registered. The present second phase study will be conducted in the centres of Dakar and Bamako. Patients' selection will be performed in the existing database to obtain 6 subgroups of 40 SS patients with one of the main vascular chronic complications or none of them, for a total of 240 patients. Selected patients will be recalled during one year in parallel in the 2 recruiting centres. Clinical phenotyping, usual biology, functional microvascular functions (peripheral arterial tonometry), and blood/plasma viscosities analyses will be performed in the African recruiting centres after training of technicians, PhD and MD students by the French partners. Plasma samples, microparticles and extracellular DNA, will be prepared and frozen for further analyses in the partner's laboratories. DNA will be collected for each subject. A principal component analysis will isolate clusters of clinical complications, functional and biological markers and a multivariate logistic regression will quantify the effect of these markers on the risk of vasculopathy, with adjustment on all known SCD modifying factors.
Expected results: 1) The identification of high risk SCD patients for chronic vascular complications using new biomarkers, 2) A better understanding of chronic vascular disease process at the mechanistic and biological (viscosity, hemolysis, erythrocyte derived microparticles, inflammatory cytokines and receptors) levels, 3) The identification of endophenotypes and constitution of DNA bank for further genetic studies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| osteonecrosis | – | Biological: biological analysis, Other: peripheral arterial tonometrySickle cell patients with osteonecrosis as a vascular main complication |
| leg ulcer | – | Biological: biological analysis, Other: peripheral arterial tonometrySickle cell patients with leg ulcer as a vascular main complication |
| microalbuminuria | – | Biological: biological analysis, Other: peripheral arterial tonometrySickle cell patients with microalbuminuria as a vascular main complication |
| pulmonary hypertension | – | Biological: biological analysis, Other: peripheral arterial tonometrySickle cell patients with pulmonary hypertension as a vascular main complication |
| stroke | – | Biological: biological analysis, Other: peripheral arterial tonometrySickle cell patients with strocke as a vascular main complication |
| priapism | – | Biological: biological analysis, Other: peripheral arterial tonometrySickle cell patients with priapism as a vascular main complication |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- The study population is sickle cell patients with extreme phenotypes (SS-hemoglobin) with a steady state.
Key inclusion criteria (1 listed)
- sickle cell patients with extreme phenotypes: SS-hemoglobin
Key exclusion criteria (3 listed)
- transfusion in the previous 2 months
- vaso-occlusive crisis in the previous 15 days
- infection in the previous 8 days
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 |
|---|---|---|
| biological analysisbiological analysis will be performed in the 6 groups of patients | Biological | leg ulcer, microalbuminuria, osteonecrosis, priapism, pulmonary hypertension, stroke |
| peripheral arterial tonometryperipheral arterial tonometry will be performed in the 6 groups of patients | Other | leg ulcer, microalbuminuria, osteonecrosis, priapism, pulmonary hypertension, stroke |
Primary outcome
Description of microcirculation by peripheral arterial tonometry
Time frame: 1 year
EndoPAT2000®, Itamar Medical Ltd,
7 secondary outcomes
Measure of blood viscosities
Time frame: 1 year
Dosage of microparticles
Time frame: 1 year
Determination of free hemoglobin
Time frame: 1 year
Measurement of neutrophil extracellular trap (NET)
Time frame: 1 year
Measurement of proinflammatory cytokines by ELISA
Time frame: 1 year
Genotyping of the alpha-globin gene associated with persistence of fetal hemoglobin
Time frame: 1 year
Genotyping of the polymorphisms associated with persistence of fetal hemoglobin
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2017.
- Background: PubMed 27030156Ranque B, Menet A, Diop IB, Thiam MM, Diallo D, Diop S, Diagne I, Sanogo I, Kingue S, Chelo D, Wamba G, Diarra M, Anzouan JB, N'Guetta R, Diakite CO, Traore Y, Legueun G, Deme-Ly I, Belinga S, Boidy K, Kamara I, Tharaux PL, Jouven X. Early renal damage in patients with sickle cell disease in sub-Saharan Africa: a multinational, prospective, cross-sectional study. Lancet Haematol. 2014 Nov;1(2):e64-73. doi: 10.1016/S2352-3026(14)00007-6. Epub 2014 Oct 28.
- Background: PubMed 27582423Ranque B, Menet A, Boutouyrie P, Diop IB, Kingue S, Diarra M, N'Guetta R, Diallo D, Diop S, Diagne I, Sanogo I, Tolo A, Chelo D, Wamba G, Gonzalez JP, Abough'elie C, Diakite CO, Traore Y, Legueun G, Deme-Ly I, Faye BF, Seck M, Kouakou B, Kamara I, Le Jeune S, Jouven X. Arterial Stiffness Impairment in Sickle Cell Disease Associated With Chronic Vascular Complications: The Multinational African CADRE Study. Circulation. 2016 Sep 27;134(13):923-33. doi: 10.1161/CIRCULATIONAHA.115.021015. Epub 2016 Aug 31.
- Background: PubMed 17084951Kato GJ, Gladwin MT, Steinberg MH. Deconstructing sickle cell disease: reappraisal of the role of hemolysis in the development of clinical subphenotypes. Blood Rev. 2007 Jan;21(1):37-47. doi: 10.1016/j.blre.2006.07.001. Epub 2006 Nov 7.
- Background: PubMed 21264896Hebbel RP. Reconstructing sickle cell disease: a data-based analysis of the "hyperhemolysis paradigm" for pulmonary hypertension from the perspective of evidence-based medicine. Am J Hematol. 2011 Feb;86(2):123-54. doi: 10.1002/ajh.21952.
- Background: PubMed 21893999Tharaux PL. Endothelin in renal injury due to sickle cell disease. Contrib Nephrol. 2011;172:185-199. doi: 10.1159/000328699. Epub 2011 Aug 30.
- Background: PubMed 24223994Connes P, Lamarre Y, Hardy-Dessources MD, Lemonne N, Waltz X, Mougenel D, Mukisi-Mukaza M, Lalanne-Mistrih ML, Tarer V, Tressieres B, Etienne-Julan M, Romana M. Decreased hematocrit-to-viscosity ratio and increased lactate dehydrogenase level in patients with sickle cell anemia and recurrent leg ulcers. PLoS One. 2013 Nov 4;8(11):e79680. doi: 10.1371/journal.pone.0079680. eCollection 2013.
- Background: PubMed 20395414Bunn HF, Nathan DG, Dover GJ, Hebbel RP, Platt OS, Rosse WF, Ware RE. Pulmonary hypertension and nitric oxide depletion in sickle cell disease. Blood. 2010 Aug 5;116(5):687-92. doi: 10.1182/blood-2010-02-268193. Epub 2010 Apr 15.
- Background: PubMed 16291595Kato GJ, McGowan V, Machado RF, Little JA, Taylor J 6th, Morris CR, Nichols JS, Wang X, Poljakovic M, Morris SM Jr, Gladwin MT. Lactate dehydrogenase as a biomarker of hemolysis-associated nitric oxide resistance, priapism, leg ulceration, pulmonary hypertension, and death in patients with sickle cell disease. Blood. 2006 Mar 15;107(6):2279-85. doi: 10.1182/blood-2005-06-2373. Epub 2005 Nov 15.
- Background: PubMed 19610078Kato GJ, Hebbel RP, Steinberg MH, Gladwin MT. Vasculopathy in sickle cell disease: Biology, pathophysiology, genetics, translational medicine, and new research directions. Am J Hematol. 2009 Sep;84(9):618-25. doi: 10.1002/ajh.21475.
- Background: PubMed 22983573Nouraie M, Lee JS, Zhang Y, Kanias T, Zhao X, Xiong Z, Oriss TB, Zeng Q, Kato GJ, Gibbs JS, Hildesheim ME, Sachdev V, Barst RJ, Machado RF, Hassell KL, Little JA, Schraufnagel DE, Krishnamurti L, Novelli E, Girgis RE, Morris CR, Rosenzweig EB, Badesch DB, Lanzkron S, Castro OL, Goldsmith JC, Gordeuk VR, Gladwin MT; Walk-PHASST Investigators and Patients. The relationship between the severity of hemolysis, clinical manifestations and risk of death in 415 patients with sickle cell anemia in the US and Europe. Haematologica. 2013 Mar;98(3):464-72. doi: 10.3324/haematol.2012.068965. Epub 2012 Sep 14.
- Background: PubMed 16712855Aessopos A, Farmakis D, Tsironi M, Diamanti-Kandarakis E, Matzourani M, Fragodimiri C, Hatziliami A, Karagiorga M. Endothelial function and arterial stiffness in sickle-thalassemia patients. Atherosclerosis. 2007 Apr;191(2):427-32. doi: 10.1016/j.atherosclerosis.2006.04.015. Epub 2006 May 18.
- Background: PubMed 12225389Tharaux PL, Girot R, Kanfer A, Dussaule JC, Gaitz JP, Tribout L, Baudot N, Vayssairat M. Cutaneous microvascular blood flow and reactivity in patients with homozygous sickle cell anaemia. Eur J Haematol. 2002 Jun;68(6):327-31. doi: 10.1034/j.1600-0609.2002.02701.x.
- 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.
Brigitte Ranque, MD principal investigator
Institut National de la Santé Et de la Recherche Médicale, France
2 sites in 2 countries: 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 Cardiologie et Développement at 2 sites in 2 countries.
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 1 inclusion and 3 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 24 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Mali, Senegal. 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 Brigitte Ranque, MD (0156092772, brigitte.ranque@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Description of microcirculation by peripheral arterial tonometry (time frame: 1 year). 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 NCT03352986, which also lists 25 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 24 Nov 2017.