HDL2: Lipid Balance in Adult Sickle Cell Patients

Sponsor

Centre Hospitalier Universitaire de Pointe-a-Pitre (Other)

Overall Status

Recruiting

CT.gov ID

NCT05780775

Collaborator

Direction Générale de l'Offre de Soins (Other)

350

Enrollment

Locations

Anticipated Duration (Months)

175

Patients Per Site

2.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study aims to describe and/or searches for, in cohorts of adult sickle cell anemia (SCA) and SC sickle cell patients living in the French West Indies and followed by SCD Reference and Competence Centers: 1-lipids profiles and associations at steady state with occurrence of sickle cell disease (SCD) complications, 2-lipids profile evolution during and after prospective acute complications (vasoocclusive crises (VOC) and priapism), 3-lipids profile variation (inter /intra individuals) during 4 prospective years, 4- Genetic primary modulators of SCD complications, 5- insulin resistance (HOMA), free fatty acids and glycerol dosages, 6- lipids enzymes, lipidome and functionality of HDL in sub-groups of SCD population.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease Dyslipidemia Complication	Other: HDL2	N/A

Detailed Description

Cohorts of sickle cell disease patients including sickle cell anemia (SCA) and SC sickle cell patients living in Guadeloupe and Martinique and followed by the Sickle cell disease (SCD) Reference and Competence Centers of French West Indies.
Lipid profile includes total cholesterol, HDL-cholesterol, non-HDL-cholesterol, LDL-cholesterol and triglycerides, apolipoprotein A-I and B.

Medical histories and prospective collection of SCD complications include retinopathy, deafness, tinnitus, osteonecrosis, leg ulcers, strokes, acute chest syndrome, VOC, priapism, pulmonary arterial hypertension (PAH) and PAH sd (echocardiography diagnosed when tricuspid regurgitant jet velocity ≥2.5 m/sec), kidney disease: chronic renal insufficiency and/or nephropathy.

Objective 4: to describe genetic primary modulators of SCD complications: fetal hemoglobin, alpha-thalassemia, haplotypes of beta S gene.
Objective 5 will be performed in the entire cohort at inclusion and during prospective complications (VOC, priapism).
Objective 6 will be performed in a sub-group of 90 individuals (n=15 with VOC and n= 15 without VOC, n=15 with priapism and n=15 without priapism, n= 15 with pulmonary arterial hypertension syndrome (PAH Sd) and n=15 without PAH Sd), as well as in a subgroup of n = 15 patients prospectively experiencing VOC and n = 15 patients prospectively experiencing priapism.

A collection of plasma is performed to fulfill objective 6, as well as a collection of blood cells for later researches.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

350 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Basic Science

Official Title:

Study of Lipid Balance in Adult Sickle Cell SS or SC Patients at Steady State and According to Clinical Phenotypes and During Acute Complications Acronym : "HDL2"

Actual Study Start Date :

Nov 30, 2022

Anticipated Primary Completion Date :

Nov 30, 2028

Anticipated Study Completion Date :

Nov 30, 2028

Outcome Measures

Primary Outcome Measures

/ Lipids profiles at steady state, in sickle cell anemia and SC sickle cell adult patients, classified according to occurrence of complications. [6 years]
Cohorts of sickle cell disease patients include sickle cell anemia (SCA) and SC sickle cell patients living in Guadeloupe and Martinique and followed by the Sickle cell disease (SCD) Reference and Competence Centers of French West Indies. Lipid profile includes total cholesterol, HDL-cholesterol, non-HDL-cholesterol, LDL-cholesterol and triglycerides, apolipoproteins A-I and B. Collection of medical histories and of prospective SCD complications include retinopathy, deafness, tinnitus, osteonecrosis, leg ulcers, strokes, acute chest syndrome, VOC, priapism, pulmonary arterial hypertension (PAH) and PAH sd (echocardiography diagnosed when tricuspid regurgitant jet velocity ≥2.5 m/sec), kidney disease: chronic renal insufficiency and/or nephropathy

Secondary Outcome Measures

Kinetic study of lipids profile during hospitalized vasoocclusive crisis (VOC, with or without ACS) and Priapism, at return to steady state at first annual check-up, and one year after this last measurement [6 years]
Past and prospective collection of previously listed SCD complications
Study of variation of lipid profile, at steady state, during a 4 years period study intra and inter individual levels. [6 years]
total cholesterol
Study of variation of lipid profile, at steady state, during a 4 years period study intra and inter individual levels. [6 years]
HDL-cholesterol
Study of variation of lipid profile, at steady state, during a 4 years period study intra and inter individual levels. [6 years]
non HDL-cholesterol
Study of variation of lipid profile, at steady state, during a 4 years period study intra and inter individual levels. [6 years]
LDL-cholesterol
Study of variation of lipid profile, at steady state, during a 4 years period study intra and inter individual levels. [6 years]
triglycerides
Study of variation of lipid profile, at steady state, during a 4 years period study intra and inter individual levels. [6 years]
Apolipoproteins A-I and B
Description of genetic primary modulators of SCD complications. [6 years]
Fetal hemoglobin,
Description of genetic primary modulators of SCD complications. [6 years]
alpha-thalassemia,
Description of genetic primary modulators of SCD complications. [6 years]
haplotypes of beta S gene
Dosages of Insulin resistance (HOMA), [6 years]
Plasmatic insulinemia and glycemia (HOMA) will be performed in the entire cohort at inclusion and during prospective complications (VOC, priapism); lipids dosages
free fatty acids [6 years]
kinetic study of free fatty acids at inclusion and during prospective complications (VOC, priapism);
plasmatic glycerol. [6 years]
Kinetic study of plasmatic at inclusion and during prospective complications (VOC, priapism);
Dosages of lipids enzymes, lipidome and functionality of HDL at steady state [6 years]
The dosages of CETP (Cholesteryl Ester Transfer Protein) enzymes activities
Dosages of lipids enzymes, lipidome and functionality of HDL at steady state [6 years]
The dosages of L-CAT (Lécithine Cholestérol Acyl Transférase) enzymes activities
Dosages of lipids enzymes, lipidome and functionality of HDL at steady state [6 years]
The dosages of HDL lipidome,
Dosages of lipids enzymes, lipidome and functionality of HDL at steady state [6 years]
The dosages of HDL functionality,
Dosages of lipids enzymes, lipidome and functionality of HDL at steady state [6 years]
The dosages of free fatty acid
Dosages of lipids enzymes, lipidome and functionality of HDL at steady state [6 years]
The dosages of glycerol

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Aged from 18 years and over
Be affected with Sickle cell anemia or SC sickle cell
Living in French Caribbean Islands of Guadeloupe or Martinique and followed by physicians issued from a French West Indies Sickle Cell Reference or Competence Center
At steady state in the last month (without acute complication)
To have given a written consent after information on the study.

Exclusion Criteria:

Other hemoglobinopathies than sickle cell disease
Pregnancy or lactation
Patient under judicial protection or without freedom
Patient not affiliated with a social security system
Patient hospitalized for transfusion or bleeding in the last 3 months

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Unité Transversale de la Drépanocytose	Pointe-à-Pitre	Guadeloupe	France	97159
2	Centre de Référence de la Drépanocytose	Le Lamentin	Martinique	France	97292