Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa

NCT05681598 | Completed

• 1 other identifier: ZRDC2015PR088
• Study Type: interventional
• Target: 166
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to evaluate the efficacy of hydroxyurea (HU) in improving disease severity in adult patients with sickle cell anemia in Kinshasa (Democratic Republic of Congo). This study aims to:

• assess the safety and efficacy of HU treatment in the Congolese environment;
• assess the reversibility of chronic cardiac lesions. Participants will take hydroxyurea for two years. The effects of the treatment will be evaluated periodically by clinical evaluation, biological tests, and echocardiographic exploration.

Conditions

Sickle-Cell Anaemia

Keywords

sickle cell anemia; hydroxyurea; adult patients; kinshasa

Outcome Measures

Primary Outcomes (11)

• changes from baseline in body mass index (BMI) to year 2

  weight in kilograms and height in meters will be combined to report BMI in kg/m\^2. The measurements obtained after 1 year and after 2 years of treatment will be compared with the baseline.

  baseline, year 1 and year 2

• changes from baseline in the frequency of Acute clinical events to year 2

  The number of vaso-occlusive crises and the number of transfusions and the number of days of hospitalization will be recorded each month and compiled to provide an annual assessment of the severity of the disease. The overall assessment will be 24 months.

  baseline, year 1 and year 2

• changes from baseline in complete blood count to year 2

  A complete blood count will be assessed every 3 months up to 24 months of HU treatment. The values measured during the treatment will be compared to the basal values.

  baseline, 3, 6, 9, 12, 15, 18, 21 and 24 months

• changes from baseline in Fetal hemoglobin to year 2

  The fetal hemoglobin rate will be measured every 3 months up to 24 months of HU treatment and its values compared to its initial value.

  baseline, 3, 6, 9, 12, 15, 18, 21 and 24 months

• changes from baseline in left ventricle dilation to year 1

  Left ventricular internal diameter in diastole (LVID), mesured by cardiac ultrasound, indexed to body surface area (cm/m²) will be measured at baseline and compared with measurements taken every 4 months, over a total period of 12 months of HU treatment.

  baseline, 4, 8 and 12 months

• changes from baseline in the thickness of left ventricular walls to year 1

  The thickness of the walls of the left ventricle (interventricular septum and posterior wall of the left ventricle) expressed in "cm" were measured on echocardiography. The measurements taken every 4 months were compared to the basal measurement. The overall assessment will cover 12 months of treatment.

  baseline, 4, 8 and 12 months

• changes from baseline in the tricuspid regurgitation jet velocity to year 1

  The velocity of the tricuspid regurgitation jet will be evaluated by cardiac ultrasound with continuous Doppler. A velocity greater than or equal to 2.5m/s indicates pulmonary hypertension. The measurement will be made every four months, for a total period of 12 months.

  baseline, 4, 8 and 12 months

• changes from baseline in the pulmonary acceleration time (PaccT) to year 1

  In cardiac ultrasound short -axis view, with the pulsed-wave Doppler placed at the center of the trans-pulmonary valve jet, the PaccT is the time interval between the onset of ejection and the peak flow velocity (in milliseconds). Pulmonary hypertension will be diagnosed when PaccT was less than 90 milliseconds. The measurement will be made every four months, for a total period of 12 months.

  baseline, 4, 8 and 12 months

• Changes from baseline in E and A mitral to year 1

  The transmitral Doppler velocities will be studied, with the pulsed-wave Doppler sample volume placed at the tips of the mitral valve leaflets from the apical four chambers view. Diastolic peaks velocity early (E) and later (A) will be recorded in cm/second. The measurements will be obtained at starting and every 4 months during a year of treatment.

  baseline, 4, 8 and 12 months

• Changes from baseline in E' mitral to year 1

  The pulsed-wave tissue Doppler imaging (TDI) will be used to provide ventricular wall motion velocity measurements by positioning the sample volume at the parietal side of the mitral valve annuli. The velocity of the early diastolic wave (E') will be measured in cm/s. Baseline value and values obtained every 4 months will be recorded.

  baseline, 4, 8 and 12 months

• Changes from baseline in the E/E' mitral ratio to year 1

  The calculated E/E' mitral ratio less than 8 indicates low filling pressures of the left ventricle. Baseline value and values obtained every 4 months during treatment will be compared.

  baseline, 4, 8 and 12 months

Study Arms (1)

Hydroxyurea treatment

EXPERIMENTAL

participants were treated with hydroxyurea

Drug: Hydroxyurea

Interventions

Hydroxyurea DRUG

The treatment started with a dose of 15mg/kg/day of HU. This initial dose was increased in steps of 5mg/kg/day every three months up to 35mg/kg/day or a maximal tolerated dose.

Hydroxyurea treatment

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• sickle cell anemia confirmed by DNA testing;
• moderate to severe clinical severity of sickle cell anemia;
• fetal hemoglobin lower than 15%.

You may not qualify if:

• poor compliance to follow-up consultation during the observational year
• participant already treated with HU
• pregnant women
• breastfeeding women
• congenital heart disease
• pulmonary disease

Sponsors & Collaborators

• University of Kinshasa: lead
• KU Leuven: collaborator

Study Sites (1)

University of Kinshasa

Kinshasa, Democratic Republic of the Congo

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Study Officials

• Prosper Lukusa Tshilobo

  Center of human genetics. Faculty of medicine. University of Kinshasa

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal investigator

Model Details: all eligible patients received during the study period were included and received treatment.

Study Record Dates

• First Submitted: October 6, 2022
• First Posted: January 12, 2023
• Study Start: August 30, 2017
• Primary Completion: May 16, 2020
• Study Completion: May 16, 2020
• Last Updated: January 12, 2023

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)