Sickle-Cell AnaemiaNCT05681598
Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa
A study testing Hydroxyurea in people with Sickle-Cell Anaemia, run by University of Kinshasa at 1 site in Democratic Republic of the Congo.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Sickle-Cell Anaemia
- What is being tested?
- Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 166 participants
- Where
- 1 siteDemocratic Republic of the Congo
- Study dates
- 30 Aug 2017to 16 May 2020
- 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 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- sickle cell anemia confirmed by DNA testing;
- moderate to severe clinical severity of sickle cell anemia;
- fetal hemoglobin lower than 15%.
Who cannot join
- poor compliance to follow-up consultation during the observational year
- participant already treated with HU
- pregnant women
- breastfeeding women
- congenital heart disease
- pulmonary disease
Trial locations and contacts
1 site in 1 country.
Democratic Republic of the Congo1 site
University of Kinshasa
Kinshasa
About this study
Official title: Diagnosis and Treatment With Hydroxyurea of Sickle Cell Anemia in Democratic Republic of the Congo
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.
Read the detailed description
Sickle cell disease is common in sub-Saharan Africa, particularly in the Democratic Republic of the Congo (DRC). It is characterized by chronic hemolytic anemia with the need for transfusions, painful osteoarticular crises, and chronic organ damage, including the heart.
Hydroxyurea (HU) is a drug widely used in sickle cell patients in wealthy countries, while it is little used in poor countries with a high incidence of the disease.
This prospective study will focus on homozygous sickle cell participants, naïve to HU treatment. Participants will take HU in gradually increasing doses. They will be monitored for the possible occurrence of side effects. The effectiveness of the treatment will be evaluated according to the reduction in painful crises, and the need for blood transfusion; as well as based on biological changes and the reversibility or stabilization of cardiovascular complications.
- Conditions
- Sickle-Cell Anaemia
- Being tested
- Hydroxyurea
- Keywords
- sickle cell anemia, hydroxyurea, adult patients, kinshasa
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Hydroxyurea treatment | Experimental | participants were treated with hydroxyurea Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: baseline, 4, 8 and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2023
- Study start30 Aug 2017
- Primary completion16 May 2020
- Study completion16 May 2020
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 Sickle-Cell AnaemiaStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- all eligible patients received during the study period were included and received treatment.
- Masking
- None (open label)
- Enrolment
- 166 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kinshasa
- Sponsor type
- University, hospital or other
- Collaborators
- KU Leuven
- Responsible party
- Principal investigator, Paul Lumbala Kabuyi, University of Kinshasa
- Investigators
- Prosper Lukusa Tshilobo, Study director, Center of human genetics. Faculty of medicine. University of Kinshasa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05681598
- Sponsor's protocol ID
- ZRDC2015PR088
- Status last verified
- Dec 2022
- First submitted
- 6 Oct 2022
- Last updated
- 12 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Mbayabo G, Kabuyi PL, Ngole M, Lumaka A, Race V, Matthijs G, Minga TM, Devriendt K, Van Geet C, Lukusa PT. Evaluation of The Use of Hydroxyurea in Treating Children With Sickle Cell Anemia in Central Africa's Rural Area. EJHaem. 2026 Aug 10;7(4):e70378. doi: 10.1002/jha2.70378. eCollection 2026 Aug.
PubMed 42577886
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Diagnosis and Treatment With Hydroxyurea of Sickle Cell Anemia in Democratic Republic of the Congo
- ClinicalTrials.gov ID
- NCT05681598
- Acronym / sponsor ID
- ZRDC2015PR088
- Conditions
- Sickle-Cell Anaemia
- Keywords
- sickle cell anemia, hydroxyurea, adult patients, kinshasa
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 166 participants (actual)
- Lead sponsor
- University of KinshasaUniversity, hospital or other
- Collaborators
- KU Leuven
- Study start
- 30 Aug 2017 (actual)
- Primary completion
- 16 May 2020 (actual)
- Study completion
- 16 May 2020 (actual)
- First posted
- 12 Jan 2023 (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.
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.
Detailed description
Sickle cell disease is common in sub-Saharan Africa, particularly in the Democratic Republic of the Congo (DRC). It is characterized by chronic hemolytic anemia with the need for transfusions, painful osteoarticular crises, and chronic organ damage, including the heart.
Hydroxyurea (HU) is a drug widely used in sickle cell patients in wealthy countries, while it is little used in poor countries with a high incidence of the disease.
This prospective study will focus on homozygous sickle cell participants, naïve to HU treatment. Participants will take HU in gradually increasing doses. They will be monitored for the possible occurrence of side effects. The effectiveness of the treatment will be evaluated according to the reduction in painful crises, and the need for blood transfusion; as well as based on biological changes and the reversibility or stabilization of cardiovascular complications.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- all eligible patients received during the study period were included and received treatment.
| Arm | Type | Receives |
|---|---|---|
| Hydroxyurea treatment | Experimental | Drug: Hydroxyureaparticipants were treated with hydroxyurea |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- sickle cell anemia confirmed by DNA testing;
- moderate to severe clinical severity of sickle cell anemia;
- fetal hemoglobin lower than 15%.
Key exclusion criteria (6 listed)
- poor compliance to follow-up consultation during the observational year
- participant already treated with HU
- pregnant women
- breastfeeding women
- congenital heart disease
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 |
|---|---|---|
| HydroxyureaThe 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. | Drug | Hydroxyurea treatment |
Primary outcome
changes from baseline in body mass index (BMI) to year 2
Time frame: baseline, year 1 and 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.
changes from baseline in the frequency of Acute clinical events to year 2
Time frame: baseline, year 1 and 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.
changes from baseline in complete blood count to year 2
Time frame: baseline, 3, 6, 9, 12, 15, 18, 21 and 24 months
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.
changes from baseline in Fetal hemoglobin to year 2
Time frame: baseline, 3, 6, 9, 12, 15, 18, 21 and 24 months
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.
changes from baseline in left ventricle dilation to year 1
Time frame: baseline, 4, 8 and 12 months
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.
changes from baseline in the thickness of left ventricular walls to year 1
Time frame: baseline, 4, 8 and 12 months
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.
changes from baseline in the tricuspid regurgitation jet velocity to year 1
Time frame: baseline, 4, 8 and 12 months
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.
changes from baseline in the pulmonary acceleration time (PaccT) to year 1
Time frame: baseline, 4, 8 and 12 months
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.
Changes from baseline in E and A mitral to year 1
Time frame: baseline, 4, 8 and 12 months
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.
Changes from baseline in E' mitral to year 1
Time frame: baseline, 4, 8 and 12 months
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.
Changes from baseline in the E/E' mitral ratio to year 1
Time frame: baseline, 4, 8 and 12 months
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.
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 12 Jan 2023.
- Derived from this study: PubMed 42577886Mbayabo G, Kabuyi PL, Ngole M, Lumaka A, Race V, Matthijs G, Minga TM, Devriendt K, Van Geet C, Lukusa PT. Evaluation of The Use of Hydroxyurea in Treating Children With Sickle Cell Anemia in Central Africa's Rural Area. EJHaem. 2026 Aug 10;7(4):e70378. doi: 10.1002/jha2.70378. eCollection 2026 Aug.
- 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.
Prosper Lukusa Tshilobo study director
Center of human genetics. Faculty of medicine. University of Kinshasa
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hydroxyurea in people with Sickle-Cell Anaemia, run by University of Kinshasa at 1 site in Democratic Republic of the Congo.
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 3 inclusion and 6 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 12 Jan 2023. 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 Democratic Republic of the Congo. 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?
changes from baseline in body mass index (BMI) to year 2 (time frame: baseline, year 1 and year 2). The study lists 11 primary outcomes in all. 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 NCT05681598, 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 12 Jan 2023.