Sickle Cell DiseasePhase 2KID-BIDNCT06578507
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
A Phase 2 study testing Hydroxycarbamid in people with Sickle Cell Disease, run by Theravia at 7 sites in 2 countries.
Trial summary
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of… Read more about this study
- Who is it for?
- Ages 9 months to 11 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Hydroxycarbamid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 21 Jan 2025to Feb 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 9 months to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, and, if possible, assent from the children,
- HbSS or HbSβ0 SCD,
- Aged between 9 months and 11 years old,
- Hydroxycarbamide naïve,
- Parent(s) or legally acceptable representative(s) capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements,
- Contraception criterion, if applicable: for patients who are sexually active
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- Affiliated to a social security plan or beneficiary of a similar insurance plan,
- Patient must meet the following laboratory values : Absolute Neutrophil Count ≥ 1.0x109/L, Platelets ≥ 75x109/L and Haemoglobin (Hgb) > 5.5 g/dL,
- Transcranial Doppler (TCD) in the last 12 months indicating low risk for stroke is required for children over 18 months of age.
Who cannot join
- Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding the inclusion visit,
- Patients who have had chronic blood transfusion or transfusion in the last 3 months preceding the inclusion visit,
- Patients treated with other SCD-modifying therapies,
- Patient with a stage 3, 4 or 5 chronic kidney disease,
- Patients known to be infected with human immunodeficiency virus, hepatitis B virus, or hepatitis C virus,
- Known hypersensitivity or allergy to the excipients,
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- Any surgical or medical condition or any significant illness that, in the opinion of the investigator, constitutes a risk or a contraindication to the participation of the patient to the study, or that may interfere with the objectives, conduct or evaluation of the study,
- Female patients who are pregnant or lactating,
- Any documented history of a clinical stroke or intracranial haemorrhage, or an uninvestigated neurologic finding within the past 12 months.
Trial locations and contacts
7 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
Nesrine Flissi contact
7 sites in 2 countries
France6 sites, 5 recruiting
- Recruiting
Centre Hospitalier Intercommunal Créteil
Créteil
Cecile Arnaud, MD
Cecile Arnaud, MD
- Recruiting
GHEF- Site de Marne-la-Vallée
Jossigny
Kokou AGBO KPATI, kagbokpati@ghef.fr
- Recruiting
Hôpital Bicêtre
Le Kremlin-Bicêtre
Corinne Guitton, MD
Corinne Guitton, MD
- Suspended
Institut d'Hématologie et d'oncologie pédiatrique - IHOPe
Lyon
- Recruiting
Hôpital Necker-Enfants malades
Paris
Josephine Brice, MD
Josephine Brice, MD
- Recruiting
CH Saint-Denis
Saint-Denis
Aurore Malric, aurore.malric@ch-stdenis.fr
Aurore Malric
French Guiana1 site, 1 recruiting
- Recruiting
Centre hospitalier de Cayenne
Cayenne
Narcisse Elenga, MD, PhD
Narcisse Elenga, MD, PhD
About this study
Official title: Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease: a Single-group, Non-randomised, Open-label Study (KID-BID)
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age.
Participants will:
- Take Hydroxycarbamide twice a day every day for 12 months
- Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months
- Conditions
- Sickle Cell Disease
- Being tested
- Hydroxycarbamid
- Keywords
- Hydroxycarbamide, Pharmacokinetic
- 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.
HydroxycarbamidDrug
Also called Hydroxyurea
Hydroxycarbamide Paediatric dispersible tablets will be provided in the form of film-coated dispersible tablets containing 50 mg of hydroxycarbamide.
The IMP will be administered as half-strength twice daily, based on the body weight of the patient.
| Study group | Role | What they receive |
|---|---|---|
| Hydroxycarbamide Pediatric dispersible tablet | Experimental | Hydroxycarbamide Pediatric dispersible tablet will be administered twice daily during 12 months. Receives: Drug: Hydroxycarbamid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC)
Measured: 1, 3, 6, 9 and 12 months after treatment initiation
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through time to obtain the maximum concentration (Tmax)
Measured: 1, 3, 6, 9 and 12 months after treatment initiation
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through maximum plasma concentration (Cmax)
Measured: 1, 3, 6, 9 and 12 months after treatment initiation
Secondary outcomes (11)
Absolute mean change from baseline in HbF levels
Measured: Baseline, 3, 6, 9 and 12 months after treatment initiation
HC plasma concentrations and HbF levels
Measured: Baseline, 3, 6, 9 and 12 months after treatment initiation
Daily AUC (AUC0-24h) at maintenance dose derived from the final PPK model
Measured: Baseline, 3, 6, 9 and 12 months after treatment initiation
Proportion of patients with a relative difference in Cmax ≥ 30% from BID maintenance dose relative to the one simulated on a once daily regimen giving an equivalent AUC0-24h.
Measured: Baseline, 3, 6, 9 and 12 months after treatment initiation
Absolute mean change from baseline in haematological parameters
Measured: Baseline,1, 3, 6, 9 and 12 months after treatment initiation
Acceptability score based on a hedonic face scale evaluated by the child from 3 years old
Measured: 3 months after treatment initiation
Acceptability score based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s)
Measured: 3 months after treatment initiation
Distribution of the scores related to the ease of using the administration kit for treatment administration to the child based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s)
Score 1 : very difficult to score 5 : very easy
Measured: 3 months after treatment initiation
Compliance with Hydroxycarbamide Paediatric dispersible tablets administered BID by treatment unit accountability calculated by the pharmacy (patient will bring the kits with used and unused bottles to the pharmacy at each visit)
Measured: Baseline, 1, 3, 6, 9, 12 months after treatment initiation
Number of SCD events occurring during the study
Measured: Baseline, 1, 3, 6, 9, 12 months after treatment initiation
Number of adverse events (AEs) and percentage of patients reporting at least one AE
Measured: Baseline, 1, 3, 6, 9, 12 months after treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2024
- Study start21 Jan 2025
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2028 (estimated)
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Theravia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Josephine Brice, MD, Principal investigator, Hôpital Necker-Enfants Malades
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06578507
- Sponsor's protocol ID
- SIK-FR-24-1
- Acronym
- KID-BID
- Status last verified
- Sep 2026
- First submitted
- 22 Aug 2024
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease: a Single-group, Non-randomised, Open-label Study (KID-BID)
- ClinicalTrials.gov ID
- NCT06578507
- Acronym / sponsor ID
- KID-BID / SIK-FR-24-1
- Conditions
- Sickle Cell Disease
- Keywords
- Hydroxycarbamide, Pharmacokinetic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- TheraviaIndustry (pharma, biotech, devices)
- Study start
- 21 Jan 2025 (actual)
- Primary completion
- Feb 2028 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 29 Aug 2024 (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 purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age.
Participants will:
- Take Hydroxycarbamide twice a day every day for 12 months
- Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hydroxycarbamide Pediatric dispersible tablet | Experimental | Drug: HydroxycarbamidHydroxycarbamide Pediatric dispersible tablet will be administered twice daily during 12 months. |
Who may qualify
- Ages
- 9 months to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, and, if possible, assent from the children,
- HbSS or HbSβ0 SCD,
- Aged between 9 months and 11 years old,
- Hydroxycarbamide naïve,
- Parent(s) or legally acceptable representative(s) capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements,
Key exclusion criteria (9 listed)
- Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding the inclusion visit,
- Patients who have had chronic blood transfusion or transfusion in the last 3 months preceding the inclusion visit,
- Patients treated with other SCD-modifying therapies,
- Patient with a stage 3, 4 or 5 chronic kidney disease,
- Patients known to be infected with human immunodeficiency virus, hepatitis B virus, or hepatitis C virus,
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 |
|---|---|---|
| HydroxycarbamidAlso: HydroxyureaHydroxycarbamide Paediatric dispersible tablets will be provided in the form of film-coated dispersible tablets containing 50 mg of hydroxycarbamide. The IMP will be administered as half-strength twice daily, based on the body weight of the patient. | Drug | Hydroxycarbamide Pediatric dispersible tablet |
Primary outcome
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC)
Time frame: 1, 3, 6, 9 and 12 months after treatment initiation
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through time to obtain the maximum concentration (Tmax)
Time frame: 1, 3, 6, 9 and 12 months after treatment initiation
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through maximum plasma concentration (Cmax)
Time frame: 1, 3, 6, 9 and 12 months after treatment initiation
11 secondary outcomes
Absolute mean change from baseline in HbF levels
Time frame: Baseline, 3, 6, 9 and 12 months after treatment initiation
HC plasma concentrations and HbF levels
Time frame: Baseline, 3, 6, 9 and 12 months after treatment initiation
Daily AUC (AUC0-24h) at maintenance dose derived from the final PPK model
Time frame: Baseline, 3, 6, 9 and 12 months after treatment initiation
Proportion of patients with a relative difference in Cmax ≥ 30% from BID maintenance dose relative to the one simulated on a once daily regimen giving an equivalent AUC0-24h.
Time frame: Baseline, 3, 6, 9 and 12 months after treatment initiation
Absolute mean change from baseline in haematological parameters
Time frame: Baseline,1, 3, 6, 9 and 12 months after treatment initiation
Acceptability score based on a hedonic face scale evaluated by the child from 3 years old
Time frame: 3 months after treatment initiation
Acceptability score based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s)
Time frame: 3 months after treatment initiation
Distribution of the scores related to the ease of using the administration kit for treatment administration to the child based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s)
Time frame: 3 months after treatment initiation
Compliance with Hydroxycarbamide Paediatric dispersible tablets administered BID by treatment unit accountability calculated by the pharmacy (patient will bring the kits with used and unused bottles to the pharmacy at each visit)
Time frame: Baseline, 1, 3, 6, 9, 12 months after treatment initiation
Number of SCD events occurring during the study
Time frame: Baseline, 1, 3, 6, 9, 12 months after treatment initiation
Number of adverse events (AEs) and percentage of patients reporting at least one AE
Time frame: Baseline, 1, 3, 6, 9, 12 months after treatment initiation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- 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.
Nesrine Flissi contact
Josephine Brice, MD principal investigator
Hôpital Necker-Enfants Malades
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxycarbamid in people with Sickle Cell Disease, run by Theravia at 7 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 9 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in France, French Guiana. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Laura Thomas-bourgneuf (+ 33 1 49 70 95 83, laura.thomas-bourgneuf@theravia.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC) (time frame: 1, 3, 6, 9 and 12 months after treatment initiation). The study lists 3 primary outcomes in all. 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 1 Oct 2026.