Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

NCT ID: NCT06578507

Last Updated: 2025-09-17

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-01-21
• Study Completion Date: 2027-02-28

Brief 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 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Hydroxycarbamide Pediatric dispersible tablet

Hydroxycarbamide Pediatric dispersible tablet will be administered twice daily during 12 months.

Group Type: EXPERIMENTAL

Hydroxycarbamid

Intervention Type: DRUG

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.

Interventions

Hydroxycarbamid

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.

Intervention Type: DRUG

Other Intervention Names

Hydroxyurea

Eligibility Criteria

Inclusion Criteria

• 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
• 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.

Exclusion Criteria

• 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,
• 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.

• Minimum Eligible Age: 9 Months
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Theravia

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Josephine Brice, MD

Role: PRINCIPAL_INVESTIGATOR

Hôpital Necker-Enfants Malades

Locations

Centre Hospitalier Intercommunal Créteil

Créteil, , France

Site Status: RECRUITING

GHEF- Site de Marne-la-Vallée

Jossigny, , France

Site Status: RECRUITING

Hôpital Bicêtre

Le Kremlin-Bicêtre, , France

Site Status: RECRUITING

Institut d'Hématologie et d'oncologie pédiatrique - IHOPe

Lyon, , France

Site Status: ACTIVE_NOT_RECRUITING

Hôpital Necker-Enfants malades

Paris, , France

Site Status: RECRUITING

Centre hospitalier de Cayenne

Cayenne, , French Guiana

Site Status: RECRUITING

Countries

France; French Guiana

Central Contacts

Laura Thomas-bourgneuf

Role: CONTACT

laura.thomas-bourgneuf@theravia.com

+ 33 1 49 70 95 83

Nesrine Flissi

Role: CONTACT

nesrine.flissi@theravia.com

Facility Contacts

Cecile Arnaud, MD

Role: primary

Kokou AGBO KPATI

Role: primary

kagbokpati@ghef.fr

Corinne Guitton, MD

Role: primary

Josephine Brice, MD

Role: primary

Narcisse Elenga, MD, PhD

Role: primary

Other Identifiers

SIK-FR-24-1

Identifier Type: -

Identifier Source: org_study_id