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

NCT06578507 | Recruiting Phase 2

• 1 other identifier: SIK-FR-24-1
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 5

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

Keywords

Hydroxycarbamide; Pharmacokinetic

Outcome Measures

Primary Outcomes (3)

• Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC)

  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)

  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)

  1, 3, 6, 9 and 12 months after treatment initiation

Secondary Outcomes (11)

• Absolute mean change from baseline in HbF levels

  Baseline, 3, 6, 9 and 12 months after treatment initiation

• HC plasma concentrations and HbF levels

  Baseline, 3, 6, 9 and 12 months after treatment initiation

• Daily AUC (AUC0-24h) at maintenance dose derived from the final PPK model

  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.

  Baseline, 3, 6, 9 and 12 months after treatment initiation

• Absolute mean change from baseline in haematological parameters

  Baseline,1, 3, 6, 9 and 12 months after treatment initiation

Study Arms (1)

Hydroxycarbamide Pediatric dispersible tablet

EXPERIMENTAL

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

Drug: Hydroxycarbamid

Interventions

Hydroxycarbamid 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.

Also known as: Hydroxyurea

Hydroxycarbamide Pediatric dispersible tablet

Eligibility Criteria

• Age: 9 Months - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Theravia: lead

Study Sites (6)

Centre Hospitalier Intercommunal Créteil

Créteil, France

RECRUITING

GHEF- Site de Marne-la-Vallée

Jossigny, France

RECRUITING

Hôpital Bicêtre

Le Kremlin-Bicêtre, France

RECRUITING

Institut d'Hématologie et d'oncologie pédiatrique - IHOPe

Lyon, France

ACTIVE NOT RECRUITING

Hôpital Necker-Enfants malades

Paris, France

RECRUITING

Centre hospitalier de Cayenne

Cayenne, French Guiana

RECRUITING

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

• Josephine Brice, MD

  Hôpital Necker-Enfants Malades

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Laura Thomas-bourgneuf

CONTACT

+ 33 1 49 70 95 83; laura.thomas-bourgneuf@theravia.com

Nesrine Flissi

CONTACT

nesrine.flissi@theravia.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2024
• First Posted: August 29, 2024
• Study Start: January 21, 2025
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: September 17, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (5)