Interest of Famotidine in Children With Sickle Cell Disease

NCT05084521 | Completed Phase 2 FDA Drug

• 2 other identifiers: APHP2011332021-001351-13
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether oral famotidine, a histamine type 2 receptor antagonist already widely used with very few side effects in other indications in children, is effective in reducing endothelial expression of P-selectin in children with sickle cell disease (SCD). This pilot study will constitute the essential prerequisite for a randomized clinical trial comparing the efficacy of famotidine with that of placebo in the prevention of vaso-occlusive crises in SCD patients.

Conditions

Sickle Cell Disease

Keywords

Sickle cell disease; Famotidine; Histamine; P-selectin; Vaso-occlusive crisis

Outcome Measures

Primary Outcomes (1)

• Difference in plasma concentration of soluble P-selectin

  Measurement by ELISA technique before and after 29 days of treatment

  29 days

Secondary Outcomes (6)

• Difference in plasma concentration of soluble adhesion molecule: E-selectin

  29 days

• Difference in plasma concentration of soluble adhesion molecule: VCAM-1

  29 days

• Difference in plasma concentration of soluble adhesion molecule: ICAM-1

  29 days

• Differences in blood values: hemoglobin, reticulocytes, AST, free bilirubin, LDH, and CRP

  29 days

• Occurrence of serious or non-serious adverse event(s)

  36 days

Study Arms (1)

Famotidine

EXPERIMENTAL

Suspension of famotidine, 0.5 mg/kg/12h (with a maximum dose of 80 mg/day, regardless of the patient's weight) during 29 days.

Drug: Famotidine 400 mg/50 mL

Interventions

Famotidine 400 mg/50 mL DRUG

Administration of a suspension of famotidine, 0.5 mg/kg/12h (with a maximum dose of 80 mg/day, regardless of the patient's weight) during 29 days.

Famotidine

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• child or adolescent aged 1 year to 17 years and 10 months, followed at the Necker-Enfants malades Hospital for a SS or Sβ0 SCD;
• for young girl of childbearing age (≥ 15 years old), a negative pregnancy test;
• signed informed consent of the 2 parents or legal representative(s) and of the child of expressive age or the adolescent;
• beneficiary of social security coverage or entitled (excluding AME)

You may not qualify if:

• treatment with crizanlizumab (anti-P-selectin antibody);
• treatment with atazanavir/ritonavir in combination with tenofovir;
• known hypersensitivity to famotidine or to other histamine type 2 (H2) receptor antagonists;
• cardiovascular history such as: arrhythmia, AVB (atrioventricular block), QT prolongation;
• renal failure characterized by creatinine clearance \<60 mL/min;
• hepatic cytolysis (ALT ≥ 3N);
• neutropenia (\<1 G/L), thrombocytopenia (\<80 G/L), reticulopenia (\<80 G/L);
• predictable poor adherence to treatment;
• pregnancy or breastfeeding;
• participation in another interventional research involving the human person;
• red blood cell transfusion;
• introduction of hydroxyurea or modification of hydroxyurea doses;
• introduction of L-glutamine or modification of L-glutamine doses;
• introduction of voxelotor or modification of voxelotor doses;
• taking oral or IV corticosteroids or any other immunomodulatory treatment;

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• INSERM U 1163 - Laboratory of molecular mechanisms of hematologic disorders and therapeutic implications, Paris: collaborator
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Necker - Enfants malades Hospital; Department of Pediatrics and Infectious Diseases

Paris, 75015, France

Location

Related Publications (1)

• Allali S, Marquant F, Rignault-Bricard R, Taylor M, Brice J, de Montalembert M, Maciel TT, Elie C, Hermine O. Oral famotidine reduces the plasma level of soluble P-selectin in children with sickle cell disease. Br J Haematol. 2024 Jan;204(1):346-351. doi: 10.1111/bjh.19111. Epub 2023 Sep 18.

  PMID: 37722599 RESULT

MeSH Terms

Conditions

Anemia, Sickle Cell; Vaso-Occlusive Crises

Interventions

Famotidine

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)

Thiazoles; Sulfur Compounds; Organic Chemicals; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Slimane ALLALI, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

• Olivier HERMINE, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: October 15, 2021
• First Posted: October 19, 2021
• Study Start: January 12, 2022
• Primary Completion: December 12, 2022
• Study Completion: December 12, 2022
• Last Updated: October 8, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)