Interest of Famotidine in Children With Sickle Cell Disease

• Status: Not yet recruiting
• Trial end date: 2023-07-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborator: INSERM U 1163 - Laboratory of molecular mechanisms of hematologic disorders and therapeutic implications, Paris
• Treatments: Famotidine

Criteria

Inclusion Criteria:

• 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;

• having at least one vaso-occlusive crisis in the year prior to inclusion;

• 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)

Exclusion Criteria:

• 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;

• planned bone marrow transplant or gene therapy within one month of inclusion.

Within 3 months prior to inclusion:

• 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;

• taking an antihistamine treatment

In the month preceding inclusion:

• occurrence of a vaso-occlusive crisis, acute chest syndrome or any vaso-occlusive phenomenon (acute splenic sequestration, priapism, stroke, occlusion of the central retinal artery, papillary necrosis);

• occurrence of fever (≥ 38°C) or any infectious episode, febrile or not, suspected or confirmed, of a viral, bacterial, fungal or parasitic nature ;

• occurrence of an acute hemolytic episode (increase in jaundice and pallor, decrease in hemoglobin level of ≥ 1 g/dL compared to baseline hemoglobin, increase in LDH and/or AST and/or free bilirubin deemed significant by the child's referring physician).