Interest of famotidine in reducing endothelial expression of P-selectin in children with sickle cell disease: pilot study, single-center, prospective, non-comparative

Phase 1

• Conditions: major sickle cell syndrome; MedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2021-001351-13-FR
• Lead Sponsor: Assistance Publique des Hôpitaux de Paris(AP-HP)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-09-09
• Last Update Posted: 29 November 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

- child or adolescent aged from 1 year to at most 17 years and 10 months, followed at the Necker-Enfants Malades Hospital for a major sickle cell syndrome SS or Sß0;
- having at least one vaso-occlusive crisis in the year prior to inclusion;
- signed informed consent of the 2 parents or legal representative (s), and, oral and if possible signed consent of the child of expressive age or the adolescent;
- beneficiary of social security coverage or entitled (excluding SMA)
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 (ALAT = 3N)
- neutropenia (<1 G / L), thrombocytopenia (<80 G / L), reticulopenia (<80 G / L);
- predictable poor adherence to treatment;
- participation in another interventional research involving the human person.
- bone marrow transplant or gene therapy project within one month of inclusion

Within 3 months prior to inclusion:
- erythrocyte transfusion;
- introduction of hydroxyurea or modification of the doses of hydroxyurea;
- introduction of L-glutamine or modification of the doses of L-glutamine;
- 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 = 1g / dL compared to baseline hemoglobin, increase in LDH and / or AST and / or free bilirubin deemed significant by the child's referring physician).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified