major sickle cell syndrome MedDRA version: 21.0 Level: PT Classification code 10040644 Term: Sickle cell disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: FAMOTIDINE 40mg/5mL, powder for oral suspension
Product Name: FAMOTIDINE
Product Code: NDC 68180-150-01
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: FAMOTIDINE
Oth
Sponsors
Assistance Publique des Hôpitaux de Paris(AP-HP)
Eligibility
Sex/Gender
All
Inclusion criteria
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
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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the effect of famotidine on P-selectin expression after 29 days of treatment.;Secondary Objective: - To assess the effect of famotidine on the expression of other endothelial activation markers after 29 days of treatment; - To assess the effect of famotidine on the biomarkers of hemolysis and inflammation after 29 days of treatment; - To assess the adverse effects of famotidine in a pediatric population suffering from sickle cell disease;Primary end point(s): The primary endpoint will be the difference in the plasma concentration of soluble P-selectin measured by ELISA technique (Human P-selectin / CD62P Quantikine ELISA kit, R&D) before and after 29 days of treatment with famotidine.;Timepoint(s) of evaluation of this end point: Follow-up visit / end of treatment: D29 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints will be: - the differences in plasma concentration of soluble adhesion molecules E-selectin, VCAM-1, and ICAM-1 measured by ELISA technique (Human E-selectin / CD62E, Human sVCAM-1 / CD106 and Human ICAM-1 / CD54 Quantikine ELISA kits, R&D) before and after 29 days of treatment with famotidine. - The differences in blood values ??of hemoglobin, reticulocytes, ASAT, free bilirubin, LDH, and CRP (measured in the hematology / hemostasis and biochemistry laboratories of the Necker-Enfants Malades hospital) before and after 29 days of treatment by famotidine. - Occurrence of serious or non-serious adverse event (s) ;Timepoint(s) of evaluation of this end point: - Inclusion visit: D0 - Follow-up visit / end of treatment: D29 - Phone call / end of research: D36 | — |
Countries
France
Contacts
Public ContactDRCI, Hôpital St Louis,
Assistance Publique des Hôpitaux de Paris(AP-HP)
Outcome results
None listed