FAMODREP : Interest of Famotidine in Reducing Endothelial Expression of P-selectin in Children With Sickle Cell Disease: Pilot Study, Single-center, Prospective, Non-comparative
- Conditions
- Sickle Cell DiseaseTherapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2022-502144-12-00
- Lead Sponsor
- Assistance Publique Hopitaux De Paris
- Brief Summary
2022-502144-12-00_RESULTATS-EC_20231122_FAMODREP
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 30
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 XML File Identifier: DB4LUVFyBpLmaU7F8LN/rGiD0kI= Page 10/21 sickle cell syndrome SS or Sß0, having at least one vaso-occlusive crisis in the year prior to inclusion, for young girls of childbearing age (= 15 years): a negative pregnancy test, 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)
treatment with crizanlizumab (anti-P-selectin antibody), participation in another interventional research involving the human person, bone marrow transplant or gene therapy project within one month of inclusion, 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, pregnancy or breastfeeding
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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, To assess CVO occurrence;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.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):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.;Secondary end point(s):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.;Secondary end point(s):Occurrence of serious or non-serious adverse event (s);Secondary end point(s):CVO occurrence