Interest of Famotidine in Children With Sickle Cell Disease (NCT05084521) | Clinical Trial Compass
CompletedPhase 2/3
Interest of Famotidine in Children With Sickle Cell Disease
France30 participantsStarted 2022-01-12
Plain-language 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.
Who can participate
Age range
1 Year – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Difference in plasma concentration of soluble P-selectin