FAMODREP : Interest of Famotidine in Reducing Endothelial Expression of P-selectin in Children With Sickle Cell Disease: Pilot Study, Single-center, Prospective, Non-comparative

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

12 Jan 2022 → 12 Dec 2022

Decision date (initial)

2022-12-20

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2022-502144-12-00

EudraCT number

2021-001351-13

ClinicalTrials.gov

NCT05084521

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

To assess the effect of famotidine on P-selectin expression after 29 days of treatment.

Secondary objectives 4

1. To assess the effect of famotidine on the expression of other endothelial activation markers after 29 days of treatment
2. To assess the effect of famotidine on the biomarkers of hemolysis and inflammation after 29 days of treatment
3. To assess the adverse effects of famotidine in a pediatric population suffering from sickle cell disease
4. To assess CVO occurrence

Conditions and MedDRA coding

Sickle Cell Disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 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 Sβ0
2. having at least one vaso-occlusive crisis in the year prior to inclusion
3. for young girls of childbearing age (≥ 15 years): a negative pregnancy test
4. 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
5. beneficiary of social security coverage or entitled (excluding SMA)

Exclusion criteria 11

1. treatment with crizanlizumab (anti-P-selectin antibody)
2. treatment with atazanavir / ritonavir in combination with tenofovir
3. known hypersensitivity to famotidine or to other histamine type 2 (H2) receptor antagonists
4. cardiovascular history such as: arrhythmia, AVB (atrioventricular block), QT prolongation
5. Renal failure characterized by creatinine clearance <60 mL / min
6. hepatic cytolysis (ALAT ≥ 3N)
7. neutropenia (<1 G / L), thrombocytopenia (<80 G / L), reticulopenia (<80 G / L)
8. predictable poor adherence to treatment
9. pregnancy or breastfeeding
10. participation in another interventional research involving the human person
11. bone marrow transplant or gene therapy project within one month of inclusion

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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 endpoints 4

1. 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.
2. 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.
3. Occurrence of serious or non-serious adverse event (s)
4. CVO occurrence

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Slimane ALLALI

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Slimane ALLALI

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Documents 1 file

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications