Efficacy of Nintedanib per os as a treatment for epistaxis in HHT diseaseEPICURE

Phase 1

• Conditions: Hereditary Hemorrhagic Telangiectasia; MedDRA version: 20.1Level: LLTClassification code 10019887Term: Hereditary hemorrhagic telangiectasiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-002593-31-FR
• Lead Sponsor: Hospices Civils de Lyon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-05
• Last Update Posted: 16 December 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

?Age > 18 years old
?Patients who have given their free informed and signed consent
?Patients affiliated to a social security scheme or similar
?Patients monitored for clinically confirmed HHT and/or with molecular biology confirmation
?Patient with an Epistaxis Severity Score (ESS) > 4 (target population of patients with important consequences on quality of life)

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

•Pregnant woman or woman of child bearing potential not using two effective methods of birth control (one barrier and one highly effective non-barrier) for at least 1 month prior to trial and/or committing to using it until 3 months after the end of treatment.
•Woman who are breast feeding.
•Patient who are protected adults under the terms of the law (French Public Health Code).
•Participation in another interventional clinical trial which may interfere with the proposed trial (judgment of the investigator).
•Clinical evidence of active infection.
•(AST, ALT > 1,5 fold upper limit of normal (ULN) and/or Bilirubin > 1,5 fold upper limit of normal (ULN).
•Severe renal impairment (Creat Clearance <30 mL/min) estimated by the Cockcroft-Gault equation.
•Presence of non-treated pulmonary arteriovenous malformations accessible to a treatment on CT scan within 5 years.
•Patients with hemoptysis or hematuria within 12 weeks prior to inclusion.
•Patients with active gastro-intestinal (GI) bleeding or GI ulcers.
•Presence of cerebral arteriovenous malformation on MRI done within 5 years prior inclusion.
•Patients who require full-dose therapeutic anticoagulation (e.g. vitamin K antagonist or heparin, dabigatran) or high dose antiplatelet therapy.
•Patients with known coronary artery disease or recent history of myocardial infarction (within 1 year).
•Known inherited predisposition to thrombosis or thrombotic events (including stroke and transient ischemic attack) within 12 months prior to inclusion.
•Patients with QTc prolongation (on ECG, less than 3 months).
•Hypersensitivity to nintedanib, peanut or soya, or to any of the excipients.
•Patient who incompletely filled in epistaxis grids within 8 weeks prior to inclusion.
•Patient who have received intravenous bevacizumab within 6 months prior to inclusion.
•Patient who had surgery (including ENT surgery) within 12 weeks prior to inclusion.
•Unhealed wound.
•Planned major surgery within the next 3 months, including liver transplantation, major abdominal or intestinal surgery.

Study & Design

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