Nintedanib for Hereditary haemorrhagic telangiectasia

Inclusion criteria

• {"criterion_text":"- Signed informed consent"}
• {"criterion_text":"- Definite HHT disease (defined as the presence of a pathogenic mutation in one of the HHT genes, or the presence of 3 out of 4 Curaçao clinical criteria2 )"}
• {"criterion_text":"- Aged ≥18 years at the time of informed consent"}
• {"criterion_text":"- Moderate to serious epistaxis (Epistaxis Severity Score ESS≥2.5)"}
• {"criterion_text":"- Absence of cerebral arteriovenous malformation demonstrated by brain imaging"}

Exclusion criteria

• {"criterion_text":"- Women who are pregnant or breastfeeding because of the potential dangerous effect of the treatment on the fetus or infant"}
• {"criterion_text":"- Any other serious underlying medical condition that could interfere with study treatment and potential adverse events"}
• {"criterion_text":"- Any mental or other impairment that may compromise compliance with the study requirements."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Acute infection"}
• {"criterion_text":"- AST or ALT or ALKP or GGT or total bilirubin >1.5x (or >2.5x in patients known for Gilbert’s syndrome) the upper limit of normal"}
• {"criterion_text":"- Renal clearance by Cockcroft-Gault formula <30 ml/min"}
• {"criterion_text":"- Untreated pulmonary arteriovenous malformation"}
• {"criterion_text":"- Hemoptysis or hematuria within the last 12 months"}
• {"criterion_text":"- Ulcus or active gastric bleeding within the last 12 months"}
• {"criterion_text":"- Anticoagulant or antiplatelets treatment"}
• {"criterion_text":"- Coronary heart disease"}
• {"criterion_text":"- Participation in another interventional clinical trial which may interfere with the proposal trial (judgment of the investigator)"}
• {"criterion_text":"- Thrombotic event within the last 12 months"}
• {"criterion_text":"- Long QT syndrome (on ECG performed at screening)"}
• {"criterion_text":"- Known allergy to Nintenanib, soya, peanuts"}
• {"criterion_text":"- Bevacizumab, pazopanib or other anti-angiogenic treatments within the last 12 months"}
• {"criterion_text":"- Concomitant treatment with ketoconazole, erythromycin, rifampicin, carbamazepine, phenytoin, St John’s Wort"}
• {"criterion_text":"- Surgery within the last 3 months or planned within the next 9 months"}
• {"criterion_text":"- Recent unhealed wound"}

Primary endpoints

• {"endpoint_text":"- Proportion of patients with at least 30% reduction of monthly epistaxis duration after 16 weeks of study treatment (at V6, week 24) compared to baseline (V1, week 8) assessed in nintedanib arm and in placebo arm.","definition_or_measurement_approach":"Comparison of proportion of patients achieving ≥30% reduction in monthly epistaxis duration after 16 weeks of study treatment versus baseline."}
• {"endpoint_text":"- The monthly epistaxis duration after 16 weeks of study treatment is defined as the average of epistaxis duration during the last 12 weeks of study treatment (minutes/4-weeks period averaged for weeks 12 to 24, i.e. V3 to V6)","definition_or_measurement_approach":"Monthly epistaxis duration after 16 weeks = average epistaxis duration during weeks 12 to 24, expressed as minutes per 4-week period (mean of V3 to V6)."}
• {"endpoint_text":"- The monthly epistaxis duration at baseline is defined as the average of epistaxis duration during the observation period (minutes/4-weeks period averaged for weeks 0 to 8, i.e. V0 to V1).","definition_or_measurement_approach":"Baseline monthly epistaxis duration = average epistaxis duration during observation period weeks 0 to 8, expressed as minutes per 4-week period (mean of V0 to V1)."}

France

Earliest CTIS Part Ii Submission Date

11-02-2025

Latest Decision Or Authorization Date

03-11-2025

Processing Time Days

265

Number Of Sites

2

Number Of Participants

28

Primary sponsor

Full Name

Hospices Civils De Lyon

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France

Third parties

• {"country":"","full_name":"CHUV Lausanne university hospital","duties_or_roles":"Monetary support","organisation_type":""}
• {"country":"","full_name":"Boehringher Ingelheim","duties_or_roles":"Monetary support","organisation_type":""}