Propranolol for Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients

NCT04113187 | Terminated Phase 3

• 2 other identifiers: CHUBX 2015/322016-002770-12
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder of angiogenesis associated with disabling epistaxis. Management of these nose bleedings requires more effective treatment. Propranolol, a beta-blocker, is a potentially useful therapeutic considering its anti-angiogenic properties. Our objective is to explore the efficacy of propranolol, three months after its introduction, on the cumulative duration of epistaxis in HHT patients.

Conditions

Osler Weber Rendu Disease; Hereditary Hemorrhagic Telangiectasia

Keywords

angiogenesis; epistaxis; propranolol

Outcome Measures

Primary Outcomes (1)

• Cumulative duration of epistaxis (in minutes)

  6 months after baseline (Day 0)

Secondary Outcomes (9)

• Frequency of epistaxis (number of episodes) per month

  At baseline (Day 0), 3 months and 6 months after baseline

• Number of cutaneous telangiectasia on hands and face

  At baseline (Day 0), 3 months and 6 months after baseline.

• Levels of hemoglobin

  At baseline (Day 0), 3 months and 6 months after baseline.

• Levels of ferritin

  At baseline (Day 0), 3 months and 6 months after baseline.

• Number of red blood cells transfusions

  At baseline (Day 0), 3 months and 6 months after baseline.

Study Arms (2)

Propranolol arm

EXPERIMENTAL

Drug: Propranolol treatment

Placebo arm

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Propranolol treatment DRUG

40 mg twice a day (morning and evening), per os, during three months

Propranolol arm

Placebo DRUG

per os, twice a day (morning and evening) during three months

Placebo arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years
• Confirmed diagnosis of HHT : 3 or more Curaçao criteria (spontaneous and recurrent epistaxis; multiple telangiectasia at characteristic sites; visceral lesions such as gastrointestinal telangiectasia or arteriovenous malformations; family history: a first degree relative with HHT according to these criteria ) or mutations of genes encoding for ALK1, ENG or SMAD4
• Patient insured under the French social security system
• Free and informed consent signed by investigator and patient

You may not qualify if:

• Pregnancy or breast-feeding
• Current beta-blocker treatment
• Hypersensitivity to the active substance or excipient
• Patients with type I or type II diabetes, treated with insulin, sulphonylureas or meglitinides
• Patients with heart failure
• Patients with liver failure
• Patients with hepatic arteriovenous malformations responsible for high-output cardiac failure or severe hepatic dysfunction
• Patients with severe psoriasis (PASI\>10)
• Contra-indication to beta-blocker treatment : asthma, chronic obstructive bronchopneumopathy, atrioventricular block of second or third degrees without pacemaker, Prinzmetal's angina, bradycardia \< 50bpm, Raynaud's phenomenon, oblitering arteriopathy of the lower limbs, low blood pressure, non-treated pheochromocytoma
• Protected adult according to french law

Sponsors & Collaborators

• University Hospital, Bordeaux: lead
• AMRO-HHT-France - Association Maladie de Rendu-Osler: collaborator

Study Sites (1)

CHU de Bordeaux - service de médecine interne

Bordeaux, France

Location

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic; Epistaxis

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nose Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Respiratory; Signs and Symptoms

Study Officials

• Anne CONTIS, MD

  University Hospital, Bordeaux

  PRINCIPAL INVESTIGATOR

• Antoine BENARD, MD, PhD

  University Hospital, Bordeaux

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2019
• First Posted: October 2, 2019
• Study Start: June 23, 2020
• Primary Completion: May 19, 2022
• Study Completion: May 19, 2022
• Last Updated: June 23, 2026

Record last verified: 2022-06

Locations

FR (1)