Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept

BVMC 6207

Hereditary hemorrhagic telangiectasia (HHT) is a hereditary vascular condition characterized by the development of abnormal connections between arteries and veins throughout the body, called vascular malformations. These abnormal blood vessels are referred to as arteriovenous malformations (AVM) if they are large and telangiectasias if they are small. Telangiectasias develop due to irregular growth of blood vessels. Anti-angiogenic therapy, such as the drug Apo-Timop, curbs the growth of new blood vessels. Apo-Timop is included in a class of medications called beta-blockers. Anti-angiogenic therapies exert their beneficial effects in a number of ways: by disabling the agents that activate and promote cell growth, or by directly blocking the growing blood vessel cells. The investigators think that anti-angiogenic therapy may lead to the shrinking of telangiectasia in people with HHT. The investigators hope that this study will provide us with proof of this concept and might lead to the development and study of anti-angiogenic therapies to help improve the lives of individuals with vascular malformations.

2013-05-01

2019-08-30

2019-08-30

Completed

Early phase I

5

Inclusion Criteria: 1. Definite clinical or genetic diagnosis of HHT 2. Known ENG or ALK1 mutation (personal or familial) 3. Age\>=18 years 4. At least 5 typical (round/ovoid, not spider or linear) cutaneous telangiectasia (size range 2-5mm) on hands (not including lesions on over inter-phalangeal joints) or face Exclusion Criteria: 1. Contraindication to systemic beta-blocker (severe asthma, severe COPD, sinus bradycardia, 2nd or 3rd degree AV block, overt heart failure, hypotension, allergy/intolerance/ hypersensitivity to timolol) 2. Current treatment with systemic beta-blocker 3. Current participation in other therapeutic trial for HHT 4. Current pregnancy or breastfeeding.

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2023-09-21