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

Summary

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.

Conditions

• Hereditary Hemorrhagic Telangiectasia

Interventions

DRUG

Topical timolol maleate

* Topical timolol maleate 0.5% drops * Applied twice daily for 12 weeks (84 days) or until disappearance of lesions * Study drops will be applied to 3 cutaneous telangiectasias per patient

DRUG

placebo saline drops

Applied twice daily for 12 weeks (84 days) or until disappearance of lesions to 1 cutaneous telangiectasias per patient.

Sponsors & Collaborators

• University of California, San Francisco

  collaborator OTHER

• The Hospital for Sick Children

  collaborator OTHER

• University of Toronto

  collaborator OTHER

• Sunnybrook Health Sciences Centre

  collaborator OTHER

• Toronto Metropolitan University

  collaborator OTHER

• National Institute of Neurological Disorders and Stroke (NINDS)

  collaborator NIH

• Unity Health Toronto

  lead OTHER

Principal Investigators

• Marie E Faughnan, MD MSc FRCPC · Unity Health Toronto

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-05-31

Primary Completion

2019-08-30

Completion

2019-08-30

Countries

• Canada

Study Locations