Phase 2
N=10
Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
Hereditary Hemorrhagic Telangiectasia
Bottom Line
View on ClinicalTrials.gov: NCT01752049 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Sep 2023
Primary outcome: Primary: Change in Lesion Area of Treated Telangiectasia. — -6; -8 mm^2
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Topical timolol maleate (Drug); placebo saline drops (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Unity Health Toronto
- Primary completion
- Aug 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Lesion Area of Treated Telangiectasia. |
-6; -8 | — |
| SECONDARY Descriptive Changes in Histopathology in Baseline vs Treated Lesions |
— | — |
| SECONDARY Serum Angiogenic Markers |
— | — |
| SECONDARY Stability of Area of Untreated Telangiectasias Over the 84 Day Period |
— | — |
| SECONDARY Blood Flow Velocity and Volume Flow Rates |
— | — |
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.
Eligibility Criteria
Inclusion Criteria
- Definite clinical or genetic diagnosis of HHT
- Known ENG or ALK1 mutation (personal or familial)
- Age>=18 years
- 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
- 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)
- Current treatment with systemic beta-blocker
- Current participation in other therapeutic trial for HHT
- Current pregnancy or breastfeeding.
Data sourced from ClinicalTrials.gov (NCT01752049). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.