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

• Register: ClinicalTrials.gov
• Last refreshed on: 29 November 2021
• Main ID: NCT01752049
• Date of registration: 14/12/2012
• Prospective Registration: Yes
• Primary sponsor: Unity Health Toronto
• Public title: Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
• Scientific title: Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
• Date of first enrolment: May 2013
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01752049
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: N/A

Countries of recruitment

Canada

Contacts

• Name: Marie E Faughnan, MD MSc FRCPC
• Affiliation: Unity Health Toronto

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hereditary Hemorrhagic Telangiectasia

Intervention(s)

Drug: placebo saline drops

Drug: Topical timolol maleate

Primary Outcome(s)

Mean reduction in lesion area (compared with baseline measurement) of treated telangiectasia. [Time Frame: 84 days]

Secondary Outcome(s)

1. From Tissue: Descriptive changes in histopathology in baseline vs treated lesions, vessel density and distribution of capillaries, arterioles and venules. [Time Frame: 84 days]

2. From speckle variance OCT: Changes in lesion area, blood flow velocity and volume flow rates (treated vs baseline/ placebo). [Time Frame: 84 days]

4. Stability of area of untreated telangiectasias over the 84 day period (placebo group) [Time Frame: 84 days]

3. Serum angiogenic markers (Aushon Blood-based Biomarkers in Clinical Research kit, analyzing 5- angiogenic biomarkers): Endoglin, BMP-9, VEGF+, TGF-beta1, TSP-1 [Time Frame: at baseline and 84 days.]

Secondary ID(s)

2U54NS065705-06

BVMC 6207

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Sunnybrook Health Sciences Centre

Ryerson University

University of California, San Francisco

University of Toronto

National Institute of Neurological Disorders and Stroke (NINDS)

The Hospital for Sick Children