Trial Outcomes & Findings for Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept (NCT NCT01752049)

Results Overview

Change in lesion area (compared with baseline measurement) of treated telangiectasia.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

5 participants

Primary outcome timeframe

84 days

Results posted on

2023-09-21

Participant Flow

Each Participant received both treatment and placebo

Unit of analysis: Telangiectasia (Lesions)

Participant milestones

Participant milestones
Measure	Topical Timolol Maleate Topical timolol maleate 0.5% drops will be applied twice daily for 12 weeks (84 days) or until disappearance of lesions on 3 cutaneous telangiectasias per patient (total of 5 patients)	Placebo placebo saline drops will be applied twice daily for 12 weeks (84 days) or until disappearance of lesions to one cutaneous telangiectasias per patient (same 5 patient as Topical treatment group)
Overall Study STARTED	5 15	5 5
Overall Study COMPLETED	5 15	5 5
Overall Study NOT COMPLETED	0 0	0 0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Study Participants n=5 Participants Drug: • Topical timolol maleate 0.5% drops * 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 telangiectasia per patient). 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 4 cutaneous telangiectasias per patient (timolol drops for 3 telangiectasia per patient and placebo drops to 1 telangiectasia per patient).
Age, Continuous	66 years STANDARD_DEVIATION 5 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Canada	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: 84 days

Population: 5 participants enrolled, each participant have Hereditary Hemorrhagic Telangiectasia (HHT) and have at least 5 typical telangiectasias on their hands or face.

Change in lesion area (compared with baseline measurement) of treated telangiectasia.

Outcome measures

Outcome measures
Measure	Topical Timolol Maleate n=15 Lesion (Telangiectasia) 5 Participants received 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	Placebo n=5 Lesion (Telangiectasia) The same 5 participants will receive placebo saline drops on one cutaneous telangiectasia. Applied twice daily for 12 weeks (84 days) or until disappearance of lesions to one cutaneous telangiectasias per patient.
Change in Lesion Area of Treated Telangiectasia.	-6 mm^2 Standard Deviation 11	-8 mm^2 Standard Deviation 17

SECONDARY outcome

Timeframe: 84 days

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Topical Timolol Maleate n=5 participants at risk 5 patients received Topical timolol maleate 0.5% drops. Applied twice daily for 12 weeks (84 days) Study drops were applied to 3 cutaneous telangiectasias per patient. Each patient had 2 lesional biopsies (one at baseline, and one at end of treatment)	Placebo n=5 participants at risk The same 5 patients received placebo saline drops for one telangiectasia each. The placebo drops were applied twice daily for 12 weeks (84 days)
Skin and subcutaneous tissue disorders Minor infection at biopsy site	20.0% 1/5 • Number of events 1 • 84 days	20.0% 1/5 • Number of events 1 • 84 days
Skin and subcutaneous tissue disorders Minor bleeding at biopsy site	20.0% 1/5 • Number of events 1 • 84 days	20.0% 1/5 • Number of events 1 • 84 days

Additional Information

Dr. Marie Faughnan

St. Michael's Hospital

Phone: 416-864-6060

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place