A Phase 1/2 interventional study of Topical timolol maleate and placebo saline drops in Hereditary Hemorrhagic Telangiectasia, sponsored by Unity Health Toronto. Completed at 1 site in Canada. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-09-21.
Sponsored by Unity Health Toronto · Phase 1/2, Interventional, and Treatment
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.
This is a small study of 5 patients from St. Michael's Hospital who have HHT and at least 5 typical telangiectasias.
Patients who anticipate a major surgery during this study or are pregnant, breast feeding or on other beta blocker medication may not enroll in this study.
This study lasts 12 weeks (84 days). During this time, subjects will apply a drop of either Apo-timop 0.5% or a placebo solution to 4 telangiectasias twice daily.
The active study medication is called Apo-Timop and is a clear liquid solution stored in a bottle. An eye dropper is used for application.
Subjects will receive four numbered bottles for every 28 day period as well as a photo which indicates which bottle is to be applied to which telangiectasia.
Neither the subject nor the research staff will know which telangiectasia will receive the placebo.
Apo-timop, is not part of the standard therapeutic regimen for HHT. It is a Health Canada approved medication which is applied as an eye drop, that has been shown to reduce pressure in the eye and is commonly used for glaucoma.
106 studies on the registry are indexed under Telangiectasia, Hereditary Hemorrhagic; 24 are open to participants now.
This study's enrollment of 5 is below the median of 33 across 61 interventional studies indexed under Telangiectasia, Hereditary Hemorrhagic.
Browse Telangiectasia, Hereditary Hemorrhagic studies →Unity Health Toronto is the lead sponsor of 447 studies on the registry; 82 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
COPD
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Drug: Topical timolol maleate
placebo saline drops -Applied twice daily for 12 weeks (84 days) or until disappearance of lesions to one cutaneous telangiectasias per patient.
Drug: placebo saline 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
Also known as: Topical timolol maleate 0.5% drops
Applied twice daily for 12 weeks (84 days) or until disappearance of lesions to 1 cutaneous telangiectasias per patient.
Change in Lesion Area of Treated Telangiectasia.
Change in lesion area (compared with baseline measurement) of treated telangiectasia.
Time frame: 84 days
Descriptive Changes in Histopathology in Baseline vs Treated Lesions
Time frame: 84 days
Serum Angiogenic Markers
Time frame: 84 days
Stability of Area of Untreated Telangiectasias Over the 84 Day Period
Time frame: 84 days
Blood Flow Velocity and Volume Flow Rates
Time frame: 84 days
Each Participant received both treatment and placebo
| Milestone | Topical Timolol Maleate | Placebo |
|---|---|---|
| Started | 5 | 5 |
| Completed | 5 | 5 |
| Not completed | 0 | 0 |
Change in lesion area (compared with baseline measurement) of treated telangiectasia.
| mm^2 | Topical Timolol Maleate | Placebo |
|---|---|---|
| Change in Lesion Area of Treated Telangiectasia. | -6 ± 11 | -8 ± 17 |
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
Collected over 84 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Topical Timolol Maleate | 0/5 (0%) | 0/5 (0%) | 1/5 (20%) |
| Placebo | 0/5 (0%) | 0/5 (0%) | 1/5 (20%) |
| Event | Topical Timolol Maleate | Placebo |
|---|---|---|
| Minor infection at biopsy siteSkin and subcutaneous tissue disorders | 1/5 | 1/5 |
| Minor bleeding at biopsy siteSkin and subcutaneous tissue disorders | 1/5 | 1/5 |
Total 5 participants treated (15 treatment lesions and 5 placebo lesions)
| Age, Continuous(years) | All Study Participants |
|---|---|
| Mean | 66 ± 5 |
| Sex: Female, Male(Participants) | All Study Participants |
|---|---|
| Female | 2 |
| Male | 3 |
| Race (NIH/OMB)(Participants) | All Study Participants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 3 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | All Study Participants |
|---|---|
| Canada | 5 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
The record gives a start of May 2013, a primary completion of Aug 2019 and a completion of Aug 2019. It does not say whether these dates are actual or estimated.
Results were first posted in Sep 2023, 4 years after primary completion.
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This study is completed, as verified in Aug 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Timolol
Telangiectasia, Hereditary Hemorrhagic →