Overview

Doxycycline for Hereditary Hemorrhagic Telangiectasia

Status:
Recruiting
Trial end date:
2021-12-01
Target enrollment:
0
Participant gender:
All
Summary
This study will investigate the effectiveness of oral doxycycline for the treatment of recurrent nasal hemorrhage in Hereditary Hemorrhagic Telangiectasia (HHT) subjects. The primary outcome for the trials will be the reduction of epistaxis severity (minutes of bleeding per week). The biological outcomes of interest are the regression of vascular malformations as well as tissue and circulation biomarkers of the relevant mechanistic pathways. A Phase II, randomized double-blind placebo-controlled crossover trial. Approximately 30 subjects with HHT, with moderate-severe recurrent epistaxis will participate in the randomized double-blind placebo-controlled cross over trial. Subject will be treated with a 6-month course of doxycycline 100mg twice daily or placebo twice daily.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
St. Michael's Hospital, Toronto
Unity Health Toronto
Collaborators:
Barrow Neurological Institute
Duke University
Feinstein Institute for Medical Research
Sunnybrook Health Sciences Centre
University of Pittsburgh
Treatments:
Doxycycline
Criteria
Inclusion Criteria:

- Age >+ 18 years

- Clinical HHT diagnosis or genetic diagnosis of HHT

- Known personal or familial endoglin (ENG), ALK1 or SMAD4 mutation

- Epistaxis at least 15 min per week (mean for past month)

- At least two skin telangiectases

- >2mm diameter available for excisional biopsy,

- at least two other telangiectases (skin or mucosal) available for micro-imaging

- Ability to give written informed consent

- including compliance with the requirements of the study

Exclusion Criteria:

- Allergy/intolerance to the study drug or related agents

- Unstable medical illness

- Acute infection

- Creatinine > upper limit of normal (ULN)

- Liver transaminases (AST or ALT) >= 2x ULN

- Recent (within 2 month) use of study drug or other tetracycline agents

- Women who are pregnant

- Breastfeeding

- Plan to become pregnant during of the study

- Beta human chorionic gonadotropin (BHCG) level <6 IUL (re-test if 6-24 IU/L)

- Specific contra-indications for study drug