CompletedPhase 2

Doxycycline for Hereditary Hemorrhagic Telangiectasia

Canada13 participantsStarted 2018-09-12

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

The reduction in epistaxis (nose bleeding) severity over 96 weeks

Timeframe: daily for 96 weeks

Trial details

NCT IDNCT03397004

SponsorUnity Health Toronto

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-02-18