Doxycycline for Hereditary Hemorrhagic Telangiectasia (HHT)

(HHT Trial)

Recruiting in Palo Alto (17 mi)

Overseen by Marie E Faughnan, MD MSc FRCPC

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Unity Health Toronto

Prior Safety Data

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Research Team

Marie E Faughnan, MD MSc FRCPC

Principal Investigator

St. Michael's Hospital / The University of Toronto

Eligibility Criteria

Adults over 18 with Hereditary Hemorrhagic Telangiectasia (HHT) who experience frequent nosebleeds and have specific HHT-related genetic mutations. Participants must not be pregnant, breastfeeding, planning to become pregnant, or have unstable illnesses or allergies to doxycycline.

Inclusion Criteria

I have been diagnosed with HHT either through clinical evaluation or genetic testing.

I am older than 18 years.

I or my family have a mutation in the ENG, ALK1, or SMAD4 gene.

Exclusion Criteria

Your liver transaminase levels are more than twice the normal limit.

Breastfeeding

I currently have an infection.

Treatment Details

Interventions

• Doxycycline Hyclate (Tetracycline Antibiotic)
• Placebo (Drug)

Trial Overview The trial is testing if doxycycline can reduce the severity of nosebleeds in HHT patients compared to a placebo. It's a Phase II study where participants will take either doxycycline or a placebo for six months and then switch treatments.

Participant Groups

Group I: doxycycline Hyclate Active Control 1 Intervention

subjects will be treated with a 6-month course of doxycycline oral capsule at a dose of 100mg twice daily

Group II: Placebo Placebo Group 1 Intervention

subjects will be given a placebo oral capsule twice daily for 6-months

Doxycycline Hyclate is already approved in Canada for the following indications:

Approved in Canada as Doxycycline Hyclate for:

• Acne
• Malaria
• Skin infections
• Sexually transmitted infections
• Lyme disease

Who Is Running the Clinical Trial?

Unity Health Toronto

Lead Sponsor

Trials

572

Recruited

470,000+

Dr. Sharon Straus

Unity Health Toronto

Chief Medical Officer

MD and MSc in Clinical Epidemiology, University of Toronto

Altaf Stationwala

Unity Health Toronto

Chief Executive Officer

Bachelor's degree in Health Administration, University of Ottawa

University of Pittsburgh

Collaborator

Trials

1,820

Recruited

16,360,000+

David Apelian

University of Pittsburgh

Chief Executive Officer since 2019

PhD in Molecular Biology from Rutgers University, MD from the University of Medicine and Dentistry of New Jersey, MBA from Quinnipiac University

Pamela D. Garzone

University of Pittsburgh

Chief Medical Officer

PhD in Clinical Science from the University of Pittsburgh

Duke University

Collaborator

Trials

2,495

Recruited

5,912,000+

Mary E. Klotman

Duke University

Chief Executive Officer since 2017

MD from Duke University School of Medicine

Michelle McMurry-Heath

Duke University

Chief Medical Officer since 2020

MD from Duke University School of Medicine

Sunnybrook Health Sciences Centre

Collaborator

Trials

693

Recruited

1,569,000+

Dr. Rajin Mehta

Sunnybrook Health Sciences Centre

Chief Medical Officer

MD from University of Toronto

Dr. Andrew J. Smith

Sunnybrook Health Sciences Centre

President and CEO since 2017

MD, MSc from University of Toronto; Surgical Oncology Fellowship at Memorial Sloan-Kettering Cancer Center

Feinstein Institute for Medical Research

Collaborator

Trials

22

Recruited

5,700+

Barrow Neurological Institute

Collaborator

Trials

27

Recruited

7,100+