Research Studies/Clinical Trials

YOU can help advance HHT Research!

There are several HHT related research studies currently taking place. You or a family member may be interested in furthering the advancement of HHT research by participating in one of these clinical research studies. You can contact the primary researcher directly, call the HHT Foundation office at 800-448-6389.

Information on active clinical trials is presented as a service to the HHT community. However, anyone visiting these pages should be aware that the information contained has been submitted directly by those responsible for creating the trials. They bear sole responsibility for the accuracy of information relating to their trial(s). Clinical trials are designed to test the effectiveness and safety of drugs not yet approved for market.
 
Not all doctors will participate in any given clinical trial, and not all patients will qualify for these trials. Any decisions regarding clinical trials are serious ones and must be made after careful consultation with your healthcare provider.

Click HERE to read the most current HHT Clinical Research Trials.

NORTH AMERICAN STUDY OF EPISTAXIS  (NOSE Study)

Prinicipal Investigator - Dr. James Gossage, HHT Foundation Medical Director

This will be the largest multi-centered study of nosebleed treatment ever conducted. The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays for patients with HHT-related nosebleeds and definitively determine which of these are beneficial. Each of these sprays will approach the problem from a different mechanism of action.  The U.S. Federal Drug Administration (FDA) has reviewed our application and approved the usage of these three agents.

Click HERE to read the latest news article about the NOSE Study.

Research Plan
140 patients with moderate to severe epistaxis (nosebleeds) secondary to HHT will be randomized to receive one of four intranasal sprays for a period of 12 weeks and then followed for an additional 12 weeks off therapy. Enrollment in this study will occur over a period of 12-18 months. The primary measure of efficacy will be the frequency of epistaxis. Secondary measures will include the duration of epistaxis, Hoag Epistaxis Severity Score (ESS), a quality of life survey, satisfaction of treatment, hemoglobin level, and transfusion requirements.

The Nasal Sprays

1.  Saline spray  (Placebo)
2. Estriol  (a low dose estrogen)
3. Tranexamic Acid  (a drug that promotes clotting)
4. Bevacizumab  (Avastin - a drug that can reduce blood vessel growth)

Avastin has both received hype about effectiveness and concerns about side effects. Nearly all of the serious complications that have been reported so far have occurred with an intravenous dose of 300-400 mg (the dose typically used to treat cancer). The dose that will be used in the NOSE Study is 4 mg per day, about 1% as potent as the intravenous dose. We expect the side effects will be minimal. A placebo has been included to allow an accurate estimate of both efficacy and safety, and to finally determine whether any of these treatments are truly beneficial.

Participating HHT Centers

Currently Enrolling Patients

Georgia Health & Sciences University
Melissa James, RN
706-721-8391
mjames@georgiahealth.edu

Oregon Health Sciences University
Carrie Farrar
503-494-4233
farrarc@ohsu.edu

University of California, Los Angeles (UCLA)
Barbara Quinn, RN
310-481-7543
bquinn@mednet.ucla.edu

University of Utah
Renee Neuharth, BS
801-587-4877
renee.neuharth@hsc.utah.edu

Washington University
Stacy Schrum
314-362-7507
sschrum@dom.wustl.edu

Final Stages of Approval Process

 Johns Hopkins

Interested Participants
We will send email alerts to members and update the website, as each center receives their approvals and are ready to enroll. Then interested participants should contact the participating center directly. Until then, you can contact: Nicole Schaefer, Director of Education & Research Programs at nicole.schaefer@hht.org or 800-448-6389.

BRAIN AVM STUDY
 
William Young, MD
University of California, San Francisco

Marie Faughnan, MD
University of Toronto

HHT Centers of Excellence across North America are now actively recruiting HHT patients to participate in this NIH funded study.  The goal of this research is to determine what genetic and clinical factors signal high risk for hemorrhage from brain AVMs.  Ultimately, the results will help doctors make decisions about brain AVM treatment for individual patients and will drive further research in brain AVM therapies.

This is a five year study being funded by the National Institute for Health (NIH)/National Institute for Neurological Disorders and Stroke (NINDS).

Click HERE for participating centers contact information

Eligibility

• Definite Clinical or Genetic Diagnosis of  HHT
• Live in the United States or Canada
• Need HHT patients with and without Brain AVMs

We are especially interested in HHT patients who have been diagnosed with a Brain AVM, whether or not it has been treated.

Treated at an HHT Center - If you have been treated at an HHT Center, then click on the link above to contact that center for participation in the study.

Not Treated at an HHT Center - The HHT Foundation will be recruiting patients remotely who have been diagnosed with a Brain AVM and who are over 18 years of age if the patient is not affiliated with a participating HHT Center. In the future, this will extend to children under 18 years of age.

Contact
Nicole Schaefer
HHT Foundation International
(800) 448-6389
Email: nicole.schaefer@hht.org

VARIOUS CLINICAL TRIALS

You can check ongoing HHT clinical trials through the website www.clinicaltrials.gov. You must spell out Hereditary Hemorrhagic Telangiectasia in the search box. This website will not acknowledge the abbreviation HHT.