Clinical Trials

YOU can help advance HHT Research!

HHT Clinical Trials

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 Cure HHT office at 410-357-9932 or email [email protected].


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.

Ongoing Clinical Trials

Brain AVM Study

HHT patients with a Brain AVM, whether or not its been treated, should contact Nicole Schaefer by email or 410-357-9932 to determine eligibility. NO TRAVEL required, information gathering only, one hour of your time - this is all that is needed to impact this critical area of study!

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Doxycycline - Nose bleeding

Doxycycline, an inexpensive generic medication used by physicians for decades to treat bacterial infections, has displayed potent properties in blocking growth of new blood vessels inside and outside the body. The purpose of this study is to determine whether Doxycycline can be used to control nosebleeds (epistaxis) for patients with HHT.

Learn more about the Doxycycline Study at the following locations:

University of Toronto 

Fluorescein Imaging - Retinal Vascular Abnormalities (Washington University, MO)

The study team at Washington University School of Medicine in St. Louis is currently enrolling participants with HHT for a research study. The goal of the study is to determine whether the rate of HHT-related retinal vascular abnormalities might be higher than previously reported when utilizing modern-day retinal imaging techniques. To that end, the study team aims to enroll 30 patients with HHT that will come in for one office visit and undergo an ophthalmological exam, fluorescein angiography (FA) and optical coherence tomography (OCT). Participants will also complete questionnaires about their visual function and severity of their HHT.

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HHT Clinical Trials:

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


Past Clinical Trials

Bevacizumab - Nose bleeding (Stanford University, CA)

Cure HHT is excited to announce that “A Randomized Controlled Trial of Bevacizumab for HHT-Related Epistaxis” (aka “Avastin Trial”) conducted at Stanford University recruited its last patient on February 5, 2019!

THANK YOU to the 40 HHT patients who participated in the Avastin trial, many of whom traveled across the country to Palo Alto, California, and to the generous donor who established a travel reimbursement fund for those who needed financial assistance to make the journey!

Many patients with HHT suffer from recurrent, profound nose bleeds (epistaxis), which can require multiple blood transfusions, iron treatments, and trips to the operating room.

One possible way to reduce nose bleeds in HHT is by blocking the growth of the abnormal blood vessels in the nose using a medicine called Avastin (bevacizumab). This medicine has been studied in HHT patients with promising results as a safe an effective way to improve nasal bleeding.

This study’s aim was to evaluate the effectiveness and cost savings of using Avastin injection versus saline placebo in patients with HHT who also underwent cauterization surgery.

The preliminary results of this clinical trial will be presented in Puerto Rico this June at the 2019 International Scientific Conference. Cure HHT will make the final results available as soon the manuscript is published.

North American Study of Epistaxis (NOSE Study)

The first multi-center clinical trial developed and conducted by Cure HHT. This study was designed to carefully examine the benefit and safety of 3 nasal sprays for patients with HHT-related epistaxis and determine which of these agents are overall beneficial to the patient.

Each of these sprays approached the problem from a different mechanism of action. 140 patients with moderate to severe nosebleeds secondary to HHT were randomized to receive one of four intranasal sprays for a period of 12 weeks and then followed for an additional 12 weeks off therapy. A placebo arm was included to allow an accurate estimate of both benefit and safety. The primary measure of effectiveness was the frequency of epistaxis. Other measures of effectiveness included the Hoag Epistaxis Severity Score (ESS), a quality of life survey, satisfaction with treatment, hemoglobin level, and transfusion requirements.

The spray agents included in the study:

  1. Saline spray (Placebo)
  2. Estriol (a low dose estrogen)
  3. Tranexamic acid (a drug that promotes clotting)
  4. Bevacizumab (also known as Avastin, a drug that might actually reverse abnormal blood vessel growth). The dose that will be used in the NOSE Study is 4mg per day, about 1% as potent as the intravenous dose. We expect that side effects will be minimal.

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Pomalidomide - GI bleeding or Nose bleeding (Cleveland Clinic, OH)

This study will be conducted at Cleveland Clinic Main Campus and Regional Hospital Facilities. This study will evaluate patients with GI bleeding due to HHT or with HHT-related nosebleeds that require ongoing transfusion or iron replacement therapy. Patients with GI bleeding must have a requirement for at least 4 units of blood or 1 gm of iron infusion per 4 month period, while patients with nosebleeds must require 2 units of blood or 500 mg of iron per 4 month period. The effect of Pomalidomide on bleeding will be assessed during an initial 4-5 month period in which the dose of Pomalidomide is increased monthly, as needed. Patients will then be observed over a 4 month period on a dose of Pomalidomide found to be effective, then over a dose tapering period and for 6 months after stopping the drug.

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Timolol - Skin telangiectasia (Johns Hopkins, MD)

The Johns Hopkins Department of Dermatology is beginning a study to determine if topical timolol is an effective treatment of telangiectasia spots in HHT patients. The topical liquid treatment has traditionally been used for infant hemangiomas, a type of birthmark made of blood vessels, with successful results.

The goal of the study is to reduce and hopefully eliminate skin telangiectasia using the topical timolol. Current telangiectasia treatments available are laser therapy and surgical skin-grafting, both of which are costly and may be associated with significant side effects.

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