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
Researchers want to determine if the investigational drug, Pazopanib, will lessen nosebleed severity in patients with Hereditary Hemorrhagic Telangiectasia (HHT).
HHT Centers of Excellences in every region of the U.S. are now recruiting patients to participate in this study. Learn more about this study and locate a recruiting center by reading the comprehensive protocol sheet.
Adult HHT patients in the U.S. with nosebleeding of at least 20 min per week and average hemoglobin <11 are eligible to participate. Participation will cost nothing for most patients, as expenses will be compensated based on miles traveled.
Participating Sites (click/hover on location for details):
Massachusetts General Hospital
Hanny Al-Samkari, MD
VAD044 HHT Study - Vaderis
Recruitment for this trial has ended.
Study overview: Research has shown that in the small vascular malformations called telangiectases, there is one protein, called AKT, which is more active than in other parts of the vessels, where there is no telangiectases. The genetic mutations causing HHT seem to indirectly activate AKT. Although there are ways to manage this condition, there are currently no approved treatments available. The purpose of this research study is to assess how safe and well tolerated two different doses of VAD044 are in patients with HHT. It will also evaluate the effects of the two doses of VAD044 on HHT symptoms and other physiological indicators, and how the study drug enters, moves through and exits the body (Pharmacokinetics or PK).
VAD044 is a chemical molecule which is specifically targeted to inhibit AKT. VAD044 would bind to AKT and make it less active. This would in turn control or limit the activity or overgrowth of cells building the vessels, which should give the opportunity for blood vessels to return to a more inactive state and to a more normal function. The form that this study drug would be given is an oral capsule. VAD044 is considered investigational, which means that it has not been approved by the U.S. Food and Drug Administration.
Past Clinical Trials
Pomalidomide for the Treatment of HHT (PATH)
The PATH Study explored the use of the oral medication pomalidomide for the treatment of nosebleeds in HHT at 11 research centers across the United States. Adults suffering from HHT with moderate to severe nosebleeds (epistaxis) who require iron infusions or blood transfusions were eligible. During the 6-month study, patients received either pomalidomide or a matching placebo (sugar pill) in addition to their usual care.
Doxycycline - Nose Bleeding (2023)
HHT patients who lived in Missouri or Illinois with moderate to severe nosebleeds were invited to learn more about a Washington University study to assess Doxycycline’s effectiveness in reducing nosebleeds.
Doxycycline is an FDA approved antibiotic used to treat certain infections which can occur in the lungs, skin, eyes, or urinary tract. In addition to its use as an antibiotic, doxycycline also has the ability to prevent the development of new blood vessels and bleeding – which is why it was chosen for this study.
Doxycycline - Nose Bleeding
University of California (LA) and University of Toronto Doxycycline Study. More information about these trials and their findings will be posted when available.
Note: 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 was to determine whether Doxycycline can be used to control nosebleeds (epistaxis) for patients with HHT.
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!
If you participated in the Bevacizumab (Avastin) clinical trial at Stanford and are interested in learning which intervention you received (drug or placebo), please contact Erik Chan at [email protected].
The preliminary results of this clinical trial was presented in Puerto Rico at the 2019 International Scientific Conference. Cure HHT will provide the scientific article once it has been published. In the meantime, the study summary final report is available for you to review. READ Final Report for Intranasal Bevacizumab for HHT-Related Epistaxis
Fluorescein Imaging Pilot Study - Retinal Vascular Abnormalities (Washington University, MO)
The study team at Washington University School of Medicine in St. Louis enrolled 18 patients (age range, 22-65 years) with a clinical diagnosis of HHT. 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.
RESULT: Of the 18 patients recruited, fine telangiectatic vessels with capillary dilation and tortuosity were identified in 78% by fluorescein angiography (FA) imaging. Read Publication >>
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. Learn More >>
The spray agents included in the study:
- Saline spray (Placebo)
- Estriol (a low dose estrogen)
- Tranexamic acid (a drug that promotes clotting)
- 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.
Pomalidomide Pilot Study - GI bleeding or Nose bleeding (Cleveland Clinic, OH)
This pilot study was conducted at Cleveland Clinic Main Campus and Regional Hospital Facilities. This study evaluated patients with GI bleeding due to HHT or with HHT-related nosebleeds that require ongoing transfusion or iron replacement therapy. The effect of Pomalidomide on bleeding was assessed during an initial 4-5 month period in which the dose of Pomalidomide was increased monthly, as needed. Patients were then 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.
RESULT: Pomalidomide for the Treatment of HHT (PATH) has been funded by a grant from the National Heart, Lung and Blood Institute (NHLBI), and is led by researchers at the Cleveland Clinic and RTI International, based on the positive outcomes of Pomalidomide for HHT patients recruited for the pilot study. PATH is a multi-center clinical trial that will recruit 159 HHT patients.
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. Learn More >>