Recruiting Patients at Cleveland Clinic for Trial

 

Cleveland Clinic LogoPrincipal Investigator: Keith McCrae, MD

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.

Study Question: Will Pomalidomide therapy reduce bleeding and the requirement for ongoing transfusion or iron therapy  in patients with HHT by 50% or greater?

Study Goals:

  1. To determine whether the use of Pomalidomide will reduce the requirement for transfusion and intravenous iron administration for patients with chronic gastrointestinal bleeding or nosebleeds due to HHT
  2. To monitor possible bleeding episodes in patients 6 months after discontinuation of Pomalidomide therapy.

You may qualify to participate if you:

  • Are 18 years of age or older with an HHT diagnosis
  • Have GI Bleeding OR Nosbleeds related to HHT
    • GI Bleeding MUST require at least 4 units of blood or 1gm of iron infusion per 4 month period
    • Nosebleeds MUST require 2 units of blood or 500mg of iron per 4 month period
  • Platelet count >125,000
  • Do not have any conditions listed in "Who Cannot Participate" section below

Who Cannot Participate:

  • Pregnant women
  • Prior treatment with Avastin within the past 6 weeks
  • Myelodysplasia
  • Requirement for chronic aspirin, NSAID therapy or anticoagulation therapy
  • Renal insufficiency
  • Currently enrolled in other interventional trials
  • Hemophilia, advanced liver disease, or known effect in the coagulation system (not including HHT itself)

Study Procedure: 

  • Screening period including labs and signing of informed consent.
  • Oral administration of 1 mg/day of Pomalidomide, increasing to a maximum of 5 mg/day at the physician’s discretion on a monthly basis.
  • Continuation of stable dose of Pomalidomide for 4 months.
  • Gradual reduction of Pomalidomide dose between 1-5 months until discontinuation.
  • Monitoring for 6 months after discontinuation of Pomalidomide.

What is required of me?

  • Travel to the Cleveland Clinic Main Campus approximately once each month
  • Blood tests at a doctor’s office or hospital near you two weeks after each Cleveland Clinic visit
  • There will be compensation for parking on the days when coming to the main campus is required

To participate in this trial, contact the Principal Investigator:  

Keith McCrae, MD
9500 Euclid Avenue, R35
Cleveland OH 44195
(216) 445-7809

Pick Cure HHT for Your CFC

Workplace Giving

CFCCombined Federal Campaign - CFC Number 10233

Think of the Combined Federal Campaign (CFC) as the federal government's version of United Way. All federal employees and military personnel get to select a charity of choice to receive donations via cash, check or automated paycheck deductions. The CFC charity drive runs from September 1 through December 15 of each year.

To designate Cure HHT as your desired CFC charity, use our CFC Number 10233 by clicking this linkWhen searching for Cure HHT, check under the "C" section rather than "H" for HHT Foundation International.

Please pass this information on to any family or friends who employed by the Federal government or military. We need everyone's support to discover a cure for this disease.

united-way-lock-up-rgb (Small)United Way

Cure HHT receives funds via many local United Way organizations. Does your employer offer a United Way campaign? It's an easy way to support the crucial mission to find, treat and cure HHT.

To designate Cure HHT through your United Way campaign, be sure to write in the full name and address as shown below:

Cure HHT
PO Box 329
Monkton, MD 21111

 

 

Contact Cathleen Kinnear at [email protected] or 410-357-9932 with any questions.

Cure HHT Launches New Center

UPMC

21st HHT Center of Excellence in North America
UPMC and The University of Pittsburgh
Pittsburgh, PA

After a year of coordinating committee meetings, organizing the mentor process, reviewing board reports and conducting a thorough site visit, Cure HHT is happy to announce the opening of its newest HHT Center of Excellence - UPMC and The University of Pittsburgh HHT Center.

This designation means that UPMC and The University of Pittsburgh meet the rigorous criteria Cure HHT requires in order to refer patients for care.

"A major component of Cure HHT’s mission is to ensure families have access to expert care," said Executive Director Marianne Clancy. "Our HHT Center review team was truly impressed with the strong group of specialists, nurses, genetics and medical professionals at UPMC who are able to screen, treat and manage adults and children with HHT."

Cure HHT, UPMC and the University of Pittsburgh has created a genuinely integrated health delivery system with one of the strongest ENT teams in the U.S. treating HHT.

“We are honored to be part of the HHT community," said Center Director Dr. Christopher Faber. "We have leveraged the clinical excellence of the UPMC enterprise to assemble a multidisciplinary team of adult and pediatric specialists who are dedicated to the care of patients with HHT. Coupled with our research partners at the University of Pittsburgh, we are committed to providing state-of-the-art care, advancing the understanding of this condition and contributing to the goal of finding a cure."

UPMC and The University of Pittsburgh HHT Center
200 Lothrop Street
Pittsburgh, PA 15213

Contact: 412-648-6161 (request a referral to the UPMC HHT Center)

> Full Press Release
> Patient Story
> Story from the Pittsburgh Tribune-Review

Faber, Christopher MD - UPMC

Christoper Faber, MD, HHT Center Clinical Director - He coordinates a multidisciplinary clinical approach to treating HHT patients, involving specialists in otolaryngology, neurosurgery, neurology, pulmonology, gastroenterology, genetics and radiology to assess, monitor and treat patients, creating a situation where care in one specialty must complement care provided by another.

 

 

 

Roman_Beth_L

Beth Roman, PhD, HHT Center Research Director - Her laboratory research uses zebrafish, which form blood vessels using the same molecular cues that guide mammalian vascular development, to uncover biological factors that underlie HHT. Her work has identified abnormalities in the cells that line blood vessels that lead to HHT vascular malformations, an important step in developing a therapy.

 

 

 

pedia

Suneeta Madan-Khetarpal, MD, Pediatric Team Leader - Her team of pediatric specialists at the Children's Hospital of Pittsburgh UPMC will care for patients less than 18 years of age.