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?
- 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
- 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
- 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)
- 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