Pomalidomide - GI bleeding or Nose bleeding

Pomalidomide in HHT and Transfusion-Dependent Vascular Ectasia: a Phase I Study

Principal Investigator: Keith McCrae, MD

Overview

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?

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

Interested in participating?

To participate in this trial, contact the Principal Investigator:

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