Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia

Trial Information
Who is this study for? Patients with hereditary hemorrhagic telangiectasia
What treatments are being studied? Pomalidomide
Status: Recruiting
Location: See all (11) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
Summary

This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks. Mean change from baseline to 24 weeks in the Epistaxis Severity Score (ESS) will be compared between treatment groups to determine pomalidomide efficacy.

Am I eligible for this trial?
Participation Requirements
Sex:
All
Minimum Age:
18
Healthy Volunteers:
No

• A clinical diagnosis of HHT as defined by the Curacao criteria

• Age > 18 years

• Platelet count ≥ 100,000/µl

• WBC ≥ 2,500/µl

• INR ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT or PTT per local laboratory designation) by local laboratory criteria (except for patients on a stable dose of warfarin or direct oral anticoagulants)

• Epistaxis severity score ≥ 3 measured over the preceding three months, measured at the screening visit

• A requirement for anemia, as determined by local laboratory hemoglobin assessment and normal ranges, and/or parenteral infusion of at least 250 mg of iron or transfusion of 1 unit of blood over the 24 weeks preceding the screening visit

• Females of childbearing potential (FCBP) must adhere to the scheduled pregnancy testing (once very two weeks) as required in the POMALYST REMS program. FCBP must a negative serum pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14 days prior to and again within 24 hours of prescribing pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking pomalidomide. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a vasectomy.

• Ability to understand and sign informed consent

• All study participants must agree to be registered into the FDA mandated POMALYST REMS program, and be willing and able to comply with the requirements of the POMALYST REMS program

Where is this trial taking place?
United States
California
UCSF HHT Center
Recruiting
San Francisco
Florida
University of Florida
Recruiting
Gainesville
Massachusetts
Massachussets General Hospital
Recruiting
Boston
Maryland
Johns Hopkins Hospital
Recruiting
Baltimore
Minnesota
Mayo Clinic
Recruiting
Rochester
North Carolina
University of North Carolina
Recruiting
Chapel Hill
Ohio
Cleveland Clinic
Recruiting
Cleveland
Pennsylvania
University of Pennsylvania Perelman School of Medicine
Recruiting
Philadelphia
Texas
UT Southwestern Medical Center
Withdrawn
Dallas
Utah
University of Utah Healthcare
Recruiting
Salt Lake City
Wisconsin
Medical College of Wiconsin
Recruiting
Milwaukee
Who do I contact about this trial?
Primary
Keith McCrae, MD
Mccraek@ccf.org
216 444-6833
Backup
Lisa Wisniewski, RN
wisniel@ccf.org
216-312-4282
When is this trial taking place?
Start Date: October 17, 2019
Estimated Completion Date: September 1, 2023
How many participants will be in this trial?
Target number of participants: 159
What treatment is being studied in this trial?
Experimental: Pomalidomide
Oral Pomalidomide will be provided as a capsule at 4 mg/day dose. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks.
Placebo Comparator: Placebo
A placebo matching the study drug will be provided as a capsule. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks.

This content was sourced from clinicaltrials.gov

Incidence of Cereblon in Intensive Care Patients
Status:Not yet recruiting
Start Date:December 1, 2021