Pomalidomide for the Treatment of Bleeding in HHT (NCT03910244) | Clinical Trial Compass
CompletedPhase 2
Pomalidomide for the Treatment of Bleeding in HHT
United States145 participantsStarted 2019-10-17
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. A clinical diagnosis of HHT as defined by the Curacao criteria
✓. Age ≥ 18 years
✓. Platelet count ≥ 100,000/µl
✓. White Blood Count (WBC) ≥ 2,500/µl
✓. International Normalized Ratio (INR) ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT or partial thromboplastin time (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
✓. All study participants must agree to be registered into the FDA mandated POMALYST Risk Evaluation and Mitigation Strategy (REMS) program, and be willing and able to comply with the requirements of the POMALYST REMS program
✕. Hepatic insufficiency, bilirubin \> 2.0 (or \>4.0 in the setting of a prior clinical or genetic diagnosis of Gilbert's syndrome) or transaminases \> 3.0x normal
What they're measuring
1
Change From Baseline Epistaxis Severity Score
Timeframe: 4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment