A Study to Evaluate Pomalidomide to Treat Bleeding in Hereditary Hemorrhagic Telangiectasia (HHT)

Overview

About this study

The purpose of this study is to evaluate pomalidomide vs. placebo in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate-to-severe epistaxis who 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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria: 

  • A clinical diagnosis of HHT as defined by the Curacao criteria.
  • Age > 18 years old.
  • Platelet count ≥ 100 x 10^9 /L.
  • WBC ≥ 2.5 x 10^9 /L.
  • INR ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time by local laboratory criteria (aPTT).
  • Epistaxis severity score ≥ 3 measured over the preceding three months, measured at the screening visit.
  • A requirement for 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 REMS® program, and be willing and able to comply with the requirements of the POMALYST REMS® program.
  • Females of childbearing potential (FCBP)† must adhere to the pregnancy testing schedule mandated by the POMALYST REMS® program.
  • Ability to understand and sign informed consent.

†A female of childbearing poteintial is a sexually mature woman who:

  • has not undergone a hysterectomy or bilateral oophorectomy; or
  • has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).

Exclusion Criteria: 

  • Women currently breastfeeding or pregnant.
  • Renal insufficiency, serum creatinine > 2.0 mg/dl.
  • Hepatic insufficiency, bilirubin > 2.0 (or > 4.0 in the setting of a prior clinical or genetic diagnosis of Gilbert's syndrome) or transaminases > 3.0 x normal.
  • Prior treatment with thalidomide or other Immunomodulatory imide drugs (IMiDs) within previous 6 months.
  • Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks.
  • The use of octreotide or estrogens within the previous month.
  • History of prior unprovoked thromboembolism confirmed by venous ultrasound or other imaging modalities.
  • Known peripheral neuropathy, confirmed by neurologic consultation.
  • Known underlying hypoproliferative anemia (i.e., myelodysplasia, aplastic anemia).
  • Currently enrolled in other interventional trials.
  • Known hypersensitivity to thalidomide or lenalidomid.

 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Vivek Iyer, M.D., M.P.H.

Closed for enrollment

More information

Publications

Publications are currently not available
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CLS-20484521

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