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.
  • Platelet count ≥ 100,000/µl 4. WBC ≥ 2,500/µl.
  • INR ≤ 1.4 and normal 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 preceding 24 weeks.
  • 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

Exclusion Criteria: 

  • Women currently breast feeding.
  • Renal insufficiency, serum creatinine > 2.0 mg/dl.
  • GI bleeding thought to be related to hepatic insufficiency, bilirubin > 2.0 or transaminases > 3.0 x normal.
  • Prior treatment with thalidomide or other imid drugs within previous 6 months.
  • Prior treatment with bevacizumab (systemic or nasal) within previous 8 weeks.
  • The use of octreotide or estrogens within the previous month.
  • History of prior thromboembolism confirmed by venous ultrasound or other imaging modalities.
  • 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 lenalidomide.
  • The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs. 
  • Known SMAD-4 mutation.
  • Anything that in the investigator's opinion is likely to interfere with completion of the study.

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 Contact

Rochester, Minn.

Mayo Clinic principal investigator

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

Closed for enrollment

Contact information:

Sue Ann Donlinger

5072849259

donlinger.sueann@mayo.edu

More information

Publications

Publications are currently not available