Clinical Trials

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.