Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia Longitudinal Assessment Study

Completed

Conditions studied: Hereditary Hemorrhagic Telangiectasia (HHT)

In brief

This is a multicenter U.S. longitudinal study evaluating patients with hereditary hemorrhagic telangiectasia who participated in the PATH-HHT clinical trial of pomalidomide for the treatment of HHT. This study is a longitudinal assessment of safety and effectiveness of pomalidomide in HHT in clinical trial participants following completion of the double-blind, placebo-controlled study.

Key facts

Study ID

NCT07018401

Run by

Massachusetts General Hospital

People needed

62

Starts

2024-05-01

Expected to finish

2026-06-01

Last updated by the study team

2026-07-10

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• A clinical diagnosis of HHT as defined by the Curacao criteria
• Age > 18 years
• Platelet count ≥ 100 x 109/L prior to pomalidomide initiation
• WBC ≥ 2.5 x 109/L prior to pomalidomide initiation
• INR ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT) 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 month
• A requirement for anemia, as determined by local laboratory 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 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
• Prior enrollment on PATH-HHT study (NCT03910244)
• A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) 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).

You may not qualify if…

• Women currently breast feeding 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 > 3.0x normal
• Prior treatment with thalidomide or other non-pomalidomide immunomodulatory imide drugs (IMiDs) within previous 6 months
• Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks
• Prior treatment with pazopanib 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 drug 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, unless there has been a colonoscopy with normal (negative) results, or in which the patient has had no more than 5 small (in the opinion of the gastroenterologist) colonic polyps completely removed within the preceding 18 months
• Anything that in the investigator's opinion is likely to interfere with completion of the study

Where it is running

• University of California-San Diego — San Diego, California, United States
• University of California-San Francisco — San Francisco, California, United States
• University of Florida — Gainesville, Florida, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Mayo Clinic — Rochester, Minnesota, United States
• University of North Carolina, Chapel HIll — Chapel Hill, North Carolina, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• University of Utah — Salt Lake City, Utah, United States
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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