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

NCT07018401 | Completed FDA Drug

• 1 other identifier: 2022P000149
• Study Type: observational
• Target: 62
• Locations: 1 country
• Sites: 11

Brief Summary

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.

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)

Keywords

hereditary hemorrhagic telangiectasia; HHT; Osler-Weber-Rendu; pomalidomide; PATH-HHT; PATH-HHT ATLAS

Outcome Measures

Primary Outcomes (1)

• Epistaxis Severity Score

  Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding

  12 months

Secondary Outcomes (5)

• Serum hemoglobin (g/dL)

  12 months

• Hematologic Support Score

  12 months

• Intravenous iron infusion (mg elemental iron)

  12 months

• Red cell transfusion (units of packed red blood cells)

  12 months

• Incidence of treatment-emergent adverse events (safety)

  12 months

Interventions

Pomalidomide DRUG

Oral pomalidomide up to 4 mg daily

Also known as: Pomalyst

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals with hereditary hemorrhagic telangiectasia and moderate-to-severe bleeding who previously enrolled in the PATH-HHT trial

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

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• The Cleveland Clinic: collaborator
• University of Utah: collaborator
• University of Pennsylvania: collaborator
• University of North Carolina, Chapel Hill: collaborator
• University of California, San Francisco: collaborator
• Mayo Clinic: collaborator
• University of California, San Diego: collaborator
• University of Florida: collaborator
• Medical College of Wisconsin: collaborator
• Johns Hopkins University: collaborator

Study Sites (11)

University of California-San Diego

San Diego, California, 92093, United States

Location

University of California-San Francisco

San Francisco, California, 94143, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21218, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of North Carolina, Chapel HIll

Chapel Hill, North Carolina, 27599, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

• Zhang E, Kasthuri RS, Parambil J, Prasad V, Iyer VN, Whitehead KJ, Hodges PG, Pishko AM, Conrad MB, Phelan D, Rodriguez-Lopez J, McCrae KR, Al-Samkari H. Pomalidomide for hereditary hemorrhagic telangiectasia: after trial longitudinal assessment study (PATH-HHT ATLAS). Blood Adv. 2026 Mar 10;10(5):1799-1808. doi: 10.1182/bloodadvances.2025018382.

  PMID: 41512167 DERIVED

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic

Interventions

pomalidomide

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Ellen Zhang, MD

  Stanford University

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Classical Hematologist, Clinical Investigator

Study Record Dates

• First Submitted: May 28, 2025
• First Posted: June 12, 2025
• Study Start: May 1, 2024
• Primary Completion: April 8, 2025
• Study Completion: June 1, 2026
• Last Updated: July 10, 2026

Record last verified: 2025-06

Locations

US (11)