Pomalidomide for the Treatment of Bleeding in HHT

(PATH-HHT Trial)

Recruiting at16 trial locations

Overseen by Keith McCrae, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: The Cleveland Clinic

Prior Safety Data

Breakthrough Therapy

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?

This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or 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.

Research Team

Keith McCrae, MD

Principal Investigator

The Cleveland Clinic

Eligibility Criteria

Inclusion Criteria

Age > 18 years

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.

You have been diagnosed with a condition called HHT (Hereditary Hemorrhagic Telangiectasia) based on specific criteria.

Exclusion Criteria

You have received bevacizumab treatment (either through injections or nasal spray) within the last 6 weeks.

You have taken octreotide or oral estrogen medications in the last month.

You have experienced a specific type of skin rash called erythema nodosum while taking thalidomide or similar medications.

Treatment Details

Interventions

• Placebo (Other)
• Pomalidomide (Immunomodulatory Agent)

Participant Groups

Group I: Pomalidomide Experimental Treatment 1 Intervention

Oral Pomalidomide will be provided as a capsule at 4 mg/day dose. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks.

Group II: Placebo Placebo Group 1 Intervention

A placebo matching the study drug will be provided as a capsule. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks.

Pomalidomide is already approved in Canada, Japan for the following indications:

Approved in Canada as Pomalyst for:

• Multiple myeloma
• Kaposi's sarcoma

Approved in Japan as Imnovid for:

• Multiple myeloma

Who Is Running the Clinical Trial?

The Cleveland Clinic

Lead Sponsor

Trials

1,072

Recruited

1,377,000+

David Peter

The Cleveland Clinic

Chief Medical Officer

MD, board-certified in Hospice and Palliative Medicine

Tomislav Mihaljevic

The Cleveland Clinic

Chief Executive Officer since 2018

MD from University of Zagreb School of Medicine

RTI International

Collaborator

Trials

201

Recruited

942,000+

Dr. Anuja Purohit

RTI International

Chief Medical Officer

MD from Duke University School of Medicine

Tim J. Gabel

RTI International

Chief Executive Officer since 2022

Adjunct appointments at University of Wyoming and UNC Gillings School of Global Public Health