Pomalidomide for the Treatment of Bleeding in HHT

• Register: ClinicalTrials.gov
• Last refreshed on: 6 May 2024
• Main ID: NCT03910244
• Date of registration: 08/04/2019
• Prospective Registration: Yes
• Primary sponsor: The Cleveland Clinic
• Public title: Pomalidomide for the Treatment of Bleeding in HHT PATH-HHT
• Scientific title: Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia
• Date of first enrolment: October 17, 2019
• Target sample size: 145
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03910244
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Keith McCrae, MD
• Affiliation: The Cleveland Clinic

Key inclusion & exclusion criteria

Inclusion Criteria:

1. A clinical diagnosis of HHT as defined by the Curacao criteria

2. Age > 18 years

3. Platelet count = 100,000/µl

4. WBC = 2,500/µl

5. INR = 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT or PTT per
local laboratory designation) by local laboratory criteria (except for patients on a
stable dose of warfarin or direct oral anticoagulants)

6. Epistaxis severity score = 3 measured over the preceding three months, measured at the
screening visit

7. A requirement for anemia, as determined by local laboratory hemoglobin assessment and
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

8. 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.

9. Ability to understand and sign informed consent

10. 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:

1. Women currently breast feeding

2. Renal insufficiency, serum creatinine > 2.0 mg/dl

3. 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

4. Prior treatment with thalidomide or other imid drugs within previous 6 months

5. Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks*

6. Prior treatment with pazopanib within previous 6 weeks*

7. The use of octreotide or oral estrogens within the previous month*

8. History of prior unprovoked thromboembolism confirmed by venous ultrasound or other
imaging modalities

9. Peripheral neuropathy, confirmed by neurologic consultation

10. Known underlying hypoproliferative anemia (i.e. myelodysplasia, aplastic anemia)

11. Currently enrolled in other interventional trials

12. Known hypersensitivity to thalidomide or lenalidomide.

13. The development of erythema nodosum if characterized by a desquamating rash while
taking thalidomide or similar drugs.

14. 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

15. Anything that in the investigator's opinion is likely to interfere with completion of
the study

- * Use of these treatments is not permitted during study participation.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Telangiectasia, Hereditary Hemorrhagic

Intervention(s)

Drug: Pomalidomide Oral Product

Drug: Placebo oral capsule

Primary Outcome(s)

Change in Epistaxis Severity Score [Time Frame: Baseline to 24 weeks]

Secondary Outcome(s)

Amount of Packed red blood cell Transfusions [Time Frame: Baseline to 24 weeks]

Change in PROMIS Satisfaction with Social Roles and Activities [Time Frame: Baseline to 24 weeks, Baseline to 12 weeks and Baseline to 28 weeks]

Change in average weekly epistaxis duration [Time Frame: Baseline to 24 weeks, Baseline to 12 weeks and Baseline to 28 weeks]

Change in HHT-Specific QOL [Time Frame: Baseline to 24 weeks, Baseline to 12 weeks and Baseline to 28 weeks]

Amount of Parenteral Iron Administration [Time Frame: Baseline to 24 weeks]

Secondary ID(s)

133646

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

RTI International