P1101 for Essential Thrombocythemia

Recruiting in Palo Alto (17 mi)

+41 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: PharmaEssentia

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing a new medication called Ropeginterferon alfa-2b-njft (P1101) for adults with Essential Thrombocythemia, a blood disorder. The medication helps the body control blood cell production to prevent the disease from worsening. Interferons have been studied in similar conditions for over 30 years, but earlier versions had safety and tolerability issues.

Eligibility Criteria

Adults diagnosed with Essential Thrombocythemia (ET) as per WHO criteria, with a platelet count over 450 × 10^9/L and good liver and kidney function. Participants must be at least 18 years old, agree to use birth control, and not breastfeed during the study. Those previously treated with ANA or HU are eligible but not if they've had poor responses or intolerance to interferon alfa therapy.

Inclusion Criteria

My liver is functioning well according to recent tests.

I haven't had treatment to reduce my blood cell counts, or I've only had hydroxyurea and/or anagrelide.

I have or have not been treated with ANA before, regardless of why I stopped.

+6 more

Exclusion Criteria

I am not allergic to IFN-α or its ingredients.

Pregnant or lactating females

I need someone legally authorized to make decisions for me.

+4 more

Participant Groups

The trial is testing Ropeginterferon alfa-2b-njft (P1101) in adults with ET to evaluate its effectiveness, safety, and how well patients tolerate it. This single-arm study means all participants will receive P1101 without comparison to another treatment.

1Treatment groups

Experimental Treatment

Group I: Ropeginterferon alfa-2b (P1101)Experimental Treatment1 Intervention

Pre-filled Syringe, Q2W, SC injection