P1101 for Essential Thrombocythemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called Ropeginterferon alfa-2b-njft (P1101) for individuals with essential thrombocythemia, a condition where the body produces too many platelets, causing blood clotting issues. The study aims to evaluate the treatment's effectiveness and safety. It targets adults needing treatment due to symptoms like night sweats, fatigue, or blood flow problems that don't improve with aspirin. Those who have found other treatments like HU ineffective or intolerable may also be suitable candidates. Participants should have a high platelet count and be prepared to follow study instructions. As a Phase 2 trial, this research focuses on assessing the treatment's efficacy in an initial, smaller group of people.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications. However, if you have been on hydroxyurea (HU), you must stop it at least 7 days before starting the trial medication.
Is there any evidence suggesting that Ropeginterferon alfa-2b-njft (P1101) is likely to be safe for humans?
Research has shown that ropeginterferon alfa-2b-njft, also known as P1101, is generally well-tolerated by patients. In one study, only 5.5% of patients stopped treatment due to unwanted effects, a lower rate than with some other treatments. Serious side effects related to the treatment were rare, affecting only 2.2% of participants.
Real-world data indicates that over several years, patients with essential thrombocythemia, a blood disorder, responded well to the treatment. Importantly, the FDA has already approved ropeginterferon alfa-2b-njft for treating another similar blood disorder called polycythemia vera, suggesting it is safe to use.12345Why do researchers think this study treatment might be promising for essential thrombocythemia?
Ropeginterferon alfa-2b (P1101) is unique because it offers a novel approach to managing essential thrombocythemia by using a long-acting interferon. Traditional treatments like hydroxyurea and anagrelide primarily focus on reducing platelet counts but can come with significant side effects and frequent dosing. In contrast, P1101 is administered as a pre-filled syringe injection every two weeks, potentially improving convenience and compliance for patients. Researchers are excited about P1101 because it may not only control platelet levels effectively but also modify the underlying disease process with fewer side effects, offering a promising alternative for long-term disease management.
What evidence suggests that Ropeginterferon alfa-2b-njft (P1101) might be an effective treatment for Essential Thrombocythemia?
Research has shown that ropeginterferon alfa-2b-njft (P1101), the treatment under study in this trial, effectively treats essential thrombocythemia, a condition where the body produces too many platelets. In one study, 42.9% of patients experienced lasting improvement in their symptoms at 9 and 12 months, meaning their symptoms improved and remained stable for a significant time. Ropeginterferon also outperformed anagrelide, another treatment, particularly in high-risk patients. Therefore, strong evidence supports the effectiveness of this treatment for essential thrombocythemia.23467
Who Is on the Research Team?
Oleh Zagrijtschuk, MD, PhD
Principal Investigator
PharmaEssentia Corporation
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Ropeginterferon alfa-2b-njft (P1101) for the treatment of Essential Thrombocythemia
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extension
Participants may continue receiving treatment in an extension period
What Are the Treatments Tested in This Trial?
Interventions
- Ropeginterferon alfa-2b-njft (P1101)
Find a Clinic Near You
Who Is Running the Clinical Trial?
PharmaEssentia
Lead Sponsor