Sapablursen for Polycythemia Vera
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called sapablursen for individuals with Polycythemia Vera (PV), a condition characterized by the overproduction of red blood cells. The trial aims to determine if sapablursen is safe and effective when combined with the current standard treatment. Participants will receive either sapablursen or a placebo for 32 weeks, followed by sapablursen for 124 weeks, to assess long-term effects. Those who rely on regular blood removal (phlebotomy) to manage their PV and have stable treatment routines may be suitable candidates for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.
Do I have to stop taking my current medications for the trial?
The trial does not specify if you need to stop taking your current medications. However, if you are on Cytoreduction therapy, you must be on a stable regimen at the start of the study.
Is there any evidence suggesting that sapablursen is likely to be safe for humans?
Research has shown that sapablursen is generally safe for people. Earlier studies found it was well-tolerated by patients with polycythemia vera, a condition where the body produces too many red blood cells. These studies did not report any major safety issues. Participants handled the treatment well, with no serious side effects linked to the drug. This suggests that sapablursen may be a safe option for those considering joining a clinical trial.12345
Why do researchers think this study treatment might be promising for Polycythemia Vera?
Sapablursen is unique because it targets polycythemia vera at the genetic level, a different approach compared to traditional treatments like phlebotomy, hydroxyurea, and interferon therapy. This investigational drug works by inhibiting the production of erythropoietin, the hormone responsible for red blood cell production, which is often elevated in patients with polycythemia vera. Researchers are excited about Sapablursen because it offers a potential treatment that not only manages symptoms but also addresses the underlying cause of the disease, potentially leading to more effective and lasting results.
What evidence suggests that sapablursen might be an effective treatment for Polycythemia Vera?
Research has shown that sapablursen may help treat polycythemia vera (PV). Earlier studies found that sapablursen reduced the need for blood withdrawals and improved patients' quality of life. It also helped control hematocrit levels, which are crucial for managing PV. Another study found that sapablursen increased a protein called hepcidin, which regulates iron in the body. In this trial, participants will receive either sapablursen or a placebo for 32 weeks, followed by an open-label period with sapablursen for 124 weeks. These findings suggest that sapablursen could effectively manage polycythemia vera.12346
Who Is on the Research Team?
Clinical Team
Principal Investigator
Deciphera Pharmaceuticals, LLC
Are You a Good Fit for This Trial?
This trial is for people diagnosed with polycythemia vera who need regular blood removal (phlebotomy), have stable organ function, and are either on a steady dose of cytoreduction therapy or not. Their hematocrit must be below 45% at the start.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Blinded Treatment
Participants receive either sapablursen or placebo for 32 weeks
Open-label Treatment
Participants receive sapablursen for an additional 124 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Sapablursen
Trial Overview
The study tests sapablursen, a new drug, versus placebo in addition to standard care for polycythemia vera. The trial has three parts: an initial blinded phase, an open-label phase, and a long-term extension lasting up to about 3 years.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Sapablursen (32 Weeks) - Sapablursen (124 Weeks Open-label)
Placebo (32 Weeks) - Sapablursen (124 Weeks Open-label)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ono Pharmaceutical Co., Ltd.
Lead Sponsor
Deciphera Pharmaceuticals, LLC
Industry Sponsor
Citations
1.
ashpublications.org
ashpublications.org/ashclinicalnews/news/8912/Sapablursen-Meets-Primary-Endpoint-for-TreatmentSapablursen Meets Primary Endpoint for Treatment of ...
Evaluation of the novel TMPRSS6 antisense inhibitor sapablursen for treatment of polycythemia vera: results of the imprssion clinical trial.
Evaluation of the novel TMPRSS6 antisense inhibitor ...
Evaluation of the novel TMPRSS6 antisense inhibitor sapablursen for treatment of polycythemia vera: Results of the imprssion clinical trial.
Novel Therapies for Polycythemia Vera - PMC - NIH
The primary end point (50% improvement in MPN-SAF TSS at 16 weeks) was nonsignificantly superior in the RUX arm (43.4% vs. 29.6%; P = .139). ... Another large, ...
NCT07429266 | A Study of Sapablursen Evaluating the ...
The purpose of this study is to evaluate the efficacy and safety of sapablursen when added on to current standard of care (SOC) for Polycythemia Vera (PV) ...
Press Release
... results from the Phase 2a IMPRSSION study of sapablursen in patients with polycythemia vera (PV) at the. 67th American Society of Hematology ...
6.
deciphera.com
deciphera.com/news/deciphera-announces-oral-presentation-positive-topline-results-phase-2a-study-sapablursenDeciphera Announces Oral Presentation of Positive ...
... ○ Results from Phase 2a IMPRSSION study demonstrate sapablursen significantly reduced phlebotomy rate, controlled hematocrit and ...
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