Sapablursen for Polycythemia Vera

Not currently recruiting at 47 trial locations

Overseen By Ionis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ionis Pharmaceuticals, Inc.

Must be taking: Hydroxyurea, Interferon, Ruxolitinib

Disqualifiers: Myelofibrosis, Splenic pain, Bleeding, Immunodeficiency, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 18

Duration: 37 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests sapablursen, a new treatment for polycythemia vera, a condition where the body produces too many red blood cells. The goal is to determine if this treatment can reduce the need for blood draws and improve quality of life. Participants will receive sapablursen injections every four weeks, with two different dose levels under evaluation. Individuals diagnosed with polycythemia vera who frequently require blood removals might be suitable for this trial. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but if you are on cytoreductive therapy, you must be on a stable dose of hydroxyurea, recombinant or PEGylated interferon, or ruxolitinib for at least 3 months before joining. If you were previously on cytoreductive therapy, it must have been stopped at least 3 months before the trial.

Is there any evidence suggesting that sapablursen is likely to be safe for humans?

Research shows that sapablursen is generally safe for people. Earlier studies found it well-tolerated by patients with polycythemia vera, a condition where the body produces too many red blood cells. These studies did not identify any major safety issues.

Although the treatment remains under investigation, results so far suggest that serious side effects are uncommon. This is encouraging for those considering joining the trial. However, discussing any concerns with a healthcare provider before joining a clinical trial is always important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for polycythemia vera?

Researchers are excited about Sapablursen for polycythemia vera because it offers a new way of tackling the condition. Unlike traditional treatments that often focus on reducing blood cell production or volume directly, Sapablursen targets the underlying molecular pathways involved in the disease. It is administered via a subcutaneous injection every four weeks, which could offer more convenience compared to some existing treatments that require more frequent dosing or more invasive procedures. This novel approach could lead to better management of polycythemia vera with potentially fewer side effects.

What evidence suggests that sapablursen might be an effective treatment for polycythemia vera?

Research suggests that sapablursen may help people with polycythemia vera by reducing the need for blood draws to lower red blood cell levels. In this trial, participants will receive sapablursen at different dose levels. Studies have shown that patients experienced a decrease in spleen size and maintained red blood cell levels low enough to avoid these blood draws. These changes suggest that sapablursen could ease living with polycythemia vera by reducing symptoms and improving quality of life. While more information is needed to confirm these benefits, early results appear promising. ^{2 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with polycythemia vera who depend on regular blood removal (phlebotomy) to manage their condition. They must meet specific diagnostic criteria and either have not been on cytoreductive therapy or be on a stable dose for at least 3 months. People can't join if they have severe spleen issues, certain other diseases like HIV or hepatitis, recent major surgery, cancer within the last 5 years (with some exceptions), or bleeding/coagulation disorders.

Inclusion Criteria

I haven't had or don't currently need treatment to reduce my blood cell counts. If I was treated, it ended 3 months ago.

I stopped my cytoreductive therapy 3 months ago or have been on a stable dose for the same duration.

Participant must be phlebotomy dependent

Exclusion Criteria

I have been diagnosed with PPV-MF according to IWG-MRT standards.

I haven't had any serious bleeding or blood clotting issues in the last month.

I haven't had cancer in the last 5 years, except for certain skin cancers, cervical carcinoma in situ, or treated non-metastatic prostate cancer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 7 weeks

Treatment

Participants receive sapablursen by subcutaneous injection every 4 weeks for 37 weeks

37 weeks

9 visits (in-person)

Treatment Extension

Participants continue to receive sapablursen by subcutaneous injection every 4 weeks for an additional 36 weeks

36 weeks

9 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

13 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Sapablursen

Trial Overview The study tests sapablursen's effectiveness in reducing the need for phlebotomies and improving life quality in patients with polycythemia vera. It aims to see if this treatment can help manage red blood cell levels better than current methods.

How Is the Trial Designed?

Group I: Sapablursen Dose Level 2 Experimental Treatment 1 Intervention

Sapablursen will be administered by SC injection every 4 weeks

Group II: Sapablursen Dose Level 1 Experimental Treatment 1 Intervention

Sapablursen will be administered by SC injection every 4 weeks.

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11857034/

Novel Therapies for Polycythemia Vera 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, ...

2. mpncancerconnection-org.clinicaltrialconnect.com mpncancerconnection-org.clinicaltrialconnect.com/trials/NCT05143957

A Study to Evaluate Sapablursen (Formerly ISIS 702843 ... The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments ...

3. link.springer.com link.springer.com/article/10.1007/s12672-025-03703-9

Advances in polycythemia vera treatment with targeted ... Among evaluable patients, spleen volume reductions of ≥ 35% and sustained absence of phlebotomy eligibility were achieved, supporting its ...

4. mdanderson.org mdanderson.org/patients-family/diagnosis-treatment/clinical-trials/clinical-trials-index/clinical-trials-detail.ID2021-0897.html

A Phase 2a, Randomized, Open-Label Study to Evaluate ... The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments.

5. clinicaltrials.eu clinicaltrials.eu/inn/sapablursen/

Sapablursen – Application in Therapy and Current Clinical ... This article will provide an overview of the ongoing research on Sapablursen, focusing on its use in treating Polycythemia Vera and Beta Thalassemia Intermedia.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12559549/

Advances in polycythemia vera treatment with targeted ... Overall, Sapablursen was safe ... Cytoreductive therapy in younger adults with polycythemia vera: a meta-analysis of safety and outcomes.

7. withpower.com withpower.com/trial/phase-2-polycythemia-vera-11-2021-34b29

Sapablursen for Polycythemia Vera · Info for Participants Trial Overview The study tests sapablursen's effectiveness in reducing the need for phlebotomies and improving life quality in patients with polycythemia vera.