What side effects are associated with this type of intervention?

Common treatments for Polycythemia Vera that reduce red blood cell production or hematocrit levels include hydroxyurea, ruxolitinib, and interferon alfa. Hydroxyurea can cause cytopenias, mucocutaneous ulcers, diarrhea, peripheral neuropathy, skin cancer, and potential teratogenicity. Ruxolitinib's side effects include cytopenias, dizziness, headache, fatigue, bruising, elevated serum cholesterol, and increased risk of infections. Interferon alfa may cause flu-like symptoms, fatigue, depression, and liver enzyme abnormalities.

What prior FDA approvals exist?

The FDA has approved several treatments for Polycythemia Vera that aim to reduce red blood cell production or hematocrit levels. Hydroxyurea is commonly used to manage PV by decreasing the production of red blood cells. Another FDA-approved drug is ruxolitinib, a JAK1/JAK2 inhibitor, which helps control hematocrit levels and reduce spleen size. These treatments, like sapablursen, aim to manage the symptoms and complications associated with PV by targeting the overproduction of blood cells.

What other types of research exist?

Promising novel alternatives to Sapablursen for Polycythemia Vera patients include Ruxolitinib and Interferon-alpha2. Ruxolitinib is a JAK2 inhibitor that has shown efficacy in reducing hematocrit levels and controlling symptoms in PV patients, especially those resistant or intolerant to hydroxyurea. Interferon-alpha2 gradually lowers elevated cell counts and maintains them within the normal range, offering a stable long-term treatment option. Both agents have demonstrated effectiveness in clinical trials and are approved for use in PV.

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Antisense Oligonucleotide

Sapablursen for Polycythemia Vera

Phase 2

Waitlist Available

Research Sponsored by Ionis Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants do not need to be on cytoreductive therapy and do not need to have been previously treated with cytoreductive therapy. If the patient was previously on cytoreductive therapy it must have been discontinued at least 3 months prior to Screening, with all associated AEs resolved. If the patient is currently on cytoreductive therapy they must be on a stable dose of hydroxyurea, recombinant or PEGylated interferon, or ruxolitinib for at least 3 months prior to Screening.

Be older than 18 years old

Must not have

Meets criteria for post-polycythemia vera myelofibrosis (PPV-MF) as defined by the International Working Group- Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)

Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests sapablursen, an injectable medication, on patients with polycythemia vera to see if it can reduce the need for regular blood removal and improve their quality of life.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects of sapablursen are not listed here, similar medications may cause reactions at injection sites, liver problems, fatigue, potential immune system impacts leading to increased infection risk, and possible changes in blood counts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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

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I do not have an active infection needing drugs or an ongoing COVID-19 infection.

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I do not have an active infection with HIV, hepatitis C, or hepatitis B.

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I experience moderate to severe pain in my spleen area.

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I have a diagnosed immune system disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Sapablursen Dose Level 2Experimental Treatment1 Intervention

Sapablursen will be administered by SC injection every 4 weeks

Group II: Sapablursen Dose Level 1Experimental Treatment1 Intervention

Sapablursen will be administered by SC injection every 4 weeks.

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Polycythemia Vera (PV) aim to reduce red blood cell production and hematocrit levels to prevent thrombohemorrhagic complications. Hydroxyurea works by inhibiting DNA synthesis, thus reducing the proliferation of red blood cells. Interferon-alpha modulates the immune system to suppress the abnormal blood cell production. Ruxolitinib, a JAK1/2 inhibitor, targets the JAK-STAT pathway, which is often mutated in PV, thereby reducing the overproduction of blood cells. Sapablursen, an investigational drug, is being studied for its potential to reduce the need for phlebotomy and improve quality of life by targeting mechanisms that control red blood cell production. These treatments are crucial for PV patients as they help manage symptoms, reduce the risk of complications, and improve overall quality of life.