What side effects are associated with this type of intervention?

Common treatments for Polycythemia Vera, such as ruxolitinib, hydroxyurea, and erythropoiesis-stimulating agents, have various side effects. Ruxolitinib can cause increased rates of herpes zoster infection, nonmelanoma skin cancer, and anemia. Hydroxyurea may lead to cytopenias, mucocutaneous ulcers, diarrhea, peripheral neuropathy, and potential teratogenicity. Erythropoiesis-stimulating agents, while less commonly used in PV, can cause hypertension, thrombosis, and headaches. These side effects should be carefully considered when choosing a treatment plan.

What prior FDA approvals exist?

The FDA has approved several treatments for Polycythemia Vera, primarily focusing on reducing hematocrit levels and managing symptoms. Hydroxyurea, a cytoreductive agent, is commonly used to control blood cell production. Ruxolitinib, a JAK1/JAK2 inhibitor, is another FDA-approved drug that targets the JAK-STAT pathway, reducing the overproduction of blood cells. Additionally, interferon-alpha has been used to modulate the immune system and reduce erythropoiesis. These treatments aim to manage the disease by controlling blood cell proliferation and alleviating symptoms, similar to the potential mechanism of action of SLN124, which likely involves modulation of iron metabolism or erythropoiesis.

What other types of research exist?

Promising alternatives to SLN124 for Polycythemia Vera patients include JAK2 inhibitors like ruxolitinib and fedratinib, which target the JAK/STAT pathway involved in PV. Additionally, BCL2 inhibitors such as venetoclax, used in combination therapies for myeloid malignancies, may offer potential benefits. These treatments are under investigation and have shown efficacy in related hematologic conditions.

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RNAi

SLN124 for Polycythemia Vera (SLN Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Silence Therapeutics plc

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 239

Summary

This trial is testing a new drug called SLN124 in adults with Polycythemia Vera, a condition where the body makes too many red blood cells. The study aims to see if SLN124 is safe and effective. Researchers will also look at how the drug moves through and affects the body.

Who is the study for?

Adults with Polycythemia Vera (PV) who've had a skin check in the last 6 months, meet specific PV diagnostic criteria, and have stable phlebotomy history. They must not be on or have recently used certain drugs for PV, agree to contraception, and have an acceptable level of physical functioning.

What is being tested?

The study is testing SLN124 against a placebo in patients with PV. It starts with an open-label phase where everyone gets SLN124 to find safe doses, followed by a randomized phase where participants are blindly given either SLN124 or a placebo to compare effects.

What are the potential side effects?

Possible side effects include reactions at the injection site due to intolerance to components in SLN124 or GalNAc conjugates. Other risks may involve liver issues and blood disorders since eligibility excludes those with significant liver disease or abnormal blood counts.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 239

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 239

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 239 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of treatment-emergent adverse events (AEs)

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Phase 2 Blinded SLN124Experimental Treatment1 Intervention

SLN124 for subcutaneous (s.c.) injection

Group II: Phase 1 open-label SLN124Experimental Treatment1 Intervention

SLN124 for subcutaneous (s.c.) injection

Group III: Phase 2 Blinded PlaceboPlacebo Group1 Intervention

Sodium chloride for s.c. injection

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SLN124

2020

Completed Phase 1

~70

Do I qualify?Apply below to find out

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

Common treatments for Polycythemia Vera (PV) often target the overproduction of red blood cells, a hallmark of the disease. These treatments include phlebotomy, which reduces blood volume and hematocrit levels, and medications like hydroxyurea, which suppresses bone marrow activity to decrease red blood cell production. Additionally, JAK2 inhibitors, such as ruxolitinib, target the JAK-STAT pathway involved in cell proliferation. Emerging treatments like SLN124 likely focus on modulating iron metabolism or erythropoiesis, aiming to reduce excessive red blood cell production by altering the body's iron handling or the maturation of red blood cells. These mechanisms are crucial for PV patients as they help manage symptoms, reduce the risk of thrombotic events, and improve overall quality of life.

Polycythemia vera: the natural history of 1213 patients followed for 20 years. Gruppo Italiano Studio Policitemia.Acute myocardial infarction following sequential multi-vessel occlusion in a case of polycythemia vera.