SLN124 for Polycythemia Vera
(SLN Trial)

Recruiting in Palo Alto (17 mi)

+21 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Silence Therapeutics plc

Must be taking: Cytoreductive therapy

Must not be taking: Investigational drugs

Disqualifiers: Thrombosis, Major bleeding, Myelofibrosis, others

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

If you are not on cytoreductive therapy, you must have stopped any previous cytoreductive medications at least 24 weeks before starting the trial. If you are on cytoreductive therapy like hydroxyurea, interferon, busulfan, or ruxolitinib, you need to be on a stable dose for at least 12 weeks before starting the trial.

Eligibility Criteria

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.

Inclusion Criteria

I have seen a skin doctor in the last 6 months.

Suitable phlebotomy history

I have been diagnosed with polycythemia vera according to the latest criteria.

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Exclusion Criteria

History of intolerance to oligonucleotides, or GalNAc, or any component of SLN124

I have had major bleeding or needed a blood transfusion due to bleeding in the last 6 months.

Hematological parameters at screening as follows: platelets 1,000,000/µL; or white blood cell (WBC) count > 25,000/µL; or peripheral blasts < 1%

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Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Dose Escalation

Participants receive SLN124 in an open-label dose escalation to assess safety, tolerability, and pharmacokinetics

8 weeks

Randomized Double-blind Phase

Participants are randomized to receive either SLN124 or placebo in a double-blind manner to assess efficacy and pharmacodynamics

16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

Placebo (Other)
SLN124 (RNAi)

Trial OverviewThe 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.

Participant Groups

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