Study to Assess SLN124 in Patients With Polycythemia Vera

Last updated: December 17, 2025
Sponsor: Silence Therapeutics plc
Overall Status: Active - Not Recruiting

Phase

1/2

Condition

Bone Marrow Disorder

Leukemia (Pediatric)

Red Blood Cell Disorders

Treatment

SLN124

Placebo

Clinical Study ID

NCT05499013
SLN124-004
SANRECO
  • Ages > 18
  • All Genders

Study Summary

This is a Phase 1/2, multicenter study with an open-label dose escalation followed by a randomized placebo controlled and double-blind phase of SLN124 in adult patients with Polycythemia Vera (PV) to assess the safety, tolerability, efficacy, pharmacokinetic (PK), and Pharmacodynamic (PD) response of SLN124.

Eligibility Criteria

Inclusion

Phase 1 and Phase 2

Inclusion Criteria:

  • Male and female patients aged 18 years or older.

  • A confirmed diagnosis of PV according to the revised 2016 World Health Organizationcriteria:

  • Suitable phlebotomy history

  • Must agree to adhere to appropriate contraception requirements

  • Patients who are not receiving cytoreductive therapy must have been discontinuedfrom any prior cytoreductive therapy for at least 24 weeks before dosing and haverecovered from any adverse events due to cytoreductive therapy.

  • Patients receiving cytoreductive therapy with hydroxyurea, interferon, busulfan orruxolitinib must have received a stable dose of cytoreductive therapy for at least 12 weeks before dosing and with no planned change in dose.

  • Patients must have had a dermatological examination within 28 weeks prior to dosing.

  • Must have an Eastern Cooperative Oncology Group score of 0, 1, or 2.

Exclusion

Exclusion Criteria:

Phase 1 and Phase 2

  • Drug intolerance:
  1. History of intolerance to oligonucleotides, or GalNAc, or any component ofSLN124.

  2. History of intolerance to s.c. injections.

  • Clinically significant thrombosis (e.g., deep vein thrombosis or splenic veinthrombosis) within 12 weeks of screening.

  • History of major bleeding events and/or a requirement for blood transfusion therapyowing to bleeding in the last 6 months prior to screening.

  • Meets the criteria for post-PV myelofibrosis as defined by the International WorkingGroup-Myeloproliferative Neoplasms Research and Treatment

  • Any investigational drug less than 6 weeks prior to the first dose of study drug ornot recovered from effects of prior administration of any investigational agent.

  • Any investigational or marketed product using GalNAc targeting less than 48 weeksprior to administration of any investigational agent (excludes patients with PV whoparticipated in Phase 1 of this study).

  • Clinically significant co-morbidities

  • Biochemical and hematological parameters:

  1. Biochemical evidence of significant liver disease during screening

  2. Phase 1: Hematological parameters at screening as follows: platelets > 1,000,000/µL; or white blood cell (WBC) count > 25,000/µL; or peripheral blasts > 1%. b. Phase 2: Hematological parameters at screening as follows: platelets > 1,000,000/µL; or WBC count > 30,000/µL; or peripheral blasts > 1%.

Study Design

Total Participants: 69
Treatment Group(s): 2
Primary Treatment: SLN124
Phase: 1/2
Study Start date:
January 26, 2023
Estimated Completion Date:
March 31, 2030

Connect with a study center

  • Pindara Private Hospital

    Benowa, Queensland 4217
    Australia

    Site Not Available

  • Ashford Cancer Centre Research

    Kurralta Park, South Australia 5037
    Australia

    Site Not Available

  • Alfred Health

    Melbourne, Victoria 3004
    Australia

    Site Not Available

  • Peter MacCallum Cancer Centre

    Melbourne, Victoria 3000
    Australia

    Active - Recruiting

  • Epworth HealthCare

    Richmond, Victoria 3121
    Australia

    Site Not Available

  • Linear Clinical Research

    Nedlands, Western Australia 6009
    Australia

    Site Not Available

  • Research Site

    Benowa 2176127,
    Australia

    Site Not Available

  • Research Site

    Hobart 2163355,
    Australia

    Site Not Available

  • Research Site

    Kurralta Park 8348005,
    Australia

    Site Not Available

  • Research Site

    Melbourne 2158177,
    Australia

    Site Not Available

  • Research Site 2

    Melbourne 2158177,
    Australia

    Site Not Available

  • Linear Clinical Research

    Nedlands 2064874,
    Australia

    Site Not Available

  • Research Site

    Richmond 2151649,
    Australia

    Site Not Available

  • MHAT Dr Nikola Vasiliev AD

    Kyustendil, 2500
    Bulgaria

    Site Not Available

  • Medical Centre Leo Clinic EOOD

    Plovdiv, 4003
    Bulgaria

    Site Not Available

  • Research Site

    Plovdiv 728193,
    Bulgaria

    Site Not Available

  • Research Site 2

    Plovdiv 728193,
    Bulgaria

    Site Not Available

  • Research Site

    Toronto 6167865,
    Canada

    Site Not Available

  • Research Site

    Freiburg im Breisgau 2925177,
    Germany

    Site Not Available

  • Research Site

    Hanover 2910831,
    Germany

    Site Not Available

  • Research Site

    Alessandria 3183299,
    Italy

    Site Not Available

  • Research Site

    Meldola 3173635,
    Italy

    Site Not Available

  • Hospital Sultanah Aminah

    Johor Bahru, Johor 80100
    Malaysia

    Site Not Available

  • Hospital Tengku Ampuan Afzan

    Kuantan, Pahang 25100
    Malaysia

    Site Not Available

  • Hospital Umum Sarawak

    Kuching, Sarawak 93586
    Malaysia

    Site Not Available

  • Hospital Ampang

    Ampang, Selangor 68000
    Malaysia

    Site Not Available

  • Hospital Sultanah Nur Zahirah

    Kuala Terengganu, Terengganu 20400
    Malaysia

    Site Not Available

  • Research Site

    Johor Bahru 1732752,
    Malaysia

    Site Not Available

  • Research Site

    Kuala Terengganu 1734705,
    Malaysia

    Site Not Available

  • Research Site

    Kuantan 1735227,
    Malaysia

    Site Not Available

  • Research Site

    Kuching 1735634,
    Malaysia

    Site Not Available

  • Research Site

    Gdansk 3099434,
    Poland

    Site Not Available

  • Uniwersyteckie Centrum Klinlczne

    Gdańsk,
    Poland

    Site Not Available

  • PRATIA Hematologia Sp. z o. o.

    Katowice,
    Poland

    Site Not Available

  • Research Site

    Katowice 3096472,
    Poland

    Site Not Available

  • Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie

    Lublin,
    Poland

    Site Not Available

  • Research Site

    Lublin 765876,
    Poland

    Site Not Available

  • Centrum Medyczne Pratia Poznan Sp. z o. o.

    Skorzewo,
    Poland

    Site Not Available

  • Specjalistyczny Szpital Im Dra. A.

    Wałbrzych, 58-300
    Poland

    Site Not Available

  • Research Site

    Barcelona 3128760,
    Spain

    Site Not Available

  • Research Site

    Madrid 3117735,
    Spain

    Site Not Available

  • Research Site

    Hammond 4326868, Louisiana 4331987 70403
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Mount Sinai Hospital

    New York, New York 10029
    United States

    Site Not Available

  • Research Site

    Buffalo 5110629, New York 5128638 14203
    United States

    Site Not Available

  • Research Site

    New York 5128581, New York 5128638 10029
    United States

    Site Not Available

  • Duke Cancer Institute

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Research Site

    Columbus 4509177, Ohio 5165418 43210
    United States

    Site Not Available

  • North Houston Cancer Clinics

    Huntsville, Texas 77340
    United States

    Site Not Available

  • Renovatio Clinical

    The Woodlands, Texas 77380
    United States

    Site Not Available

  • Research Site

    Huntsville 4699540, Texas 4736286 77340
    United States

    Site Not Available

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