Study to Assess SLN124 in Patients With Polycythemia Vera

Last updated: April 19, 2024
Sponsor: Silence Therapeutics plc
Overall Status: Active - Recruiting

Phase

1/2

Condition

Leukemia (Pediatric)

Bone Marrow Disorder

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

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 discontinued fromany prior cytoreductive therapy for at least 24 weeks before dosing and have recoveredfrom 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 12weeks before dosing and with no planned change in dose.
  • Patients must have had a dermatological examination within 6 months prior toscreening.
  • Must have an Eastern Cooperative Oncology Group score of 0, 1, or 2.

Exclusion

Exclusion Criteria:

  • 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.
  • Clinically significant co-morbidities
  • Biochemical and hematological parameters:
  1. Biochemical evidence of significant liver disease during screening
  2. Hematological parameters at screening as follows: platelets 1,000,000/µL; orwhite blood cell (WBC) count > 25,000/µL; or peripheral blasts < 1%.

Study Design

Total Participants: 65
Treatment Group(s): 2
Primary Treatment: SLN124
Phase: 1/2
Study Start date:
January 26, 2023
Estimated Completion Date:
June 30, 2025

Connect with a study center

  • Pindara Private Hospital

    Benowa, Queensland 4217
    Australia

    Active - Recruiting

  • Ashford Cancer Centre Research

    Kurralta Park, South Australia 5037
    Australia

    Active - Recruiting

  • Alfred Health

    Melbourne, Victoria 3004
    Australia

    Active - Recruiting

  • Peter MacCallum Cancer Centre

    Melbourne, Victoria 3000
    Australia

    Active - Recruiting

  • Epworth HealthCare

    Richmond, Victoria 3121
    Australia

    Active - Recruiting

  • Linear Clinical Research

    Nedlands, Western Australia 6009
    Australia

    Active - Recruiting

  • MHAT Dr Nikola Vasiliev AD

    Kyustendil, 2500
    Bulgaria

    Active - Recruiting

  • Medical Centre Leo Clinic EOOD

    Plovdiv, 4003
    Bulgaria

    Active - Recruiting

  • Hospital Sultanah Aminah

    Johor Bahru, Johor 80100
    Malaysia

    Active - Recruiting

  • Hospital Tengku Ampuan Afzan

    Kuantan, Pahang 25100
    Malaysia

    Active - Recruiting

  • Hospital Umum Sarawak

    Kuching, Sarawak 93586
    Malaysia

    Active - Recruiting

  • Hospital Ampang

    Ampang, Selangor 68000
    Malaysia

    Active - Recruiting

  • Hospital Sultanah Nur Zahirah

    Kuala Terengganu, Terengganu 20400
    Malaysia

    Active - Recruiting

  • Uniwersyteckie Centrum Klinlczne

    Gdańsk,
    Poland

    Active - Recruiting

  • PRATIA Hematologia Sp. z o. o.

    Katowice,
    Poland

    Active - Recruiting

  • Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie

    Lublin,
    Poland

    Active - Recruiting

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

    Skorzewo,
    Poland

    Active - Recruiting

  • Specjalistyczny Szpital Im Dra. A.

    Wałbrzych, 58-300
    Poland

    Active - Recruiting

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • Mount Sinai Hospital

    New York, New York 10029
    United States

    Active - Recruiting

  • Duke Cancer Institute

    Durham, North Carolina 27705
    United States

    Site Not Available

  • North Houston Cancer Clinics

    Huntsville, Texas 77340
    United States

    Completed

  • Renovatio Clinical

    The Woodlands, Texas 77380
    United States

    Active - Recruiting

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