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Study to Assess SLN124 in Patients With Polycythemia Vera (SLN)

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ClinicalTrials.gov Identifier: NCT05499013
Recruitment Status : Recruiting
First Posted : August 12, 2022
Last Update Posted : April 22, 2024
Sponsor:
Information provided by (Responsible Party):
Silence Therapeutics plc

Tracking Information
First Submitted Date  ICMJE July 29, 2022
First Posted Date  ICMJE August 12, 2022
Last Update Posted Date April 22, 2024
Actual Study Start Date  ICMJE January 26, 2023
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 10, 2022)
  • Incidence of treatment-emergent adverse events (AEs) [ Time Frame: Day 239 ]
    Safety and tolerability will be reported separately following open-label dose escalation phase and double-blind phase
  • Assessment of the number of phlebotomies at intervals [ Time Frame: 6 months prior to dosing to Day 239 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 10, 2022)
  • Pharmacokinetic: area under the plasma concentration (AUC) [ Time Frame: Day 127 ]
  • Pharmacokinetic: peak plasma concentration (Cmax) [ Time Frame: Day 127 ]
  • Pharmacodynamic: change in haematocrit [ Time Frame: Day 1 to Day 239 ]
  • Pharmacodynamic: Change in Transferrin saturation (TSAT) [ Time Frame: Day 1 to Day 239 ]
  • Pharmacodynamic: Change in Hepcidin [ Time Frame: Day 1 to Day 239 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Assess SLN124 in Patients With Polycythemia Vera
Official Title  ICMJE Phase 1/2 Study With an Open-label Dose Escalation Phase Followed by a Randomized, Double-blind Phase of SLN124 in Patients With Polycythemia Vera
Brief 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Phase 1 is an open-label, dose-finding study. Phase 2 is a randomized, double-blind, placebo-controlled study.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Polycythemia Vera
Intervention  ICMJE
  • Drug: SLN124
    SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
  • Drug: Placebo
    sodium chloride, solution for injection
Study Arms  ICMJE
  • Experimental: Phase 1 open-label SLN124
    SLN124 for subcutaneous (s.c.) injection
    Intervention: Drug: SLN124
  • Experimental: Phase 2 Blinded SLN124
    SLN124 for subcutaneous (s.c.) injection
    Intervention: Drug: SLN124
  • Placebo Comparator: Phase 2 Blinded Placebo
    Sodium chloride for s.c. injection
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 10, 2022)		 65
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2025
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male and female patients aged 18 years or older.
  • A confirmed diagnosis of PV according to the revised 2016 World Health Organization criteria:
  • Suitable phlebotomy history
  • Must agree to adhere to appropriate contraception requirements
  • Patients who are not receiving cytoreductive therapy must have been discontinued from any prior cytoreductive therapy for at least 24 weeks before dosing and have recovered from any adverse events due to cytoreductive therapy.
  • Patients receiving cytoreductive therapy with hydroxyurea, interferon, busulfan or ruxolitinib 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 6 months prior to screening.
  • Must have an Eastern Cooperative Oncology Group score of 0, 1, or 2.

Exclusion Criteria:

  • Drug intolerance:

    1. History of intolerance to oligonucleotides, or GalNAc, or any component of SLN124.
    2. History of intolerance to s.c. injections.
  • Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 12 weeks of screening.
  • History of major bleeding events and/or a requirement for blood transfusion therapy owing to bleeding in the last 6 months prior to screening.
  • Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment
  • Any investigational drug less than 6 weeks prior to the first dose of study drug or not recovered from effects of prior administration of any investigational agent.
  • Any investigational or marketed product using GalNAc targeting less than 48 weeks prior 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; or white blood cell (WBC) count > 25,000/µL; or peripheral blasts < 1%.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Silence Therapeutics Patient Information +44 (0) 20 3457 6900 patient-info@silence-therapeutics.com
Listed Location Countries  ICMJE Australia,   Bulgaria,   Malaysia,   Poland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05499013
Other Study ID Numbers  ICMJE SLN124-004
SANRECO ( Other Identifier: Silence Therapeutics )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Silence Therapeutics plc
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Silence Therapeutics plc
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Silence Therapeutics plc
Verification Date April 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP