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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Polycythemia Vera Drug: SLN124 Drug: Placebo Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 65 participants
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
Official Title: 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
Actual Study Start Date : January 26, 2023
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : June 2025

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Phase 1 open-label SLN124
SLN124 for subcutaneous (s.c.) injection
 Drug: SLN124
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Experimental: Phase 2 Blinded SLN124
SLN124 for subcutaneous (s.c.) injection
 Drug: SLN124
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Placebo Comparator: Phase 2 Blinded Placebo
Sodium chloride for s.c. injection
 Drug: Placebo
sodium chloride, solution for injection


Outcome Measures
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Primary Outcome Measures :
  1. 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

  2. Assessment of the number of phlebotomies at intervals [ Time Frame: 6 months prior to dosing to Day 239 ]

Secondary Outcome Measures :
  1. Pharmacokinetic: area under the plasma concentration (AUC) [ Time Frame: Day 127 ]
  2. Pharmacokinetic: peak plasma concentration (Cmax) [ Time Frame: Day 127 ]
  3. Pharmacodynamic: change in haematocrit [ Time Frame: Day 1 to Day 239 ]
  4. Pharmacodynamic: Change in Transferrin saturation (TSAT) [ Time Frame: Day 1 to Day 239 ]
  5. Pharmacodynamic: Change in Hepcidin [ Time Frame: Day 1 to Day 239 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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%.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05499013


Contacts
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Contact: Silence Therapeutics Patient Information +44 (0) 20 3457 6900 patient-info@silence-therapeutics.com

Locations
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Sponsors and Collaborators
Silence Therapeutics plc
More Information
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Responsible Party: Silence Therapeutics plc
ClinicalTrials.gov Identifier: NCT05499013    
Other Study ID Numbers: SLN124-004
SANRECO ( Other Identifier: Silence Therapeutics )
First Posted: August 12, 2022    Key Record Dates
Last Update Posted: April 22, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Silence Therapeutics plc:
PV
Additional relevant MeSH terms:
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Polycythemia Vera
Polycythemia
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
 Neoplasms by Site
Neoplasms
Bone Marrow Diseases
Myeloproliferative Disorders