Study to Assess SLN124 in Patients With Polycythemia Vera
NCT ID: NCT05499013
Last Updated: 2025-12-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
69 participants
INTERVENTIONAL
2023-01-26
2030-03-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Phase 1 open-label SLN124
SLN124 for subcutaneous (s.c.) injection
SLN124
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Phase 2 Blinded SLN124
SLN124 for subcutaneous (s.c.) injection
SLN124
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Phase 2 Blinded Placebo
Sodium chloride for s.c. injection
Placebo
sodium chloride, solution for injection
Interventions
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SLN124
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Placebo
sodium chloride, solution for injection
Eligibility Criteria
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Inclusion Criteria
* 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 28 weeks prior to dosing.
* 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 (excludes patients with PV who participated 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%.
18 Years
ALL
No
Sponsors
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Silence Therapeutics plc
INDUSTRY
Responsible Party
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Locations
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Research Site
Hammond, Louisiana, United States
Research Site
Buffalo, New York, United States
Research Site
New York, New York, United States
Research Site
Columbus, Ohio, United States
Research Site
Huntsville, Texas, United States
Research Site
Benowa, , Australia
Research Site
Hobart, , Australia
Research Site
Kurralta Park, , Australia
Research Site 2
Melbourne, , Australia
Research Site
Melbourne, , Australia
Linear Clinical Research
Nedlands, , Australia
Research Site
Richmond, , Australia
Research Site 2
Plovdiv, , Bulgaria
Research Site
Plovdiv, , Bulgaria
Research Site
Toronto, , Canada
Research Site
Freiburg im Breisgau, , Germany
Research Site
Hanover, , Germany
Research Site
Alessandria, , Italy
Research Site
Meldola, , Italy
Research Site
Johor Bahru, , Malaysia
Research Site
Kuala Terengganu, , Malaysia
Research Site
Kuantan, , Malaysia
Research Site
Kuching, , Malaysia
Research Site
Gdansk, , Poland
Research Site
Katowice, , Poland
Research Site
Lublin, , Poland
Research Site
Barcelona, , Spain
Research Site
Madrid, , Spain
Countries
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Other Identifiers
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SANRECO
Identifier Type: OTHER
Identifier Source: secondary_id
SLN124-004
Identifier Type: -
Identifier Source: org_study_id