Assessing SLN124 for treating Polycythemia Vera

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

This study is testing a new treatment called SLN124 to see if it can safely help adults with Polycythemia Vera feel better.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 65 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Silence Therapeutics plc (Industry-sponsored)
• Drugs / interventions: ruxolitinib
• Locations: 23 sites (Ann Arbor, Michigan and 22 other locations)
• Trial ID: NCT05499013 on ClinicalTrials.gov

What this trial studies

This Phase 1/2 clinical trial evaluates the safety, tolerability, and efficacy of SLN124 in adult patients diagnosed with Polycythemia Vera (PV). The study employs a multicenter approach, beginning with an open-label dose escalation followed by a randomized, placebo-controlled, double-blind phase. Participants will be monitored for pharmacokinetic and pharmacodynamic responses to the treatment. The trial aims to gather comprehensive data on how SLN124 affects patients with PV.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of Polycythemia Vera who meet specific inclusion criteria.

Not a fit: Patients with a history of intolerance to oligonucleotides or any components of SLN124 may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from Polycythemia Vera.

How similar studies have performed: While this approach is novel, similar studies targeting Polycythemia Vera have shown promise in exploring new treatment avenues.

Eligibility 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%.

Where this trial is running

Ann Arbor, Michigan and 22 other locations

• University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
• Mount Sinai Hospital — New York, New York, United States (Recruiting)
• Duke Cancer Institute — Durham, North Carolina, United States (Not_yet_recruiting)
• North Houston Cancer Clinics — Huntsville, Texas, United States (Completed)
• Renovatio Clinical — The Woodlands, Texas, United States (Recruiting)
• Pindara Private Hospital — Benowa, Queensland, Australia (Recruiting)
• Ashford Cancer Centre Research — Kurralta Park, South Australia, Australia (Recruiting)
• Peter MacCallum Cancer Centre — Melbourne, Victoria, Australia (Recruiting)
• Alfred Health — Melbourne, Victoria, Australia (Recruiting)
• Epworth HealthCare — Richmond, Victoria, Australia (Recruiting)
• Linear Clinical Research — Nedlands, Western Australia, Australia (Recruiting)
• MHAT Dr Nikola Vasiliev AD — Kyustendil, Bulgaria (Recruiting)
• Medical Centre Leo Clinic EOOD — Plovdiv, Bulgaria (Recruiting)
• Hospital Sultanah Aminah — Johor Bahru, Johor, Malaysia (Recruiting)
• Hospital Tengku Ampuan Afzan — Kuantan, Pahang, Malaysia (Recruiting)
• Hospital Umum Sarawak — Kuching, Sarawak, Malaysia (Recruiting)
• Hospital Ampang — Ampang, Selangor, Malaysia (Recruiting)
• Hospital Sultanah Nur Zahirah — Kuala Terengganu, Terengganu, Malaysia (Recruiting)
• Uniwersyteckie Centrum Klinlczne — Gdańsk, Poland (Recruiting)
• PRATIA Hematologia Sp. z o. o. — Katowice, Poland (Recruiting)
• Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie — Lublin, Poland (Recruiting)
• Centrum Medyczne Pratia Poznan Sp. z o. o. — Skorzewo, Poland (Recruiting)
• Specjalistyczny Szpital Im Dra. A. — Wałbrzych, Poland (Recruiting)

Study contacts

• Study coordinator: Silence Therapeutics Patient Information

Conditions Polycythemia Vera; PV