NCT05499013

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.

Trial Health

82
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
69

participants targeted

Target at P75+ for phase_1

Timeline
46mo left

Started Jan 2023

Longer than P75 for phase_1

Geographic Reach
9 countries

28 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress46%
Jan 2023Mar 2030

First Submitted

Initial submission to the registry

July 29, 2022

Completed
14 days until next milestone

First Posted

Study publicly available on registry

August 12, 2022

Completed
6 months until next milestone

Study Start

First participant enrolled

January 26, 2023

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2026

Expected
3.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2030

Last Updated

December 19, 2025

Status Verified

December 1, 2025

Enrollment Period

3.6 years

First QC Date

July 29, 2022

Last Update Submit

December 17, 2025

Conditions

Polycythemia Vera

Keywords

PV

Outcome Measures

Primary Outcomes (3)

  • Phase 1: Incidence of treatment-emergent adverse events (AEs)

    Safety and tolerability will be reported separately following open-label dose escalation phase and double-blind phase

    Day 239

  • Phase 1: Assessment of the number of phlebotomies at intervals

    6 months prior to dosing to Day 239

  • Phase 2: Proportion of patients who achieve response between week 18 and week 36 (placebo controlled double blind phase)

    18 to 36 weeks

Secondary Outcomes (17)

  • Phase 1: Pharmacokinetic: area under the plasma concentration (AUC)

    Day 127

  • Phase 1: Pharmacokinetic: peak plasma concentration (Cmax)

    Day 127

  • Phase 1: Pharmacodynamic: change in haematocrit

    Day 1 to Day 239

  • Phase 1: Pharmacodynamic: Change in Transferrin saturation (TSAT)

    Day 1 to Day 239

  • Phase 1: Pharmacodynamic: Change in Hepcidin

    Day 1 to Day 239

  • +12 more secondary outcomes

Study Arms (3)

Phase 1 open-label SLN124

EXPERIMENTAL

SLN124 for subcutaneous (s.c.) injection

Drug: SLN124

Phase 2 Blinded SLN124

EXPERIMENTAL

SLN124 for subcutaneous (s.c.) injection

Drug: SLN124

Phase 2 Blinded Placebo

PLACEBO COMPARATOR

Sodium chloride for s.c. injection

Drug: Placebo

Interventions

SLN124DRUG

SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).

Phase 1 open-label SLN124Phase 2 Blinded SLN124
PlaceboDRUG

sodium chloride, solution for injection

Phase 2 Blinded Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 28 weeks prior to dosing.
  • Must have an Eastern Cooperative Oncology Group score of 0, 1, or 2.

You may not qualify if:

  • Phase 1 and Phase 2
  • Drug intolerance:
  • History of intolerance to oligonucleotides, or GalNAc, or any component of SLN124.
  • 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:
  • Biochemical evidence of significant liver disease during screening
  • 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%.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

Research Site

Hammond, Louisiana, 70403, United States

Location

Research Site

Buffalo, New York, 14203, United States

Location

Research Site

New York, New York, 10029, United States

Location

Research Site

Columbus, Ohio, 43210, United States

Location

Research Site

Huntsville, Texas, 77340, United States

Location

Research Site

Benowa, Australia

Location

Research Site

Hobart, Australia

Location

Research Site

Kurralta Park, Australia

Location

Research Site 2

Melbourne, Australia

Location

Research Site

Melbourne, Australia

Location

Linear Clinical Research

Nedlands, Australia

Location

Research Site

Richmond, Australia

Location

Research Site 2

Plovdiv, Bulgaria

Location

Research Site

Plovdiv, Bulgaria

Location

Research Site

Toronto, Canada

Location

Research Site

Freiburg im Breisgau, Germany

Location

Research Site

Hanover, Germany

Location

Research Site

Alessandria, Italy

Location

Research Site

Meldola, Italy

Location

Research Site

Johor Bahru, Malaysia

Location

Research Site

Kuala Terengganu, Malaysia

Location

Research Site

Kuantan, Malaysia

Location

Research Site

Kuching, Malaysia

Location

Research Site

Gdansk, Poland

Location

Research Site

Katowice, Poland

Location

Research Site

Lublin, Poland

Location

Research Site

Barcelona, Spain

Location

Research Site

Madrid, Spain

Location

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative Disorders

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Phase 1 is an open-label, dose-finding study. Phase 2 is a randomized, double-blind, placebo-controlled study.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 29, 2022

First Posted

August 12, 2022

Study Start

January 26, 2023

Primary Completion (Estimated)

September 1, 2026

Study Completion (Estimated)

March 1, 2030

Last Updated

December 19, 2025

Record last verified: 2025-12

Locations

US(5)CA(1)ES(2)DE(2)AU(7)MY(4)PL(3)IT(2)BU(2)