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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
69
Research Site
Hammond, Louisiana, United States
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
Time frame: Day 239
Phase 1: Assessment of the number of phlebotomies at intervals
Time frame: 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)
Time frame: 18 to 36 weeks
Phase 1: Pharmacokinetic: area under the plasma concentration (AUC)
Time frame: Day 127
Phase 1: Pharmacokinetic: peak plasma concentration (Cmax)
Time frame: Day 127
Phase 1: Pharmacodynamic: change in haematocrit
Time frame: Day 1 to Day 239
Phase 1: Pharmacodynamic: Change in Transferrin saturation (TSAT)
Time frame: Day 1 to Day 239
Phase 1: Pharmacodynamic: Change in Hepcidin
Time frame: Day 1 to Day 239
Phase 2: Comparison of the effect of SLN124 vs placebo
Number of phlebotomies
Time frame: Over 36 weeks
Phase 2: Comparison of the effect of SLN124 vs placebo
Proportion of patients who achieve a response
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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
...and 18 more locations
Time frame: Over 36 weeks
Phase 2: Comparison of the effect of SLN124 vs placebo
Safety and tolerability
Time frame: Over 36 weeks
Phase 2: Comparison of the effect of SLN124 vs placebo
Hematology parameters and biomarkers of iron metabolism
Time frame: Over 36 weeks
Phase 2: In the double-blind extension period and in the OLE period:
Assess long-term safety and tolerability of SLN124
Time frame: Week 37 to Week 181
Phase 2: In the double-blind extension period and in the OLE period:
Assess long-term effects of SLN124 on QoL assessments
Time frame: Week 37 to Week 181
Phase 2: Assessment of SLN124 Cmax at Day 1 and Day 169 of the trial.
Time frame: Day 1 and Day 169
Phase 2: Assessment of SLN124 PD
Hepcidin
Time frame: Changes from Week 1 to Week 181
Phase 2: Assessment of QoL
MPN-SAF-TSS
Time frame: Changes from Week 1 to Week 181
Phase 2: Assessment of QoL
PGI-C
Time frame: Changes from Week 1 to Week 181
Phase 2: Pharmacodynamic: Change in haematocrit
Time frame: Changes from Week 1 to Week 181
Phase 2: Pharmacodynamic: Change in Hepcidin
Time frame: Changes from Week 1 to Week 181