This clinical trial is a Phase 2/3 study that will determine the recommended dose and dose regimen of AG-236 and evaluate the efficacy, safety, and pharmacokinetics of AG-236 in polycythemia vera (PV) by testing how well AG-236 works compared to placebo to increase the number of subjects with no phlebotomy and absence of phlebotomy eligibility over a 13-week period. In addition, the long-term effect of AG-236 on efficacy and safety will be explored in an open-label extension portion.
PV diagnosis according to 2022 World Health Organization (WHO) criteria.
At least 3 documented phlebotomies in the 6 months before randomization or at least 5 documented phlebotomies in the 12 months before randomization; and
At least 1 documented phlebotomy within the 3 months before randomization.
No phlebotomy within 6 days before randomization (not including the day of phlebotomy and the day of randomization in the 6-day count).
Use of any investigational or marketed N-acetylgalactosamine (GalNAc) targeting product less than 48 weeks before randomization or not recovered from effects of prior administration of any investigational or marketed GalNAc targeting product taken more than 48 weeks before randomization.
Currently receiving treatment with a hepcidin mimetic; the last dose must have been administered ≥6 months before randomization.
Currently receiving treatment with or history of any use of TMPRSS6 targeting molecules (antisense oligonucleotide [ASO], siRNA, antibodies) before randomization.
Clinically significant, active and/or uncontrolled hepatobiliary, renal, cardiac, or pulmonary disease.
Sequential
AG-236
DrugSC Injection
Placebo
DrugSC Injection
7
Treatment groupsSee each treatment group below.
Phase 2 and Phase 3: Percentage of Subjects With No Phlebotomy and Absence of Phlebotomy Eligibility
Phase 2 and Phase 3: Number of Phlebotomies During the Double-blind Period
Phase 2: Percentage of Subjects Achieving Hematocrit (HCT) Response During the Double-blind Period
HCT response is defined as having at most one HCT assessment ≥45 percent (%) during the double-blind period.
Phase 2 and Phase 3: Time to First HCT ≥45%
Phase 2 and Phase 3: Number of Subjects With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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This trial is not recruiting at the moment. You can still explore other options:
