MC# 21-13 - A Phase II Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera

  • Agent(s): PTG-300
  • Disease Type(s): Polycythemia Vera
  • Phase(s): II
  • Drug Classification(s): Other
  • Molecular Target(s): n/a

Mechanism of Action

PTG-300 mimics endogenous hepcidin, a protein primarily produced in hepatocytes, and increases hepcidin levels.  As hepcidin plays a key role in the homeostasis of systemic iron, PTG-300 may serve to normalize iron levels.

Purpose

In this study, the sponsor and investigators want to learn:

  • How much of PTG-300 can be given with an acceptable level of side effects
  • If PTG-300 can reduce the level of hematocrit in your blood
  • The effects of PTG-300 (good and bad)
  • The safety and effectiveness of PTG-300 for reducing the need for phlebotomies
  • How much of PTG-300 is absorbed into the blood and how fast it is removed
Inclusion Criteria
  1. Male and female subjects aged 18 years or older
  2. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of PV
  3. Records of all phlebotomies performed for at least 28 weeks (and preferably up to 52 weeks) before dosing
  4. Women of childbearing potential and men agree to use medically acceptable contraception (<1% annual failure rate) during the study and for 90 days after the last dose of study drug
  5. Subjects who are not receiving cytoreductive therapy must have been discontinued from any prior cytoreductive therapy for at least 24 weeks before Screening and have recovered from any AEs due to cytoreductive therapy
  6. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib must have received cytoreductive therapy for at least 24 weeks and be on a stable dose or have a decreasing dose (Medical Monitor approval required) for at least 8 weeks before dosing with no planned change in dose
Exclusion Criteria
  1. Pregnant or lactating females
  2. Active or chronic bleeding within 4 weeks of Screening
  3. Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)
  4. Known primary or secondary immunodeficiency
  5. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study

Location

  • Dallas, TX - Mary Crowley Cancer Research - Medical City
More Info: https://clinicaltrials.gov/ct2/show/NCT04057040

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Re: MC# 21-13