Clinical Trial: NCT04057040 - My Cancer Genome

NCT04057040

Description:

This is a Phase 2 study with an open-label dose escalation phase followed by a blinded withdrawal phase and an open label extension. The study is designed to monitor the PTG-300 safety profile and to obtain preliminary evidence of efficacy of PTG-300 for the treatment of phlebotomy-requiring polycythemia vera.

Related Conditions:

Polycythemia Vera

Recruiting Status:

Recruiting

Phase:

Phase 2

URL:

https://clinicaltrials.gov/show/NCT04057040

Trial Eligibility

Document

Title

Brief Title: Hepcidin Mimetic in Patients With Polycythemia Vera
Official Title: A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera

Clinical Trial IDs

ORG STUDY ID: PTG-300-04
NCT ID: NCT04057040

Conditions

Polycythemia Vera

Interventions

Drug	Synonyms	Arms
PTG-300		Blinded Withdrawal (Part 2) PTG-300 or placebo
Placebo		Blinded Withdrawal (Part 2) PTG-300 or placebo

Purpose

Detailed Description

      Phase 2 study in approximately thirty subjects previously diagnosed with Polycythemia Vera
      who require phlebotomy on a routine basis. There is a 16 week dose finding phase followed by
      a dose stabilization phase. Subjects who successfully complete dose stabilization will be
      entered into a 12 week randomized withdrawal phase to confirm the response. Subsequently
      patients will enter into a 1 year open label extension to investigate long term safety.

Trial Arms

Name	Type	Description	Interventions
Experimental Dose Escalation (Part 1)	Experimental	PTG-300 Subcutaneous (SC) weekly. Each subject's PTG-300 dose is increased at 4 week intervals until the subject reaches the maximum planned dose or has a prespecified decrease in hematocrit from baseline.	PTG-300
Blinded Withdrawal (Part 2) PTG-300 or placebo	Experimental	PTG-300 or placebo Subcutaneous (SC) weekly.	PTG-300 Placebo
Experimental Open label extension (Part 3) PTG-300	Experimental	PTG-300 Subcutaneous (SC) weekly	PTG-300

Eligibility Criteria

        Main Inclusion Criteria: All subjects must meet ALL of the following inclusion criteria to
        be enrolled.

          1. Male and female subjects aged 18 years or older.

          2. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of
             polycythemia vera.

          3. Records of all phlebotomies performed for at least 24 weeks (preferably up to 52
             weeks) before screening are available.

          4. 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 adverse events due to cytoreductive therapy.

          5. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib
             must be on a stable dose for at least 24 weeks and be on a stable dose for at least 8
             weeks before screening and with no planned change in dose.

        Main Exclusion Criteria: Subjects must meet NONE of the following exclusion criteria to be
        enrolled:

          1. Active or chronic bleeding within 4 weeks of screening.

          2. Meets the criteria for post-PCV myelofibrosis as defined by the International Working
             Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).

          3. Known primary or secondary immunodeficiency.

          4. Any surgical procedure requiring general anesthesia within 1 month prior to screening
             or planned elective surgery during the study.

Maximum Eligible Age:	N/A
Minimum Eligible Age:	18 Years
Eligible Gender:	All
Healthy Volunteers:	No

Primary Outcome Measures

Measure:	Proportion of subjects with absence of phlebotomy eligibility
Time Frame:	Week 42
Safety Issue:
Description:	"Phlebotomy eligibility" is defined as a confirmed hematocrit >45% that was ≥3% higher than baseline level or a confirmed hematocrit >48%

Secondary Outcome Measures

Measure:	Change from baseline in serum iron
Time Frame:	42 weeks
Safety Issue:
Description:

Measure:	Change from baseline in transferrin saturation (TSAT)
Time Frame:	42 weeks
Safety Issue:
Description:

Measure:	Change from baseline in serum transferrin
Time Frame:	42 weeks
Safety Issue:
Description:

Details

Phase:	Phase 2
Primary Purpose:	Interventional
Overall Status:	Recruiting
Lead Sponsor:	Protagonist Therapeutics, Inc.

Trial Keywords

polycythemia vera

Last Updated

November 5, 2020

Clinical Trials /

Hepcidin Mimetic in Patients With Polycythemia Vera