History of Changes for Study: NCT04057040

Hepcidin Mimetic in Patients With Polycythemia Vera

Latest version (submitted November 14, 2023) on ClinicalTrials.gov

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Study Record Versions

Version	Submitted Date	Changes
1	August 13, 2019	None (earliest Version on record)
2	August 30, 2019	Contacts/Locations and Study Status
3	December 16, 2019	Recruitment Status, Study Status, Arms and Interventions, Contacts/Locations and Eligibility
4	December 18, 2019	Recruitment Status, Study Status and Contacts/Locations
5	September 25, 2020	Contacts/Locations, Study Design, Study Status, Arms and Interventions and Study Description
6	November 4, 2020	Study Status and Study Design
7	September 21, 2021	Recruitment Status, Contacts/Locations, Outcome Measures, Study Design, Study Status, Arms and Interventions, Eligibility and Study Description
8	January 10, 2022	Recruitment Status, Study Status, Contacts/Locations, Study Design and Study Identification
9	April 12, 2022	Recruitment Status, Study Status and Contacts/Locations
10	November 14, 2023	Study Status

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Study NCT04057040
Submitted Date: August 13, 2019 (v1)

Study Identification


Unique Protocol ID:	PTG-300-04
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
Secondary IDs:

Study Status


Record Verification:	August 2019
Overall Status:	Recruiting
Study Start:	August 2019
Primary Completion:	February 2021 [Anticipated]
Study Completion:	March 2021 [Anticipated]

First Submitted:	August 7, 2019
First Submitted that Met QC Criteria:	August 13, 2019
First Posted:	August 14, 2019 [Actual]

Last Update Submitted that Met QC Criteria:	August 13, 2019
Last Update Posted:	August 14, 2019 [Actual]

Sponsor/Collaborators


Sponsor:	Protagonist Therapeutics, Inc.
Responsible Party:	Sponsor
Collaborators:

Study Description

Brief Summary:

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.

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 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into 1 year open label extension to investigate long term safety.

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 2
Interventional Study Model:	Sequential Assignment
	Part 1: 28 week open-label dose escalation phase in which each subject's dose of PTG-300 is increased at 4-week intervals until the subject reaches the maximum planned dose or has a pre-specified decrease in hematocrit from baseline. After a potentially clinically active dose is found, subjects will be maintained at that dose until Week 29. Part 2: 12-week blinded randomized withdrawal phase. Subjects are randomized 1:1 to continue PTG-300 or to receive placebo. Part 3: 1 year open label extension.
Number of Arms:	3
Masking:	Double (Participant, Investigator)
Allocation:	Randomized
Enrollment:	30 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
Experimental: Dose Escalation (Part 1) PTG-300 Subcutaneous (SC) Weekly Each subject's dose is increased at 4 week intervals until the subject reaches the maximum planned dose or has a prespecified decrease in hematocrit from baseline.	Drug: PTG-300 Active Other Names: PTG-300 10 mg to 80 mg
Experimental: Blinded Withdrawal (Part 2) PTG-300 PTG-300 Subcutaneous (SC) Weekly	Drug: PTG-300 Active Other Names: PTG-300 10 mg to 80 mg
Placebo Comparator: Blinded Withdrawal (Part 2) Placebo Placebo (SC) weekly	Drug: Placebo Placebo

Outcome Measures


Primary Outcome Measures:
1.	Proportion of subjects with absence of phlebotomy eligibility [ Time Frame: Week 42 ] "Phlebotomy eligibility" is defined as a confirmed hematocrit >45% that was ≥3% higher than baseline level or a confirmed hematocrit >48%
2.	Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) [ Time Frame: 42 weeks ] The Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) is a validated tool for measurement of symptoms for patients. The MPN-SAF includes 14 disease related symptoms scored from 0 (absent) to 10 (worst imaginable) and is used to calculate a total symptom score (TSS; sum of 14 individual symptom scores).
3.	Number of Participants with Treatment-Emergent Adverse Events as Assessed by CTCAE v5.0 [ Time Frame: 101 weeks ]
Secondary Outcome Measures:
1.	Change from baseline in serum iron [ Time Frame: 42 weeks ]
2.	Change from baseline in transferrin saturation (TSAT) [ Time Frame: 42 weeks ]
3.	Change from baseline in serum transferrin [ Time Frame: 42 weeks ]

Eligibility


Minimum Age:	18 Years
Maximum Age:
Sex:	All
Gender Based:
Accepts Healthy Volunteers:	No
Criteria:	Main Inclusion Criteria: All subjects must meet ALL of the following inclusion criteria to be enrolled. Male and female subjects aged 18 years or older. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera. Receiving aspirin therapy for polycythemia vera (subjects for whom aspirin is not contraindicated). Records of all phlebotomies performed for at least 24 weeks before screening are available. 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. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib must be on a stable dose for at least 24 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: Active or chronic bleeding within 4 weeks of screening. Meets the criteria for post-PCV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT). Known primary or secondary immunodeficiency. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study.

Contacts/Locations


Central Contact Person:	Suneel Gupta Telephone: 510-474-0978 Email: s.gupta@ptgx-inc.com
Central Contact Backup:	Frank Valone Email: f.valone@ptgx.inc.com
Locations:	United States, Florida
	Moffitt [Not yet recruiting] Tampa, Florida, United States, 33612
	United States, Maryland
	Center for Cancer and Blood Disorders [Recruiting] Bethesda, Maryland, United States, 20817
	United States, Michigan
	Karmanos Cancer Center [Recruiting] Detroit, Michigan, United States, 48201
	United States, New York
	Mount Sinai [Recruiting] New York, New York, United States, 10029
	United States, Texas
	The University of Texas MD Anderson Cancer Center [Recruiting] Houston, Texas, United States, 77030
	The University of Texas at San Antonio [Recruiting] San Antonio, Texas, United States, 78229

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