Trial Review

Technical difficulties have been reported by some users of the search function and is being investigated by technical staff. Thank you for your patience and apologies for any inconvenience caused.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05210790




Registration number
NCT05210790
Ethics application status
Date submitted
14/01/2022
Date registered
27/01/2022
Date last updated
5/03/2024

Titles & IDs
Public title
A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera
Scientific title
A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients With Polycythemia Vera
Secondary ID [1] 0 0
PTG-300-11
Universal Trial Number (UTN)
Trial acronym
VERIFY
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polycythemia Vera 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - Rusfertide

Experimental: Rusfertide - Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)

Experimental: Placebo - Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)


Treatment: Drugs: Placebo
Placebo

Treatment: Drugs: Rusfertide
Experimental drug
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of subjects achieving a response who receive rusfertide compared to placebo.
Timepoint [1] 0 0
Week 20 through Week 32
Secondary outcome [1] 0 0
Comparison of mean number of phlebotomies between rusfertide and placebo.
Timepoint [1] 0 0
Week 0 to Week 32
Secondary outcome [2] 0 0
Proportion of subjects with HCT values <45% for rusfertide and placebo.
Timepoint [2] 0 0
Week 0 to Week 32
Secondary outcome [3] 0 0
Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo.
Timepoint [3] 0 0
Week 32
Secondary outcome [4] 0 0
Comparison of mean change from baseline in total MFSAF total score.
Timepoint [4] 0 0
Week 32

Eligibility
Key inclusion criteria
Main All subjects must meet ALL of the following inclusion criteria to
be enrolled. There are additional inclusion criteria.

- Male and female subjects aged 18 (or the country specific minimum age of consent >18)
years or older.

- Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of
polycythemia vera.

- At least 3 phlebotomies due to inadequate hematocrit control in 6 months before
randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1
year before randomization.

- CBC values immediately prior to randomization:

1. Hematocrit <45%,

2. WBC 4000/µL to 20,000/µL (inclusive), and

3. Platelets 100,000/µL to 1,000,000/µL (inclusive)

- Subjects receiving cytoreductive therapy at randomization must be on a stable PV
therapy regimen.

- Subjects treated with phlebotomy alone at randomization must have stopped
cytoreductive therapy 2 to 6 months before screening.

Main
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Subjects must meet NONE of the following exclusion criteria to be
enrolled. There are additional exclusion criteria.

- Clinically meaningful laboratory abnormalities at Screening.

- Subjects who require phlebotomy at hematocrit levels lower than 45%.

- Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein
thrombosis) within 2 months prior to randomization.

- Active or chronic bleeding within 2 months prior to randomization.

- History of invasive malignancies within the last 5 years, except localized cured
prostate cancer and cervical cancer.

- Subjects with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage
1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during
screen unless the cancer is adequately treated before randomization.

- Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2025
Actual
Sample size
Target
250
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
PerthSA,VIC
Recruitment hospital [1] 0 0
One Clinical Research - Nedlands
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [3] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Connecticut
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Indiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Iowa
Country [10] 0 0
United States of America
State/province [10] 0 0
Kansas
Country [11] 0 0
United States of America
State/province [11] 0 0
Louisiana
Country [12] 0 0
United States of America
State/province [12] 0 0
Maryland
Country [13] 0 0
United States of America
State/province [13] 0 0
Michigan
Country [14] 0 0
United States of America
State/province [14] 0 0
Nevada
Country [15] 0 0
United States of America
State/province [15] 0 0
New Jersey
Country [16] 0 0
United States of America
State/province [16] 0 0
New York
Country [17] 0 0
United States of America
State/province [17] 0 0
North Carolina
Country [18] 0 0
United States of America
State/province [18] 0 0
Ohio
Country [19] 0 0
United States of America
State/province [19] 0 0
Oregon
Country [20] 0 0
United States of America
State/province [20] 0 0
Pennsylvania
Country [21] 0 0
United States of America
State/province [21] 0 0
Texas
Country [22] 0 0
United States of America
State/province [22] 0 0
Utah
Country [23] 0 0
United States of America
State/province [23] 0 0
Washington
Country [24] 0 0
United States of America
State/province [24] 0 0
Wisconsin
Country [25] 0 0
Austria
State/province [25] 0 0
Graz
Country [26] 0 0
Austria
State/province [26] 0 0
Linz
Country [27] 0 0
Austria
State/province [27] 0 0
Vienna
Country [28] 0 0
Austria
State/province [28] 0 0
Wels
Country [29] 0 0
Austria
State/province [29] 0 0
Wien
Country [30] 0 0
Belgium
State/province [30] 0 0
Antwerp
Country [31] 0 0
Belgium
State/province [31] 0 0
Gent
Country [32] 0 0
Belgium
State/province [32] 0 0
Leuven
Country [33] 0 0
Canada
State/province [33] 0 0
Alberta
Country [34] 0 0
Canada
State/province [34] 0 0
Ontario
Country [35] 0 0
Canada
State/province [35] 0 0
Quebec
Country [36] 0 0
Chile
State/province [36] 0 0
Santiago
Country [37] 0 0
Chile
State/province [37] 0 0
Concepción
Country [38] 0 0
Czechia
State/province [38] 0 0
Brno
Country [39] 0 0
Czechia
State/province [39] 0 0
Olomouc
Country [40] 0 0
Czechia
State/province [40] 0 0
Prague
Country [41] 0 0
France
State/province [41] 0 0
Cedex 09
Country [42] 0 0
France
State/province [42] 0 0
Cedex 15
Country [43] 0 0
France
State/province [43] 0 0
Cedex
Country [44] 0 0
France
State/province [44] 0 0
Amiens
Country [45] 0 0
France
State/province [45] 0 0
Argenteuil
Country [46] 0 0
France
State/province [46] 0 0
Béziers
Country [47] 0 0
France
State/province [47] 0 0
Paris
Country [48] 0 0
France
State/province [48] 0 0
Pierre-Bénite
Country [49] 0 0
Germany
State/province [49] 0 0
Aachen
Country [50] 0 0
Germany
State/province [50] 0 0
Berlin
Country [51] 0 0
Germany
State/province [51] 0 0
Dresden
Country [52] 0 0
Germany
State/province [52] 0 0
Greifswald
Country [53] 0 0
Germany
State/province [53] 0 0
Hamburg
Country [54] 0 0
Germany
State/province [54] 0 0
Koblenz
Country [55] 0 0
Germany
State/province [55] 0 0
Mainz
Country [56] 0 0
Hong Kong
State/province [56] 0 0
Hong Kong
Country [57] 0 0
Hungary
State/province [57] 0 0
Budapest
Country [58] 0 0
Hungary
State/province [58] 0 0
Debrecen
Country [59] 0 0
Hungary
State/province [59] 0 0
Eger
Country [60] 0 0
Israel
State/province [60] 0 0
Haifa
Country [61] 0 0
Israel
State/province [61] 0 0
Jerusalem
Country [62] 0 0
Israel
State/province [62] 0 0
Kfar Saba
Country [63] 0 0
Israel
State/province [63] 0 0
Petach Tikva
Country [64] 0 0
Israel
State/province [64] 0 0
Tel Aviv
Country [65] 0 0
Israel
State/province [65] 0 0
Zerifin
Country [66] 0 0
Italy
State/province [66] 0 0
Forli-Cesena
Country [67] 0 0
Italy
State/province [67] 0 0
Bergamo
Country [68] 0 0
Italy
State/province [68] 0 0
Bologna
Country [69] 0 0
Italy
State/province [69] 0 0
Brescia
Country [70] 0 0
Italy
State/province [70] 0 0
Firenze
Country [71] 0 0
Italy
State/province [71] 0 0
Milano
Country [72] 0 0
Italy
State/province [72] 0 0
Pavia
Country [73] 0 0
Italy
State/province [73] 0 0
Pesaro
Country [74] 0 0
Italy
State/province [74] 0 0
Roma
Country [75] 0 0
Italy
State/province [75] 0 0
Torino
Country [76] 0 0
Italy
State/province [76] 0 0
Varese
Country [77] 0 0
Mexico
State/province [77] 0 0
Madero
Country [78] 0 0
Mexico
State/province [78] 0 0
Mexico City
Country [79] 0 0
Netherlands
State/province [79] 0 0
Dordrecht
Country [80] 0 0
Netherlands
State/province [80] 0 0
Rotterdam
Country [81] 0 0
Poland
State/province [81] 0 0
Brzozów
Country [82] 0 0
Poland
State/province [82] 0 0
Bydgoszcz
Country [83] 0 0
Poland
State/province [83] 0 0
Gdansk
Country [84] 0 0
Poland
State/province [84] 0 0
Krakow
Country [85] 0 0
Poland
State/province [85] 0 0
Lodz
Country [86] 0 0
Poland
State/province [86] 0 0
Torun
Country [87] 0 0
Poland
State/province [87] 0 0
Warszawa
Country [88] 0 0
Poland
State/province [88] 0 0
Wroclaw
Country [89] 0 0
Poland
State/province [89] 0 0
Lódz
Country [90] 0 0
Portugal
State/province [90] 0 0
Almada
Country [91] 0 0
Portugal
State/province [91] 0 0
Braga
Country [92] 0 0
Portugal
State/province [92] 0 0
Lisboa
Country [93] 0 0
Portugal
State/province [93] 0 0
Porto
Country [94] 0 0
Spain
State/province [94] 0 0
Barcelona
Country [95] 0 0
Spain
State/province [95] 0 0
Madrid
Country [96] 0 0
Spain
State/province [96] 0 0
Valencia
Country [97] 0 0
Spain
State/province [97] 0 0
Zaragoza
Country [98] 0 0
Turkey
State/province [98] 0 0
Ankara
Country [99] 0 0
Turkey
State/province [99] 0 0
Denizli
Country [100] 0 0
Turkey
State/province [100] 0 0
Izmir
Country [101] 0 0
Turkey
State/province [101] 0 0
Ortahisar
Country [102] 0 0
Turkey
State/province [102] 0 0
Sehitkamil
Country [103] 0 0
Turkey
State/province [103] 0 0
Gaziantep
Country [104] 0 0
Turkey
State/province [104] 0 0
Istanbul
Country [105] 0 0
Turkey
State/province [105] 0 0
Kayseri
Country [106] 0 0
United Kingdom
State/province [106] 0 0
Middlesex
Country [107] 0 0
United Kingdom
State/province [107] 0 0
Barnet
Country [108] 0 0
United Kingdom
State/province [108] 0 0
Boston
Country [109] 0 0
United Kingdom
State/province [109] 0 0
Harrow
Country [110] 0 0
United Kingdom
State/province [110] 0 0
London
Country [111] 0 0
United Kingdom
State/province [111] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Protagonist Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The study is designed to evaluate the safety and efficacy of rusfertide in subjects with
polycythemia vera (PV) in maintaining hematocrit control and in improving symptoms of PV.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05210790
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Study Director
Address 0 0
Country 0 0
Phone 0 0
1-888-899-1543
Fax 0 0
Email 0 0
ptgxclintrials@ptgx-inc.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05210790