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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT05210790
Ethics application status
Date submitted
14/01/2022
Date registered
27/01/2022
Date last updated
17/07/2024

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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
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
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Alabama
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Arizona
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California
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Kansas
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Graz
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Oxford

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.