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.
Brief Title
A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera
Detailed Description
Phase 3 study in approximately 250 subjects previously diagnosed with polycythemia vera (PV) who require phlebotomy on a routine basis. There is a 32-week period during which rusfertide or placebo will be added-on to each subject's ongoing therapy for polycythemia vera which may include phlebotomy only or phlebotomy plus stable doses of either of hydroxyurea, interferon and/or ruxolitinib. All subjects who successfully complete the double blind 32-week portion of the study will receive rusfertide for 124 weeks. Approximately 6 and 12 months after their last dose of rusfertide, subjects will have a post-study contact (e.g. by phone) for safety.
Categories
Completion Date
Completion Date Type
Estimated
Conditions
Polycythemia Vera
Eligibility Criteria
Main Inclusion Criteria: 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 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.
* 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 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.
Inclusion Criteria
Inclusion Criteria: 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.
* 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.
Gender
All
Gender Based
false
Keywords
Polycythemia Vera
PV
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT05210790
Org Class
Industry
Org Full Name
Protagonist Therapeutics, Inc.
Org Study Id
PTG-300-11
Overall Status
Active, not recruiting
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients With Polycythemia Vera
Primary Outcomes
Outcome Description
Response is defined as absence of phlebotomy eligibility.
Outcome Measure
Proportion of subjects achieving a response who receive rusfertide compared to placebo.
Outcome Time Frame
Week 20 through Week 32
Secondary Outcomes
Outcome Time Frame
Week 0 to Week 32
Outcome Measure
Comparison of mean number of phlebotomies between rusfertide and placebo.
Outcome Time Frame
Week 0 to Week 32
Outcome Measure
Proportion of subjects with HCT values <45% for rusfertide and placebo.
Outcome Time Frame
Week 32
Outcome Measure
Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo.
Outcome Time Frame
Week 32
Outcome Measure
Comparison of mean change from baseline in total MFSAF total score.
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Swati Goel
Investigator Email
swgoel@montefiore.org
Investigator Phone
718-920-6310 / 718-920-4137