Polycythemia Vera MedDRA version: 21.1 Level: PT Classification code 10036057 Term: Polycythaemia vera System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 21.1 Level: LLT Classification code 10036061 Term: Polycythemia vera System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: Rusfertide
Product Code: PTG-300
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Rusfertide
CAS Number: 1628323-80-7
Current Sponsor code: PTG-300
Sponsors
Protagonist Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female subjects aged 18 (or the minimum country specific age of consent if >18) years or older. 3. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera 4. Phlebotomy requiring defined as ALL of the following: a. At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization, and 5. CBC values immediately prior to randomization: a. Hematocrit =65 years) yes F.1.3.1 Number of subjects for this age range 50
Exclusion criteria
Exclusion criteria: 1. Clinically meaningful laboratory abnormalities at Screening 2. Subjects who require phlebotomy at hematocrit levels lower than 45%. 3. Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization. 4. Active or chronic bleeding within 2 months prior to randomization. 5. History of invasive malignancies within the last 5 years, except a) localized cured cancer (e.g. prostate cancer and cervical cancer). b) localized cured in situ or stage 1 squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or in situ melanoma of the skin. 6. 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 screening unless the cancer is adequately treated before randomization. 7. Received busulfan, pipobroman or 32Phosphorus within 7 months prior to screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and efficacy of rusfertide in subjects with polycythemia vera in maintaining hematocrit control.;Secondary Objective: Not applicable;Primary end point(s): Proportion of subjects achieving a response starting at Week 20 through Week 32 (inclusive) who receive rusfertide compared to placebo. A response is defined as absence of phlebotomy eligibility. Phlebotomy eligibility is defined as either: • a confirmed hematocrit =45% and that is at least 3% higher than the baseline hematocrit (value immediately prior to randomization at Week 0); or • a hematocrit =48%. ;Timepoint(s) of evaluation of this end point: Week 20 through Week 32 (inclusive) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Mean number of phlebotomies between Week 0 through Week 32 (inclusive). 2. Proportion of subjects with all hematocrit values <45% between Week 0 through Week 32 (inclusive). 3. Mean change from baseline in total fatigue score based on PROMIS® Short Form 8a at Week 32. 4. Mean change from baseline in total score based on MFSAF v4.0 at Week 32. ;Timepoint(s) of evaluation of this end point: Week 0 through Week 32 (inclusive) | — |
Countries
Australia, Austria, Belgium, Canada, Chile, Czechia, Czech Republic, France, Germany, Hong Kong, Hungary, India, Israel, Italy, Mexico, Netherlands, Poland, Portugal, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trials Information
Medpace Finland OY
Outcome results
None listed