Polycythemia Vera
Conditions
Brief summary
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%.
Detailed description
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 at end of Part 1a (Week 32) in the Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 8a total T-score, 4 - Mean change from baseline at end of Part 1a (Week 32) in the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF), 5 - Mean change from baseline at end of Part 1a (Week 32) in the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF)
Interventions
DRUGRusfertide 45mg
DRUGPlacebo to Match PTG-300 for Injection. Formulation (lyophilized powder) is based upon that of PTG-300 for Injection
DRUGwith the drug substance omitted.
DRUGRusfertide 30mg
DRUGRusfertide 10mg
DRUGRusfertide 60mg
DRUGRusfertide 20mg
Sponsors
Protagonist Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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%. | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 at end of Part 1a (Week 32) in the Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 8a total T-score, 4 - Mean change from baseline at end of Part 1a (Week 32) in the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF), 5 - Mean change from baseline at end of Part 1a (Week 32) in the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) | — |
Countries
Austria, Belgium, Czechia, France, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Spain
Outcome results
None listed