Chronic anemia due to ineffective erythropoiesis (IE) in subjects with ß thalassemia Thalassemia
Conditions
Interventions
80 mg/week
80 mg every 2 weeks
40 mg every 2 weeks
Subjects rolling over from the PTG-300-02 study, who meet the response criteria defined for this study at the last dose received in study PTG-300-02, will continue to receive the same dose in PTG-300-
3mg/week
10mg/week
20mg/week
40mg/week
Sponsors
Protagonist Therapeutics Inc
Eligibility
Sex/Gender
All
Age
12 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1. NTD and TD ß-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02. 2. Women of childbearing potential (WOCBP) and men agree to use a highly effective contraceptive measure (base on the Clinical Trial Facilitation Group [CTFG]) during the duration of the study and for 4 weeks after the last dose of study drug in the case of women and 90 days after the last dose of study drug in the case of men. 3. For WOCBP, a negative urine pregnancy test within 24 hours prior to the first dose of study medication in this study. 4. Subjects or legal guardians (in the case of minors) understand the study procedures and agree to participate in the study by giving written informed consent. 5. Subjects, or legal representative (in the case of minors) are willing and able to adhere to the study visit schedule and other protocol requirements. 6. Subjects between 12-<18 years of age understand and provide the assent to participate in the study, according to local guidelines.
Exclusion criteria
Exclusion criteria: 1. Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD) 2. Clinically meaningful laboratory abnormalities at Screening. 3. Pregnant or lactating females. 4. Current history of alcohol dependence or illicit drug use. 5. Subject has a concurrent clinically significant, unstable or uncontrolled cardiovascular, pulmonary, hepatic, renal, gastrointestinal, genitourinary, hematological, coagulation, immunological, endocrine/metabolic or other medical disorder that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the subject by their participation in the study. 6. Subject is mentally or legally incapacitated at the time of Screening visit or has a history of clinically significant psychiatric disorders that would impact the subject’s ability to participate in the trial according to the Investigator. 7. Concurrent participation in any other interventional study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Name: NTD Subjects who achieve an increase in Hgb =1.0 g/dL ;Timepoints: from pre-treatment baseline without transfusion;Measure: Hemoglobin Test;Name: NTD patients: Hgb change ;Timepoints: from pre-treatment baseline;Measure: Hemoglobin Test;Name: TD Subjects who achieve a =20% reduction in the red blood cell (RBC) units transfused over 8-week period compared to pre-treatment baseline;Timepoints: over an 8-week period;Measure: RBC units transfused;Name: TD patients: Change in the number of units of RBC required;Timepoints: from pre-treatment baseline;Measure: RBC units transfused | — |
Secondary
| Measure | Time frame |
|---|---|
| Name: NTD patients: Proportion of subjects who achieve an increase in Hgb =1.5 g/dL;Timepoints: from pre-treatment baseline without transfusion;Measure: Hgb test;Name: Proportion of subjects who achieve a maintenance dose;Timepoints: from pre-treatment baseline;Measure: Dose;Name: Hgb level;Timepoints: from pre-treatment baseline;Measure: Hgb test;Name: NTD Patients: Duration of Hgb change of =1.0 g/dL;Timepoints: from pre-treatment baseline without transfusion;Measure: Hgb test;Name: NTD Patients: Duration of Hgb change of =1.5 g/dL from;Timepoints: from pre-treatment baseline without transfusion;Measure: Hgb test;Name: Change in the following PD parameters ;Timepoints: from pre-treatment baseline;Measure: serum iron, ferritin, transferrin saturation (TSAT);Name: TD Patients: Proportion of subjects who achieve = 33% reduction in the RBC units required over an 8-week period;Timepoints: over an 8-week period;Measure: RBC units transfused;Name: TD Patients: Duration of response defined as = 20% reduction in the RBC units;Timepoints: over an 8-week period;Measure: RBC units transfused;Name: TD Patients: Number of RBC units required;Timepoints: from pre-treatment baseline;Measure: RBC units transfused;Name: TD Patients: Percent change in the RBC units required;Timepoints: from pre-treatment baseline;Measure: RBC units transfused;Name: TD: Hgb change ;Timepoints: from pre-treatment baseline;Measure: Hgb test | — |
Countries
Greece, Italy, Lebanon, Malaysia, Thailand, Tunisia, Turkey, United Kingdom, United States of America
Contacts
Public ContactAziz Zoghbi
Regional Manager
Outcome results
None listed