Ineffective Erythropoiesis, β-thalassemia
Conditions
Keywords
Chronic anemia, β-thalassemia
Brief summary
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
Detailed description
The PTG-300-03 study will study beta thalassemia patients on PTG-300 for two years. The dose and dose frequency for each subject enrolling in the PTG-300-03 study will be based on the last dose and frequency received by the subject in the previous study PTG-300-02 and the subject's response at that dose. Patients dose will be increased in a manner similar to that used on PTG-300-02 study. The maximum PTG-300 dose allowed is 80 mg/week.
Interventions
DRUGPTG-300
PTG-300 is a hepcidin Mimetic
Sponsors
Protagonist Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02.
Exclusion criteria
* Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of subjects with side effects and severity of side effects will be tabulated | Over two year Period after receiving PTG-300 | the long-term safety and tolerability of PTG-300 in Beta Thalassemia. |
Countries
Greece, Lebanon, Malaysia, Thailand, Tunisia, Turkey (Türkiye), United Kingdom, United States
Outcome results
None listed