NCT03802201

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia

A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 63 (actual)
• Sponsor: Protagonist Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment in β-thalassemia.

Detailed description

This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort and with the potential for individual titration (dose increase or decrease) within each cohort. The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment of chronic anemia in β-thalassemia and to evaluate the appropriate dosing regimen for PTG-300 in the target population

Conditions

• β-thalassemia
• Ineffective Erythropoiesis
• Chronic Anemia

Interventions

Type	Name	Description
DRUG	PTG-300	Subcutaneous (SC)

Timeline

• Start date: 2018-12-19
• Primary completion: 2020-07-31
• Completion: 2020-07-31
• First posted: 2019-01-14
• Last updated: 2021-07-16

Locations

33 sites across 9 countries: United States, Greece, Italy, Lebanon, Malaysia, Thailand, Tunisia, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study