Continuation study of PTG-300 in NTD and TD β-Thalassaemia patients
Research type
Research Study
Full title
An Open Label Extension Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-thalassemia Subjects
IRAS ID
262411
Contact name
John Porter
Contact email
Sponsor organisation
Protagonist Therapeutics, Inc.
Eudract number
2018-004423-36
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 6 months, 17 days
Research summary
Β-thalassaemia is a blood disorder that reduces the production of red blood cells and the substance that carries oxygen throughout the body called haemoglobin. The low haemoglobin and fewer red blood cells result in chronic anaemia that can then cause pale skin, weakness, fatigue, and more serious complications.
Some patients require regular blood transfusions to treat the anaemia, and these patients are considered transfusion dependent (TD). Other patients have a milder form of the disease and do not need regular blood transfusions. These patients are considered non-transfusion dependent (NTD).
Currently, there is no effective treatment for the anaemia in β-thalassaemia in this study population. For this reason, PTG-300, is being developed for the treatment of chronic anaemia in patients with β-thalassaemia.
The purpose of this study is to test the long-term safety and effectiveness of PTG-300 for the treatment of chronic anaemia in βthalassaemia. PTG-300 has been tested for safety in healthy volunteers and is currently being tested in patients with β-thalassaemia blood disorder in the PTG- 300-02 study.This is an open-label long term extension study and will enrol eligible subjects that complete the PTG-300-02 study. The safety evaluation done at the end of study PTG-300-02 will be used to confirm subject eligibility for this study. The PTG-300-03 study will consist of a study drug dose adjustment phase (titration phase) followed by a maintenance phase.
This study will include male and female patients, 12-65 years of age.
The safety of PTG-300 will be monitored by a Safety Monitoring Committee (SMC).
The current PTG-300-03 study is sponsored by Protagonist Therapeutics, Inc. and has a global target population of 84 participants with diagnosis of NTD or TD β-thalassemia. Approximately 7 of these patients will be based in the UK across 2 hospitals.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
19/SC/0248
Date of REC Opinion
4 Jul 2019
REC opinion
Further Information Favourable Opinion