PTG-300 in NTD and TD β-Thalassaemia patients with Chronic Anaemia
Research type
Research Study
Full title
A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects with Chronic Anemia
IRAS ID
251365
Contact name
John Porter
Contact email
Sponsor organisation
Protagonist Therapeutics, Inc.
Eudract number
2018-001984-21
Clinicaltrials.gov Identifier
137605, IND number
Duration of Study in the UK
1 years, 10 months, 8 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 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, other than red blood cell transfusions, there is no treatment for the anaemia in β-thalassaemia. For this reason, PTG-300 is being developed for the treatment of chronic anaemia in patients with β-thalassaemia.
PTG 300 has been tested for safety in healthy volunteers; and this will be the first study in patients.The purpose of this study is to test the safety and effectiveness of PTG 300 for the treatment of chronic anaemia in β-thalassemia and to evaluate the appropriate dose regimen.
Five PTG-300 dose levels are planned to be tested in both NTD and TD patients: 3mg weekly, 10mg weekly, 20mg weekly, 40mg weekly and 40mg every 2 weeks. Participants and researchers will know which dose is being issued.
This study will include male and female patients, 12-65 years of age. PTG-300 will be first tested in patients older than 18, and then in patients 12 to 17 years old.
The safety of PTG-300 will be monitored by a Safety Monitoring Committee (SMC) that will decide if the study can continue.
This 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 A Research Ethics Committee
REC reference
18/SC/0614
Date of REC Opinion
17 Dec 2018
REC opinion
Further Information Favourable Opinion