Safety Study for Beta Thalassemia Subjects on PTG-300

ß-thalassemia Clinical Trial

— TRANSCEND  

Official title:

An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04054921
• Other study ID #: PTG-300-03
• Status: Completed
• Phase: Phase 2
• Start date: May 10, 2019
• Est. completion date: July 31, 2020

Study information

• Verified date: February 2021
• Source: Protagonist Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: July 31, 2020
• Est. primary completion date: July 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

Inclusion Criteria:

• NTD and TD ß-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).

Related Conditions & MeSH terms

• beta-Thalassemia
• Ineffective Erythropoiesis
• ß-thalassemia
• Thalassemia

Intervention

Drug:
• PTG-300
PTG-300 is a hepcidin Mimetic

Locations

Country	Name	City	State
Greece	Athens General Hospital 'G Gennimatas'	Athens
Greece	Laiko General Hospital of Athens	Athens
Greece	University General Hospital of Patras	Patras
Lebanon	Chronic Care Center	Hazmiyeh
Malaysia	Hospital Ampang	Ampang
Malaysia	Hospital Umum Sarawak	Kuching
Thailand	Siriraj Hospital Mahidol University	Bangkok
Thailand	Khon Kaen University	Khon Kaen
Thailand	Chulalongkorn University	Pathum Wan
Thailand	Naresuan University	Phitsanulok
Tunisia	University Hospital Farhat Hached	Sousse
Turkey	Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi	Istanbul
Turkey	Ege Universitesi Tip Fakultesi	Izmir
United Kingdom	Barts Health NHS Trust	London
United States	Boston Children's Hospital	Boston	Massachusetts
United States	UCSF Benioff Children's Hospital	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
Protagonist Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Greece,  Lebanon,  Malaysia,  Thailand,  Tunisia,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of subjects with side effects and severity of side effects will be tabulated	the long-term safety and tolerability of PTG-300 in Beta Thalassemia.	Over two year Period after receiving PTG-300