Safety Study for Beta Thalassemia Subjects on PTG-300

Phase 2

Completed

• Conditions: β-thalassemia; Ineffective Erythropoiesis
• Interventions: Drug: PTG-300

• Registration Number: NCT04054921
• Lead Sponsor: Protagonist Therapeutics, Inc.

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.

Study Timeline

• Study Start: 2019-05-10
• Study First Submitted: 2019-08-08
• Study First Submitted QC: 2019-08-12
• Study First Posted: 2019-08-13
• Primary Completion: 2020-07-31
• Study Completion: 2020-07-31
• Status Verified: 2021-02
• Disposition First Submitted: 2021-07-09
• Disposition First Submitted QC: 2021-07-12
• Last Update Submitted: 2021-07-12
• Disposition First Posted: 2021-07-16
• Last Update Posted: 2021-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

Inclusion Criteria

• NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02.

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Exclusion Criteria

• Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Interventions	PTG-300	PTG-300

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (16)

UCSF Benioff Children's Hospital; 🇺🇸 Oakland, California, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Laiko General Hospital of Athens; 🇬🇷 Athens, Greece

Chronic Care Center; 🇱🇧 Hazmiyeh, Lebanon

Hospital Umum Sarawak; 🇲🇾 Kuching, Malaysia

Chulalongkorn University; 🇹🇭 Pathum Wan, Thailand

Khon Kaen University; 🇹🇭 Khon Kaen, Thailand

Siriraj Hospital Mahidol University; 🇹🇭 Bangkok, Thailand

Naresuan University; 🇹🇭 Phitsanulok, Thailand

University Hospital Farhat Hached; 🇹🇳 Sousse, Tunisia

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi; 🇹🇷 Istanbul, Turkey

Ege Universitesi Tip Fakultesi; 🇹🇷 Izmir, Turkey

Barts Health NHS Trust; 🇬🇧 London, United Kingdom

University General Hospital of Patras; 🇬🇷 Patras, Greece

Hospital Ampang; 🇲🇾 Ampang, Malaysia

Athens General Hospital 'G Gennimatas'; 🇬🇷 Athens, Greece