Safety Study for Beta Thalassemia Subjects on PTG-300 (TRANSCEND)

Protagonist Therapeutics

Status and phase: Completed; Phase 2

Conditions: Ineffective Erythropoiesis; β-thalassemia

Treatments: Drug: PTG-300

Study type: Interventional

Funder types: Industry

Identifiers

NCT04054921

PTG-300-03

Details and patient eligibility

About

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

Full 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.

Enrollment: 34 patients

Sex: All

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• NTD and TD β-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).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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34 participants in 1 patient group

Interventions

Experimental group

Description:

PTG-300

Treatment:

Drug: PTG-300