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

• ClinicalTrials.gov Identifier: NCT04054921
• Recruitment Status: Completed
• First Posted: August 13, 2019
• Last Update Posted: July 16, 2021
• Sponsor: Protagonist Therapeutics, Inc.
• Information provided by (Responsible Party): Protagonist Therapeutics, Inc.

Tracking Information

• First Submitted Date: August 8, 2019
• First Posted Date: August 13, 2019
• Last Update Posted Date: July 16, 2021
• Actual Study Start Date: May 10, 2019
• Actual Primary Completion Date: July 31, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 12, 2019)

Proportion of subjects with side effects and severity of side effects will be tabulated [ Time Frame: Over two year Period after receiving PTG-300 ]

the long-term safety and tolerability of PTG-300 in Beta Thalassemia.

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Safety Study for Beta Thalassemia Subjects on PTG-300
• Official Title: An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects
• 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 Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• β-thalassemia
• Ineffective Erythropoiesis

Intervention

Drug: PTG-300

PTG-300 is a hepcidin Mimetic

Study Arms

Experimental: Interventions

PTG-300

Intervention: Drug: PTG-300

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 4, 2021): 34
• Original Estimated Enrollment (submitted: August 12, 2019): 192
• Actual Study Completion Date: July 31, 2020
• Actual Primary Completion Date: July 31, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

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).

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Greece, Lebanon, Malaysia, Thailand, Tunisia, Turkey, United Kingdom, United States

Administrative Information

• NCT Number: NCT04054921
• Other Study ID Numbers: PTG-300-03
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Protagonist Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Protagonist Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Protagonist Therapeutics, Inc.
• Verification Date: February 2021