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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04054921
Recruitment Status : Completed
First Posted : August 13, 2019
Last Update Posted : July 16, 2021
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE August 8, 2019
First Posted Date  ICMJE August 13, 2019
Last Update Posted Date July 16, 2021
Actual Study Start Date  ICMJE May 10, 2019
Actual Primary Completion Date July 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Safety Study for Beta Thalassemia Subjects on PTG-300
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • β-thalassemia
  • Ineffective Erythropoiesis
Intervention  ICMJE Drug: PTG-300
PTG-300 is a hepcidin Mimetic
Study Arms  ICMJE Experimental: Interventions
Intervention: Drug: PTG-300
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 4, 2021)
Original Estimated Enrollment  ICMJE
 (submitted: August 12, 2019)
Actual Study Completion Date  ICMJE July 31, 2020
Actual Primary Completion Date July 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Greece,   Lebanon,   Malaysia,   Thailand,   Tunisia,   Turkey,   United Kingdom,   United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT04054921
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: No
Current Responsible Party Protagonist Therapeutics, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Protagonist Therapeutics, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Protagonist Therapeutics, Inc.
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP