Back to resultsSafety Study for Beta Thalassemia Subjects on PTG-300 (TRANSCEND)
Primary Purpose
β-thalassemia, Ineffective Erythropoiesis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
PTG-300
Sponsored by
About this trial
This is an interventional treatment trial for β-thalassemia focused on measuring Chronic anemia, β-thalassemia
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).
Sites / Locations
- UCSF Benioff Children's Hospital
- Boston Children's Hospital
- Laiko General Hospital of Athens
- Athens General Hospital 'G Gennimatas'
- University General Hospital of Patras
- Chronic Care Center
- Hospital Ampang
- Hospital Umum Sarawak
- Siriraj Hospital Mahidol University
- Khon Kaen University
- Chulalongkorn University
- Naresuan University
- University Hospital Farhat Hached
- Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
- Ege Universitesi Tip Fakultesi
- Barts Health NHS Trust
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Interventions
Arm Description
PTG-300
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Full Information
NCT ID
NCT04054921
First Posted
August 8, 2019
Last Updated
July 12, 2021
Sponsor
Protagonist Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04054921
Brief Title
Safety Study for Beta Thalassemia Subjects on PTG-300
Acronym
TRANSCEND
Official Title
An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects
Study Type
Interventional
2. Study Status
Record Verification Date
February 2021
Overall Recruitment Status
Completed
Study Start Date
May 10, 2019 (Actual)
Primary Completion Date
July 31, 2020 (Actual)
Study Completion Date
July 31, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Protagonist Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
β-thalassemia, Ineffective Erythropoiesis
Keywords
Chronic anemia, β-thalassemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
34 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Interventions
Arm Type
Experimental
Arm Description
PTG-300
Intervention Type
Drug
Intervention Name(s)
PTG-300
Intervention Description
PTG-300 is a hepcidin Mimetic
Primary Outcome Measure Information:
Title
Proportion of subjects with side effects and severity of side effects will be tabulated
Description
the long-term safety and tolerability of PTG-300 in Beta Thalassemia.
Time Frame
Over two year Period after receiving PTG-300
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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).
Facility Information:
Facility Name
UCSF Benioff Children's Hospital
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Laiko General Hospital of Athens
City
Athens
ZIP/Postal Code
11526
Country
Greece
Facility Name
Athens General Hospital 'G Gennimatas'
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
University General Hospital of Patras
City
Patras
ZIP/Postal Code
26500
Country
Greece
Facility Name
Chronic Care Center
City
Hazmiyeh
Country
Lebanon
Facility Name
Hospital Ampang
City
Ampang
ZIP/Postal Code
68000
Country
Malaysia
Facility Name
Hospital Umum Sarawak
City
Kuching
ZIP/Postal Code
93586
Country
Malaysia
Facility Name
Siriraj Hospital Mahidol University
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Khon Kaen University
City
Khon Kaen
ZIP/Postal Code
40002
Country
Thailand
Facility Name
Chulalongkorn University
City
Pathum Wan
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Naresuan University
City
Phitsanulok
ZIP/Postal Code
65000
Country
Thailand
Facility Name
University Hospital Farhat Hached
City
Sousse
ZIP/Postal Code
4000
Country
Tunisia
Facility Name
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Facility Name
Ege Universitesi Tip Fakultesi
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Facility Name
Barts Health NHS Trust
City
London
ZIP/Postal Code
E1 1BB
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety Study for Beta Thalassemia Subjects on PTG-300
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