Safety Study for Beta Thalassemia Subjects on PTG-300 (TRANSCEND)
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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. |
| ClinicalTrials.gov Identifier: NCT04054921 |
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Recruitment Status :
Completed
First Posted : August 13, 2019
Last Update Posted : February 8, 2021
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Sponsor:
Protagonist Therapeutics, Inc.
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.
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Brief Summary:
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| β-thalassemia Ineffective Erythropoiesis | Drug: PTG-300 | Phase 2 |
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 (Clinical Trial) |
| Actual Enrollment : | 34 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects |
| Actual Study Start Date : | May 10, 2019 |
| Actual Primary Completion Date : | July 31, 2020 |
| Actual Study Completion Date : | July 31, 2020 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Beta thalassemia
MedlinePlus related topics:
Thalassemia
| Arm | Intervention/treatment |
|---|---|
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Experimental: Interventions
PTG-300
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Drug: PTG-300
PTG-300 is ahepcidin Mimetic
Other Name: Experimental Part 1 |
Primary Outcome Measures :
- 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.
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| Ages Eligible for Study: | 12 Years to 65 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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).
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04054921
Locations
| United States, California | |
| UCSF Benioff Children's Hospital | |
| Oakland, California, United States, 94609 | |
| United States, Massachusetts | |
| Boston Children's Hospital | |
| Boston, Massachusetts, United States, 02215 | |
| Greece | |
| Laiko General Hospital of Athens | |
| Athens, Greece, 11526 | |
| Athens General Hospital 'G Gennimatas' | |
| Athens, Greece, 11527 | |
| University General Hospital of Patras | |
| Patras, Greece, 26500 | |
| Lebanon | |
| Chronic Care Center | |
| Hazmiyeh, Lebanon | |
| Malaysia | |
| Hospital Ampang | |
| Ampang, Malaysia, 68000 | |
| Hospital Umum Sarawak | |
| Kuching, Malaysia, 93586 | |
| Thailand | |
| Siriraj Hospital Mahidol University | |
| Bangkok, Thailand, 10700 | |
| Khon Kaen University | |
| Khon Kaen, Thailand, 40002 | |
| Chulalongkorn University | |
| Pathum Wan, Thailand, 10330 | |
| Naresuan University | |
| Phitsanulok, Thailand, 65000 | |
| Tunisia | |
| University Hospital Farhat Hached | |
| Sousse, Tunisia, 4000 | |
| Turkey | |
| Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi | |
| Istanbul, Turkey, 34093 | |
| Ege Universitesi Tip Fakultesi | |
| Izmir, Turkey, 35100 | |
| United Kingdom | |
| Barts Health NHS Trust | |
| London, United Kingdom, E1 1BB | |
Sponsors and Collaborators
Protagonist Therapeutics, Inc.
| Responsible Party: | Protagonist Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT04054921 |
| Other Study ID Numbers: |
PTG-300-03 |
| First Posted: | August 13, 2019 Key Record Dates |
| Last Update Posted: | February 8, 2021 |
| Last Verified: | February 2021 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Protagonist Therapeutics, Inc.:
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Chronic anemia β-thalassemia |
Additional relevant MeSH terms:
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Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |