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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04054921 |
Recruitment Status :
Recruiting
First Posted : August 13, 2019
Last Update Posted : December 4, 2019
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
β-thalassemia Ineffective Erythropoiesis | Drug: PTG-300 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 192 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 |
Estimated Primary Completion Date : | May 10, 2021 |
Estimated Study Completion Date : | May 10, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Interventions
PTG-300
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Drug: PTG-300
PTG-300 is ahepcidin Mimetic
Other Name: Experimental Part 1 |
- 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.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Years to 65 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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).

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
Contact: Nishit Modi, PhD | 1-888-899-1543 | ptgxclintrials@ptgx-inc.com |
United States, California | |
UCSF Benioff Children's Hospital | Recruiting |
Oakland, California, United States, 94609 | |
United States, Massachusetts | |
Boston Children's Hospital | Recruiting |
Boston, Massachusetts, United States, 02215 | |
Greece | |
Laiko General Hospital of Athens | Recruiting |
Athens, Greece, 11526 | |
Athens General Hospital 'G Gennimatas' | Recruiting |
Athens, Greece, 11527 | |
University General Hospital of Patras | Recruiting |
Patras, Greece, 26500 | |
Lebanon | |
Chronic Care Center | Recruiting |
Hazmiyeh, Lebanon | |
Malaysia | |
Hospital Ampang | Recruiting |
Ampang, Malaysia, 68000 | |
Hospital Umum Sarawak | Recruiting |
Kuching, Malaysia, 93586 | |
Thailand | |
Siriraj Hospital Mahidol University | Recruiting |
Bangkok, Thailand, 10700 | |
Khon Kaen University | Recruiting |
Khon Kaen, Thailand, 40002 | |
Chulalongkorn University | Recruiting |
Pathum Wan, Thailand, 10330 | |
Naresuan University | Recruiting |
Phitsanulok, Thailand, 65000 | |
Tunisia | |
University Hospital Farhat Hached | Recruiting |
Sousse, Tunisia, 4000 | |
Turkey | |
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi | Recruiting |
Istanbul, Turkey, 34093 | |
Ege Universitesi Tip Fakultesi | Recruiting |
Izmir, Turkey, 35100 | |
United Kingdom | |
Barts Health NHS Trust | Recruiting |
London, United Kingdom, E1 1BB |
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: | December 4, 2019 |
Last Verified: | December 2019 |
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 |
Chronic anemia β-thalassemia |
Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |