Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia (TRANSCEND)
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|ClinicalTrials.gov Identifier: NCT03802201|
Recruitment Status : Completed
First Posted : January 14, 2019
Last Update Posted : July 16, 2021
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|Condition or disease||Intervention/treatment||Phase|
|β-thalassemia Ineffective Erythropoiesis Chronic Anemia||Drug: PTG-300||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||63 participants|
|Intervention Model:||Sequential Assignment|
|Intervention Model Description:||
Multiple PTG-300 dose levels/regimens are planned to be tested for each subpopulation of β thalassemia (NTD and TD) on separate arms:
Additional Cohorts (Cohorts 5,6,9,10, and 11) will include subjects between 12 years of age and less than 18 years of age.
|Masking:||None (Open Label)|
|Official Title:||A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia|
|Actual Study Start Date :||December 19, 2018|
|Actual Primary Completion Date :||July 31, 2020|
|Actual Study Completion Date :||July 31, 2020|
Experimental: PTG-300 Active
Drug: PTG-300 Subcutaneous
- NTD: Proportion of responders at each dose [ Time Frame: 4 week period ]NTD subjects who achieve an increase in Hgb without transfusion
- TD: Proportion of clinical responders at each dose [ Time Frame: 8 week period ]TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period
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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|
Main Inclusion Criteria:
- Male and female subjects aged 18 to 65 years, inclusive (Cohorts 1-4b).
- Male and female subjects aged 12-<18 years, with a minimum weight of 30 kg (Cohorts 5 and 6).
- Documented diagnosis of β-thalassemia with no other Hgb abnormality.
Inclusion criteria applicable only for NTD β-thalassemia subjects:
- Mean Hgb < 10.0 g/dL of two measurements (one performed 7-28 days prior to dosing and the other performed within 7 days prior to dosing).
- Requirement of < 6 units RBC transfusion in a 24 week period with the last transfusion at least 8 weeks before screening.
Inclusion criteria applicable only for TD β-thalassemia subjects:
- Transfusion requirement of at least 6 units of RBC in the 24 weeks prior to screening with no transfusion free period > 45 days.
- Last RBC transfusion 5-10 days prior to dosing.
Main Exclusion Criteria:
- Subjects with Sickle Cell disease, Hgb H, Hb Bart's hydrops foetalis or hemoglobin S
- Infection requiring hospitalization or IV antimicrobial therapy, or opportunistic infection within 6 months of dosing, any infection requiring antimicrobial therapy within 2 weeks of dosing; history of infection with human immunodeficiency virus (HIV).
- Subject has a concurrent clinically significant, unstable or uncontrolled cardiovascular, pulmonary, hepatic, renal, gastrointestinal, genitourinary, hematological, coagulation, immunological, endocrine/metabolic or other medical disorder that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the subject by their participation in the study.
- Known primary or secondary immunodeficiency.
History within 6 months of screening of any of the following:
myocardial infarction, unstable angina, transient ischemic attack, decompensated heart failure requiring hospitalization, congestive heart failure (New York Heart Association Class 3 or 4), uncontrolled arrhythmias, cardiac revascularization, stroke, uncontrolled hypertension (resting systolic blood pressure [BP] > 160mmHg or resting diastolic BP > 100mmHg on more than one occasion) or uncontrolled diabetes (Hgb A1c > 9% or > one episode of severe hypoglycemia).
- Pregnant or lactating females.
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): NCT03802201
|Responsible Party:||Protagonist Therapeutics, Inc.|
|Other Study ID Numbers:||
|First Posted:||January 14, 2019 Key Record Dates|
|Last Update Posted:||July 16, 2021|
|Last Verified:||July 2021|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn