Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects… (NCT03802201) | Clinical Trial Compass
CompletedPhase 2
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
United States, Greece63 participantsStarted 2018-12-19
Plain-language summary
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment in β-thalassemia.
Who can participate
Age range12 Years – 65 Years
SexALL
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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.
✓. 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.
✓. 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.
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.
What they're measuring
1
NTD: Proportion of responders at each dose
Timeframe: 4 week period
2
TD: Proportion of clinical responders at each dose