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.
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort and with the potential for individual titration (dose increase or decrease) within each cohort. The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment of chronic anemia in β-thalassemia and to evaluate the appropriate dosing regimen for PTG-300 in the target population
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
63
Subcutaneous (SC)
NTD: Proportion of responders at each dose
NTD subjects who achieve an increase in Hgb without transfusion
Time frame: 4 week period
TD: Proportion of clinical responders at each dose
TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period
Time frame: 8 week period
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Athens General Hospital 'G Gennimatas'
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Hippokration Hospital
Thessaloniki, Greece
Presidio Ospedaliero Antonio Perrino
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Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
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AOU dell'Università degli Studi della Campania Luigi Vanvitelli
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...and 23 more locations