Beta-Thalassemia Clinical Trial
Official title:
A Phase Ib, Open-label, Repeat Dose, Study of EPEG in Beta Thalassemia Patients With Non-transfusion Dependent Thalassemia (β NTDT)
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
Following the provision of informed consent, screening visit procedures to be performed will
include: a detailed medical history (including concomitant medications), physical exam, vital
signs (non-invasive systolic and diastolic arterial blood pressure, heart rate, and
respiratory rate, temperature, and oxygen saturation by pulse oximetry,), laboratory testing
of blood samples collections for safety (hematology and chemistry), and urinalysis (dipstick
and microscopy, if necessary). A serum pregnancy test for all female patients (of
child-bearing potential) will be measured during the Screening Visit. Urine pregnancy test
for all female patients (of child-bearing potential) will be measured at each dosing visit
prior to dosing.
Eligible patients will receive either subcutaneous injection of EPEG (0.9 µg/kg, 1.2 µg/kg,
and 1.5 µg/kg,) for four weeks followed by follow up for 5-6 weeks after 4th dose of IP.
Vital signs will be recorded for study documentation at 1 hour after dosing and at discharge
of the day (to occur 2 hours after the time of dosing). All patients will receive standard of
care as per investigative site standard practice.
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