This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with transfusion-dependent beta thalassemia with myocardial iron overload.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
84
Investigative Site
Oakland, California, United States
Investigative Site
New York, New York, United States
Effect of LJPC-401 on cardiac iron
Change in cardiac T2\* magnetic resonance imaging (MRI)
Time frame: 26 Weeks
Effect of LJPC-401 on cardiac iron
Change in cardiac T2\*MRI
Time frame: 52 Weeks
Effect of LJPC-401 on hepatic iron
Change in hepatic T2\*MRI
Time frame: 52 Weeks
Effect of LJPC-401 on serum iron
Mean change in serum iron
Time frame: 56 Weeks
Effect of LJPC-401 on hemoglobin
Mean change in hemoglobin
Time frame: 52 Weeks
Effect of LJPC-401 on volume of blood transfused
Change in transfused blood volume
Time frame: 56 Weeks
Effect of LJPC-401 on its potential to elicit an immune response
Measured by blood laboratory tests and the presence of anti-drug antibodies
Time frame: 56 Weeks
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-heart rate
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-body temperature
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Investigative Site
Camperdown, New South Wales, Australia
Investigative Site
Liverpool, New South Wales, Australia
Investigative Site
Goudi, Attica, Greece
Investigative Site
Thessaloniki, Macedonia, Greece
Investigative Site
Patra, Peloponnese, Greece
Investigative Site
Cagliari, Italy
Investigative Site
Modena, Italy
Investigative Site
Napoli, Italy
...and 12 more locations
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-respiratory rate
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-blood pressure
Time frame: 56 Weeks
Effect of LJPC-401 on body weight
Change in body weight (kilograms)
Time frame: 56 Weeks