The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Active
Scientia Clinical Research Ltd
Sydney, New South Wales, Australia
RECRUITINGDoherty Clinical Trials
East Melbourne, Victoria, Australia
RECRUITINGNucleus Network
Melbourne, Victoria, Australia
RECRUITINGFrequency of treatment-emergent adverse events (TEAE)
Time frame: Up to Week 12
Area under the curve (AUClast, AUCinf)
Time frame: Up to Week 12
Maximum concentration (Cmax)
Time frame: Up to Week 12
Time to Cmax (Tmax)
Time frame: Up to Week 12
Half-life (t1/2)
Time frame: Up to Week 12
Frataxin expression at baseline and after treatment will be measured
Time frame: Up to Week 12
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