This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
32
Intravenous repeating dose
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
Time frame: 48 weeks
Incidence and severity of infusion-related reacations (IRRs)
Time frame: 48 weeks
PK parameter: Maximum concentration (Cmax) of DNL952 in serum
Time frame: 48 weeks
PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum
Time frame: 48 weeks
PK Parameter: Area under the concentration-time curve (AUC) from time zero to time of last measurable concentration (AUClast) of DNL952 in serum
Time frame: 48 weeks
PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum
single dose only
Time frame: 48 weeks
PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum
multiple doses only
Time frame: 48 weeks
PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum
Time frame: 48 weeks
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