3 results
Approved WMOWill not start
Primary* To estimate the effect of NME combination therapies on inducing a functional cure over the control arm.Secondary * To characterize the efficacy profile of NME combination therapies.* To characterize the PD profile of NME combination…
Approved WMORecruiting
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.
Approved WMOCompleted
Primary: • Assessment of the safety and tolerability of PLN-74809 Secondary:• Assessment of pharmacokinetics (PK) of PLN-74809 Exploratory:• Assessment of change from baseline in forced vital capacity (FVC) • Assessment of change from baseline in…