An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
Status:
Active, not recruiting
Trial end date:
2023-02-01
Target enrollment:
Participant gender:
Summary
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1
spinal muscular atrophy. The purpose of this study is to evaluate the safety, tolerability,
pharmacokinetics (PK), pharmacodynamics (PD) and efficacy after 13 weeks; and to estimate the
Maximum Tolerated Dose (MTD) of orally administered branaplam; and to identify the dose that
is safe for long term use as well as that can provide durable efficacy optimal dosing regimen
in patients with Type 1 SMA.