At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 40 enrolled
Drug / intervention
branaplamdrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy
In Brief
A Phase 2 clinical trial evaluating branaplam for Spinal Muscular Atrophy. Completed, enrolled 40 participants across 14 sites in 7 countries.
Detailed 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 was 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 was safe for long term use as well as that can provide durable efficacy optimal dosing regimen in patients with Type 1 SMA.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesBelgium, Bulgaria, Denmark, Germany, Italy, Poland, Russia
Collaborators--
Timeline
Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedOct 2014
Enrollment StartApr 2015
Primary CompletionDec 2022
TodayJul 2026
First PostedOct 20, 2014
Enrollment StartApr 2, 2015
Primary CompletionDec 29, 2022
TodayJul 2, 2026
Enrollment to primary: 7.7 yearsPosted 11.7 years ago
Interventions
branaplamdrug