An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Condition

Spinal Muscular Atrophy

Interventions

Drug:branaplam

ClinicalTrials.gov identifier

NCT02268552

Novartis reference number

CLMI070X2201

Phase

Phase 1/Phase 2

This study is currently recruiting participants.

Official title

An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy

Purpose

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.

Contacts and locations

Novartis contact

Novartis Pharmaceuticals 1-888-669-6682

Belgium

  • Novartis Investigative Site
    Gent , Belgium , 9000
  • Novartis Investigative Site
    Leuven , Belgium , 3000

Czechia

Denmark

Italy

Poland

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