A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy

Trial Profile

A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy

Recruiting
Phase of Trial: Phase II

Latest Information Update: 11 Aug 2017

At a glance

  • Drugs RG 7916 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms Firefish
  • Sponsors Roche
  • Most Recent Events

    • 08 May 2017 According to a PTC Therapeutics media release, data from the first part of this study are expected to be presented at scientific meetings later this year (2017) and early 2018.
    • 06 Jan 2017 Planned End Date changed from 1 Feb 2020 to 1 May 2020.
    • 05 Jan 2017 Status changed from not yet recruiting to recruiting, according to a PTC Therapeutics media release.
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