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Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)

Trial Profile

Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 06 Dec 2019

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At a glance

  • Drugs SRK-015 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Proof of concept; Therapeutic Use
  • Acronyms TOPAZ
  • Sponsors Scholar Rock
  • Most Recent Events

    • 19 Nov 2019 According to an Scholar Rock Media release, the interim results are expected in the first half of 2020 with top-line results for the full 12-month treatment period expected starting in the fourth quarter of 2020 and through the first quarter of 2021.
    • 19 Nov 2019 Results (n=29) presented in a Scholar Rock Media release.
    • 03 Jun 2019 According to an Scholar Rock Media release, data from an earlier Phase 1 trial of SRK-015 in healthy volunteers will support the evaluation for this trial.
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