VBP15-LTE

• Research type

  Research Study

• Full title

  A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

• IRAS ID

  237110

• Contact name

  Michela Guglieri

• Contact email

  michela.guglieri@newcastle.ac.uk

• Sponsor organisation

  ReveraGen BioPharma Inc.

• Eudract number

  2017-003568-10

• Clinicaltrials.gov Identifier

  NCT03038399

• Clinicaltrials.gov Identifier

  NA, NA

• Duration of Study in the UK

  2 years, 2 months, 22 days

• Research summary

  This long-term extension (LTE) study is an open-label, multicenter study to evaluate the long-term safety, tolerability, clinical efficacy, and pharmacodynamics of vamorolone at dose levels up to 6.0 mg/kg administered daily by liquid oral suspension over a Treatment Period of 24 months to young boys with Duchenne Muscular Dystrophy. Only subjects who have already taken part in a Phase IIa extension Study of vamorolone will be eligible for enrolment into this study. The LTE study is comprised of a Pretreatment Baseline Period of up to 24 hours, which begins with the signing of an informed consent form (ICF) by parents or guardians, a 24-month Treatment Period, and an up to 5-week Dose-tapering Period for subjects who decide to stop taking vamorolone treatment at the end of the study.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  17/NE/0367

• Date of REC Opinion

  5 Jan 2018

• REC opinion

  Further Information Favourable Opinion