BN43703: RO7204239 vs placebo in FSHD

• Research type

  Research Study

• Full title

  A PHASE II, MULTICENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO EVALUATE THE PHARMACODYNAMICS, SAFETY, TOLERABILITY, PHARMACOKINETICS, AND EFFICACY OF RO7204239 IN PARTICIPANTS WITH FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY

• IRAS ID

  1005836

• Contact name

  Abira Suganthan

• Contact email

  welwyn.uk_ethics@roche.com

• Sponsor organisation

  F.Hoffmann-La Roche Ltd

• Eudract number

  2021-006255-34

• Clinicaltrials.gov Identifier

  NCT05115110

• Research summary

  Lay Summary
  Background (What research question is being addressed? How is it of relevance and importance to patients and public?)
  
  The purpose of this study is to compare the effects, good or bad, of RO7204239 versus placebo on patients with facioscapulohumeral muscular dystrophy (FSHD). There are no approved treatments for FSHD and management of the disease remains largely supportive, including exercise, bone health, pain management, bracing, and surgery. Therefore, there continues to exist a high unmet need for treatments that aim to improve muscle function and improve patients’ motor abilities required for daily living.

  In this study, participants will receive either RO7204239 (the study drug) or placebo every 4 weeks for 1 year. A placebo looks like a drug but has no active ingredient. Both RO7204239 and placebo are given as a subcutaneous injection in the abdomen. Neither participants nor the study doctor can choose or know the group you are in while the study is ongoing. However, your study doctor can find out which group you are in, if your safety is at risk

  Participants will have an equal chance of receiving RO7204239 or placebo.

  After 1 year of treatment, all participants (whether they initially received RO7204239 or placebo) may have the option to receive RO7204239 for a further year.

  Eligible patients will be male or female, between 18 and 65 years of age, have Genetic confirmation of FSHD1 or FSHD2 and able to walk unassisted for 10 metres. A full list of eligibiklity criteria is outlined in the study protocol.
  
  Approximately 4 participants will take part in the study in the UK and 48 worldwide.
  
  The study is sponsored by F. Hoffmann- La Roche

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  22/EM/0146

• Date of REC Opinion

  9 Sep 2022

• REC opinion

  Further Information Favourable Opinion