BN42644 - SMA in Pediatric Patients (MANATEE)

• Research type

  Research Study

• Full title

  A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY

• IRAS ID

  1004228

• Contact name

  Head of EU Regulatory

• Contact email

  global.eudract@roche.com

• Sponsor organisation

  F.Hoffmann-La Roche Ltd

• Eudract number

  2021-003417-19

• Clinicaltrials.gov Identifier

  NCT05115110

• Research summary

  Spinal muscular atrophy (SMA) is a genetic condition makes muscles weaker and causes problems with movement. There is currently no cure for SMA. Despite the development and approval of disease modifying therapies, an unmet medical need still exists in SMA. Treated patients may not fully regain motor abilities that have been lost prior to treatment initiation.

  The purpose of this study is to compare the effects, good or bad, of a new drug called GYM329 (RO7204239) with Risdiplam compared with placebo with Risdiplam in participants with spinal muscular atrophy. In this study, a child will receive either GYM329 and Risdiplam, or placebo and risdiplam. A placebo looks like a drug but has no active ingredient.
  GYM329 is a new experimental drug, which is not yet approved by the health authorities for the treatment of spinal muscular atrophy. Risdiplam has been approved in the United States, European Union, and other countries for the treatment of spinal muscular atrophy.
  This study has two parts. Part 1, will start first to establish the best of two doses of GYM329. The best dose will be chosen for part 2 of the study. Then part 2 will start.

  Approximately 12 participants will take part in the study from 3 hospitals in the UK and 180 worldwide.

  To be eligible, participants must have been diagnosed with spinal muscular atrophy and each child will be in the study for approximately 4.5 years.
  The study is sponsored by F. Hoffman La Roche
  Research Summary; Version Number 1, 27-September- 2021

• REC name

  East Midlands - Leicester Central Research Ethics Committee

• REC reference

  21/EM/0256

• Date of REC Opinion

  5 Jan 2022

• REC opinion

  Further Information Favourable Opinion