Roche- RO7239361 [TERMINATED]
Clinical trial to Evaluate the Efficacy, Safety and Tolerability of RO7239361 in Ambulatory Boys with Duchenne muscular dystrophy
Locations:
trialTerminated
Alder Hey
,
trialTerminated
London - GOSH
,
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
Primary Outcome Measures
- Change from baseline in the North Star Ambulatory Assessment (NSAA) total score in RO7239361 treated participants
- Change from baseline in the North Star Ambulatory Assessment (NSAA) total score in placebo treated participants.
Can I take part?
Inclusion Criteria
- Diagnosed with DMD by a blood test
- Able to walk without assistance
- Minimum North Star Ambulatory Assessment (NSAA) score of 15 at screening
- Able to walk up 4 stairs in 8 seconds or less
- Weigh at least 15 kg (33 lbs)
- Taking corticosteroids for DMD
Exclusion Criteria
- Any behaviour or mental issue that will affect the ability to complete the required study procedures
- Previously or currently taking medications like androgens or human growth hormone
- Use of a ventilator during the day
- Unable to have blood samples collected or receive an injection under the skin
Other protocol defined Inclusion/Exclusion Criteria could apply.
Age
6 - 11 years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Ambulation
ambulant