Clinical trial design of anti-myostatin for neuromuscular disorders
Roche
Non-confidential report
Roche requested advice on clinical trial design focusing an anti-myostatin agent, which is considered as future potential treatment for neuromuscular disorders.
TACT offered comprehensive advice on the proposal, including discussion of functional benefit linked to muscle mass increase and variation threshold feasible to be assessed in MRI, validation of animal studies, stratification of patient cohorts based on plasma myostatin level, outcome measure selection, trial design and regulatory considerations.
TACT welcomed the opportunity to give advice on this potentially interesting and novel approach for various neuromuscular disorders, independently of the underlying genetic defect.
Name of applicant: Ksenija Gorni
Reviewed: April, 2019 in Glasgow, UK
Roche requested advice on clinical trial design focusing an anti-myostatin agent, which is considered as future potential treatment for neuromuscular disorders.
TACT offered comprehensive advice on the proposal, including discussion of functional benefit linked to muscle mass increase and variation threshold feasible to be assessed in MRI, validation of animal studies, stratification of patient cohorts based on plasma myostatin level, outcome measure selection, trial design and regulatory considerations.
TACT welcomed the opportunity to give advice on this potentially interesting and novel approach for various neuromuscular disorders, independently of the underlying genetic defect.