Phase 2, randomized, double-blind, placebo-controlled study
Topic / Pathology
- SLA
- Phase 2
Objectives
Primary objective : to evaluate the effect of PTC857 in reducing disease progression.
Secondary objectives :
To evaluate the following in subjects with ALS :
1. Safety and tolerability of PTC857
2. Respiratory function in subjects randomized to PTC857 versus placebo
3. Motor and bulbar function in subjects randomized to PTC857 versus placebo
4. Neuropsychological function in subjects randomized to PTC857 versus placebo
5. Survival in subjects randomized to PTC857 versus placebo
6. Quality of life in subjects randomized to PTC857 versus placebo
7. Pharmacokinetics (PK) of PTC857
Find out more : https://clinicaltrials.gov/ct2/show/NCT05349721
After completing the 24-week treatment period, participants have the option of receiving PTC857 open-label during the 28-week long-term extension period.
Sponsor
PTC Therapeutics
Investigator
National coordinator : Dr Véronique Danel
Status
Completed
