PTC857 for ALS
(CARDINALS Trial)
Trial Summary
What is the purpose of this trial?
This trial is testing a new drug called PTC857 to see if it can help people with ALS. Participants will either receive PTC857 or a non-active substance. If they complete the initial treatment, they can continue taking PTC857 for an extended period to further assess its effects.
Will I have to stop taking my current medications?
You can continue taking your current medications as long as they have been stable for at least 14 days before the study starts and remain unchanged during the study. However, if you are on standard ALS treatments like riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, they need to be stable for 30 days before the study and remain unchanged.
What makes PTC857 unique for treating ALS?
PTC857 is unique for ALS treatment as it is part of a clinical trial that includes a placebo-to-active treatment crossover, which is encouraged in fatal conditions like ALS to better assess the treatment's survival benefit. This approach aims to optimize trial design by potentially reducing sample sizes and placebo exposure, making it different from traditional trial methods.12345
Research Team
Subha Krishnan
Principal Investigator
PTC Therapeutics
Eligibility Criteria
This trial is for people with ALS who have had symptoms for less than 24 months and a score of at least 34 on the ALSFRS-R scale. They must not be pregnant, nursing, or planning pregnancy, have no significant respiratory issues (slow vital capacity ≥60%), and no recent use of certain ALS treatments together. Participants should not have breast cancer history or be in other studies.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive PTC857 or placebo for 24 weeks
Long-term Extension (LTE)
Participants receive open-label PTC857 for 28 weeks
Continued Long-term Extension
Participants receive open-label PTC857 for an additional 108 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Placebo
- PTC857
Find a Clinic Near You
Who Is Running the Clinical Trial?
PTC Therapeutics
Lead Sponsor
Dr. Matthew B. Klein
PTC Therapeutics
Chief Executive Officer since 2023
BA from the University of Pennsylvania, MD from Yale University School of Medicine, MS in Epidemiology from the University of Washington School of Public Health
Dr. Stuart W. Peltz
PTC Therapeutics
Chief Medical Officer since 2023
MD from Harvard Medical School