307 Participants Needed

PTC857 for ALS

(CARDINALS Trial)

Recruiting at 62 trial locations
PA
Overseen ByPatient Advocacy
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: PTC Therapeutics
Must be taking: Riluzole, Edaravone, Sodium phenylbutyrate
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human 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

SK

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

You have been diagnosed with either definite ALS or probable ALS based on the EL Escorial criteria.
My lung function is good enough to pass a basic breathing test.
You have been diagnosed with ALS within the past 24 months.
See 4 more

Exclusion Criteria

Current participation in any other investigational study with an investigational product or participation within 30 days prior to the start of the Screening Period or 5 half-lives of the previously taken investigational drug, whichever is longer
I have previously been treated with PTC857.
I have had breast cancer or a close family member has.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive PTC857 or placebo for 24 weeks

24 weeks

Long-term Extension (LTE)

Participants receive open-label PTC857 for 28 weeks

28 weeks

Continued Long-term Extension

Participants receive open-label PTC857 for an additional 108 weeks

108 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Placebo
  • PTC857
Trial OverviewThe study tests PTC857's effectiveness and safety against a placebo in treating ALS. Participants will randomly receive either PTC857 or an inactive substance to compare outcomes between the two groups.
Participant Groups
2Treatment groups
Experimental Treatment
Active Control
Group I: PTC857Experimental Treatment1 Intervention
Participants will receive PTC857 during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who enter the Long-term Extension (LTE) Period, will receive open-label PTC857 for 28 weeks. Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.
Group II: PlaceboActive Control1 Intervention
Participants will receive matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who enter the LTE Period, will receive open-label PTC857 for 28 weeks. Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.

Find a Clinic Near You

Who Is Running the Clinical Trial?

PTC Therapeutics

Lead Sponsor

Trials
75
Recruited
6,300+

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

Findings from Research

The study evaluated design modifications for phase 3 clinical trials in ALS, using data from 988 participants across 5 trials and 5,100 from population-based cohorts, which could reduce sample size by 30.5% and placebo exposure time by 35.4%.
By implementing flexible trial designs and prognostic survival models, the proposed strategies aim to enhance patient selection and minimize exposure to ineffective treatments, potentially leading to more effective ALS therapies.
Innovating Clinical Trials for Amyotrophic Lateral Sclerosis: Challenging the Established Order.van Eijk, RPA., Nikolakopoulos, S., Roes, KCB., et al.[2021]

References

Innovating Clinical Trials for Amyotrophic Lateral Sclerosis: Challenging the Established Order. [2021]
Survival analyses from the CENTAUR trial in amyotrophic lateral sclerosis: Evaluating the impact of treatment crossover on outcomes. [2022]
Therapy development for ALS: lessons learned and path forward. [2015]
Pharmacologic reversal of cortical hyperexcitability in patients with ALS. [2019]
Improving efficiency of ALS clinical trials using lead-in designs. [2007]