PTC857 for Amyotrophic Lateral Sclerosis

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare, Tampa, FL
Amyotrophic Lateral Sclerosis+2 More
PTC857 - Drug
Eligibility
18+
All Sexes
What conditions do you have?
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Study Summary

This study will assess the effects and safety of PTC857 treatment in participants diagnosed with ALS.

Eligible Conditions

  • Amyotrophic Lateral Sclerosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Amyotrophic Lateral Sclerosis

Study Objectives

1 Primary · 14 Secondary · Reporting Duration: Day 1 through Week 52

Baseline, Week 24
Change from Baseline in ALS Assessment Questionnaire (ALSAQ-40) Score at Week 24
Change from Baseline in ALS Cognitive Behavioral Screen Assessment at Week 24
Change from Baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) Score at Week 24 (Modified Intention-to-Treat [mITT] Analysis Population)
Change from Baseline in ALSFRS-R Score at Week 24 (Intention-to-Treat [ITT] Analysis Population)
Change from Baseline in Modified Norris Scale Score at Week 24
Change from Baseline in Slow Vital Capacity at Week 24
Change from Baseline in Sniff Nasal Inspiratory Pressure at Week 24
Length of Time to Needing Respiratory Support/Intubation and/or Death
Rate of Needing Respiratory Support/Intubation and/or Death
Week 52
Number of Participants with Treatment-emergent Adverse Events
Predose through Week 24
AUC of PTC857 in Cerebrospinal Fluid (CSF)
Area under the Concentration-time Curve (AUC) of PTC857 in Plasma
Cmax of PTC857 in CSF
Maximum Observed Concentration (Cmax) of PTC857 in Plasma
Week 24
Combined Assessment of Function and Survival (CAFS) Score

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Amyotrophic Lateral Sclerosis

Trial Design

2 Treatment Groups

Placebo
1 of 2
PTC857
1 of 2
Active Control
Experimental Treatment

258 Total Participants · 2 Treatment Groups

Primary Treatment: PTC857 · Has Placebo Group · Phase 2

PTC857
Drug
Experimental Group · 1 Intervention: PTC857 · Intervention Types: Drug
Placebo
Drug
ActiveComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day 1 through week 52
Closest Location: University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare · Tampa, FL
Photo of Tampa  1Photo of Tampa  2Photo of Tampa  3
N/AFirst Recorded Clinical Trial
1 TrialsResearching Amyotrophic Lateral Sclerosis
0 CompletedClinical Trials

Eligibility Criteria

Age 18+ · All Participants · 8 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have a symptom onset of at least 7 and up to 18 months before the initial screening visit.
You have a total ALSFRS-R score of at least 34 at the start of the Screening Period.
You have no significant respiratory compromise as evidenced by slow vital capacity ≥60% or a respiratory rate ≥30/min.
Female participants must have a negative breast cancer imaging screening status (not considered clinically abnormal and/or requiring further evaluation/treatment) within 6 months prior to the Screening Visit, or during the Screening Period.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.