Study to Assess the Effects of PTC857 Treatment in Participants with Amyotrophic Lateral Sclerosis ALS

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

PTC Identifier:
PTC857-CNS-001-ALS
ClinicalTrials.gov Identifier:
NCT05349721
EudraCT Identifier:
2021-006511-29
EU CT ID:
2023-510317-26-00
Terminated / Withdrawn

Trial Purpose

Age
18 - 80 Years
Sex
Both (Female and Male)
Healthy Volunteers
No

Key Participation Requirements

Inclusion Criteria:

  • ALS with preserved function, defined as:
  • a. Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit

Exclusion Criteria:

  • Females who are pregnant or nursing or plan to become pregnant during the study
  • Participants with clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular/ischemic disease or any other condition that, in the opinion of the investigator would jeopardize the safety of the participant or impact the validity of the study results
Inclusion and Exclusion Criteria
Inclusion criteria
  • ALS with preserved function, defined as:
  • a. Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit
  • b. Revised EL Escorial criteria of either:
  • (i) Clinically definite ALS
  • (ii) Clinically probable ALS
  • A total ALSFRS-R score of at least 34 at the start of the Screening Period
  • No significant respiratory compromise as evidenced by slow vital capacity ≥60% at the start of the Screening Period
  • All chronic concomitant medications (both prescription and over the counter), and non-pharmacologic therapy regimens, excluding standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, should be stable and unchanged from 14 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study
  • 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.
  • Standard-of-care therapy for the treatment of ALS (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol) should be stable and unchanged from 30 (-3) days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study.
Exclusion criteria
  • Females who are pregnant or nursing or plan to become pregnant during the study
  • Participants with clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular/ischemic disease or any other condition that, in the opinion of the investigator would jeopardize the safety of the participant or impact the validity of the study results
  • Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the investigator or the medical monitor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant
  • 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
  • Participant has previously received PTC857
  • Participant is receiving a combination of edaravone and sodium phenylbutyrate/taurursodiol treatment, where applicable, within 30 days prior to the start of the Screening Period
  • For female participants, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer

Trial Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
336

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
May 2022 - Sep 2024

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
Phase 2

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
Yes

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
N/A

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Interventional

Interventional

Participants in these studies are subject to an intervention by researchers. These trials are used to understand the effects of a particular intervention.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
N/A
Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
Drug

Allocation

This determines how study participants are assigned their treatment.

Allocation
Randomized

Randomized

This means participants are randomly assigned to the different arms of the study.

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
Treatment

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
Experimental

Experimental arm

Experimental arm: The group assigned to use the study treatment

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

Participant Ranks Based on the Combined Assessment of ALS Functional Rating Scale-Revised (ALSFRS-R) and Survival After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population)

Timeframe: Week 24

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Participant Ranks Based on the Combined Assessment of ALSFRS-R and Survival After 24 Weeks of Treatment (ITT 2 Analysis Population)

Timeframe: Week 24

Change from Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population)

Timeframe: Baseline, Week 24

Change from Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population)

Timeframe: Baseline, Week 24

Number of Participants with Treatment-emergent Adverse Events

Timeframe: Day 1 through Week 52

Change from Baseline in Slow Vital Capacity at Week 24

Timeframe: Baseline, Week 24

Change from Baseline in Sniff Nasal Inspiratory Pressure at Week 24

Timeframe: Baseline, Week 24

Change from Baseline in Modified Norris Scale Score at Week 24

Timeframe: Baseline, Week 24

Rate of Death

Timeframe: Baseline to Week 24

Length of Time to Death

Timeframe: Baseline to Week 24

Change from Baseline in ALS Assessment Questionnaire (ALSAQ-40) Score at Week 24

Timeframe: Baseline, Week 24

Change From Baseline in Neurofilament Light Chain (NfL) Activity at Week 24

Timeframe: Baseline, Week 24

Area under the Concentration-time Curve (AUC) of PTC857 in Plasma

Timeframe: Predose through Week 24

Maximum Observed Concentration (Cmax) of PTC857 in Plasma

Timeframe: Predose through Week 24

AUC of PTC857 in Cerebrospinal Fluid (CSF)

Timeframe: Predose through Week 24

Cmax of PTC857 in CSF

Timeframe: Predose through Week 24

Contact Information

Location
Status
Location
UC Irvine Health ALS and Neuromuscular Center
Orange, California, United States, 92868
Status
Recruiting
Location
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare
Tampa, Florida, United States, 33612
Status
Recruiting
Location
National Neuromuscular Research Institute
Austin, Texas, United States, 78759
Status
Recruiting
Location
University of Kansas Medical Center (KUMC) - Landon Center on Aging
Kansas City, Kansas, United States, 66205
Status
Not yet recruiting
Location
Forbes Norris MDA/ALS Research Center at California Pacific Medical Center
San Francisco, California, United States, 94109
Status
Recruiting
Location
Lewis Katz School of Medicine at Temple Universtiy
Philadelphia, Pennsylvania, United States, 19140
Status
Not yet recruiting
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