A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)

Study Purpose:

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

Study Status:



Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

PTC857, Placebo




Phase 2

Study Chair(s)/Principal Investigator(s):


Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Patient Advocacy / email hidden; JavaScript is required / 1-866-562-4620

Full Study Summary:

Participants will be randomized to 1 of 2 treatment groups: PTC857 or matching placebo. Following successful completion of the 24-week Treatment Period, participants have the option to receive open-label PTC857 in the Long-term Extension Period for 28 weeks.

Study Sponsor:

PTC Therapeutics

Estimated Enrollment:


Estimated Study Start Date:

05 / 15 / 2022

Estimated Study Completion Date:

02 / 28 / 2025

Posting Last Modified Date:

05 / 17 / 2023

Date Study Added to neals.org:

04 / 27 / 2022

Minimum Age:

18 Years

Maximum Age:

80 Years

Key Inclusion Criteria:

- Males or females aged between 18 and 80 years with a body mass index between 18 and 35 kilograms/meters squared

- ALS with preserved function, defined as:

1. Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit

2. 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 concomitant medications (both prescription and over the counter), including standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, and non-pharmacologic therapy regimens should be stable and unchanged from 30 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.

Key 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

UC Irvine Health ALS and Neuromuscular Center | Recruiting

Orange, California 92868
United States

Forbes Norris MDA/ALS Research Center at California Pacific Medical Center | Recruiting

San Francisco, California 94109
United States

Phil Smith Neuroscience Institute, Holy Cross Hospital | Recruiting

Fort Lauderdale, Florida 33308
United States

Intercoastal Medical Group, Inc. | Recruiting

Sarasota, Florida 34239
United States

University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare | Recruiting

Tampa, Florida 33612
United States

Augusta University | Recruiting

Augusta, Georgia 30912
United States

University of Kansas Medical Center (KUMC) - Landon Center on Aging | Not yet recruiting

Kansas City, Kansas 66205
United States

Henry Ford Health System Department of Neurology | Not yet recruiting

Detroit, Michigan 48202
United States

Neurology Associates, P.C. / Somnos Clinical Research | Recruiting

Lincoln, Nebraska 68506
United States

University of Nebraska Medical Center | Recruiting

Omaha, Nebraska 68198-8440
United States

Lewis Katz School of Medicine at Temple Universtiy | Not yet recruiting

Philadelphia, Pennsylvania 19140
United States

National Neuromuscular Research Institute | Recruiting

Austin, Texas 78759
United States

Nerve and Muscle Center of Texas | Not recruiting

Houston, Texas 77030
United States

Royal Brisbane and Women's Hospital | Recruiting

Brisbane, Queensland 4029

Austin Health | Recruiting

Heidelberg, Victoria 3084

CHU de Bordeaux | Recruiting

Bordeaux Cedex 33076

Hôpital Neurologique Pierre Wertheimer | Recruiting

Bron Cedex 69677

CHRU Lille - Hôpital Roger Salengro | Recruiting

Lille Cedex 59037

CHU Dupuytren 1 Limoges | Recruiting

Limoges 87000

Centrum Medyczne Neuro Protect | Recruiting

Warszawa 01-684

Hospital Universitario Vall d'Hebron ALS Unit. | Recruiting

Barcelona 08035

Hospital Universitario y Politecnico La Fe | Recruiting

Valencia 46026