Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT ID: NCT05349721
Last Updated: 2025-08-01
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE2
336 participants
INTERVENTIONAL
2022-05-15
2025-01-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT03160898
Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation
NCT02794857
Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
NCT03127267
A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT07082192
Cistanche Total Glycosides for Amyotrophic Lateral Sclerosis: A Randomized Control Trial (RCT) Study Assessing Clinical Response
NCT00753571
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
PTC857
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.
PTC857
PTC8657 will be administered as an oral solution twice a day.
Placebo
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.
Placebo
Matching placebo will be administered as an oral solution twice a day.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
PTC857
PTC8657 will be administered as an oral solution twice a day.
Placebo
Matching placebo will be administered as an oral solution twice a day.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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 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
* 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
18 Years
80 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
PTC Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UC Irvine Health ALS and Neuromuscular Center
Orange, California, United States
Forbes Norris MDA/ALS Research Center at California Pacific Medical Center
San Francisco, California, United States
Holy Cross Hospital, Phil Smith Neuroscience Institute
Fort Lauderdale, Florida, United States
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare
Tampa, Florida, United States
Augusta University
Augusta, Georgia, United States
University of Kansas Medical Center (KUMC) - Landon Center on Aging
Kansas City, Kansas, United States
Henry Ford Health System Department of Neurology
Detroit, Michigan, United States
Neurology Associates, P.C. / Somnos Clinical Research
Lincoln, Nebraska, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Providence Brain and Spine Institute
Portland, Oregon, United States
Lewis Katz School of Medicine at Temple Universtiy
Philadelphia, Pennsylvania, United States
National Neuromuscular Research Institute
Austin, Texas, United States
Nerve and Muscle Center of Texas
Houston, Texas, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
STAT Research S.A.
Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina
Iadin Srl.
Buenos Aires, , Argentina
Hospital Ramos Mejía
Buenos Aires, , Argentina
Royal Brisbane and Women's Hospital
Brisbane, Queensland, Australia
Gold Coast Hospital
Southport, Queensland, Australia
Calvary Health Care Bethlehem
Caulfield South, Victoria, Australia
Austin Health
Heidelberg, Victoria, Australia
AZ Sint-Lucas Gent
Ghent, , Belgium
UZ Leuven
Leuven, , Belgium
University hospital Brno, Department of Neurology
Brno, , Czechia
FORBELI s.r.o.
Prague, , Czechia
CHU de Bordeaux
Bordeaux, , France
Hôpital Neurologique Pierre Wertheimer
Bron, , France
CHU Gabriel Montpied
Clermont-Ferrand, , France
CHRU Lille - Hôpital Roger Salengro
Lille, , France
CHU Dupuytren 1 Limoges
Limoges, , France
CHU Gui de Chauliac (Pharmacie Saint-Eloi & Gui de Chauliac, Hopital Saint-Eloi)
Montpellier, , France
CRMR SLA - MNM du CHU de Nice
Nice, , France
Charite - Universitatsmedizin - Berlin
Berlin, , Germany
Hannover Medical School
Hanover, , Germany
University Hospital Jena
Jena, , Germany
Universitaetsklinikum Schleswig-Holstein (UKSH) Campus Luebeck, Klinik fuer Neurologie, Praezisionsneurologie
Lübeck, , Germany
University of Ulm, Dept. of Neurology
Ulm, , Germany
Centro Clinico Nemo Brescia
Brescia, , Italy
Istituti Clinici Scientifici Maugeri IRCCS
Milan, , Italy
Istituto Auxolgoico Italiano
Milan, , Italy
Azienda Ospedaliero Universitaria di Modena
Modena, , Italy
Maggiore della Carita University Hospital, Neurology department, ALS center
Novara, , Italy
ALS Clinical Research Center, University Hospital Policlinico "P Giaccone"
Palermo, , Italy
IRCCS Fondazione Mondino - Reparto Neuroncologia/Neuroinfiammazione
Pavia, , Italy
Policlinico Umberto I
Roma, , Italy
AOU Citta Della Salute e Scienza
Torino, , Italy
PTC Clinical Site
Japanese City, , Japan
UMC Utrecht
Utrecht, , Netherlands
Centrum Medyczne Neuro Protect
Warsaw, , Poland
City Clinic Research Sp. Z o.o
Warsaw, , Poland
Unidad Neuromuscular. Servicio de Neurologia Hospital Universitari Vall d'Hebron
Barcelona, , Spain
H. St Pau
Barcelona, , Spain
Hospital Universitario y Politecnico La Fe
Valencia, , Spain
Skanes universitetssjukhus, VE Neurologi
Malmo, , Sweden
Norrlands universitetssjukhus Neurologens Forskningsavdelning
Umeå, , Sweden
Countries
Review the countries where the study has at least one active or historical site.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2021-006511-29
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-510317-26-00
Identifier Type: OTHER
Identifier Source: secondary_id
PTC857-CNS-001-ALS
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.