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Quick Info
Trial Type
Treatment Type
Drug Trials
Start Date
Contact Information
Argentina, Other
Iadin Srl., Buenos Aires, C1015ABR, Argentina
Hospital Ramos Mejía, Ciudad Autonoma de Buenos Aires, CP 1221, Argentina
STAT Research S.A., Ciudad Autónoma de Buenos Aires, C1023AAB, Argentina
Australia, Other
Royal Brisbane and Women's Hospital, Brisbane, 4029, Australia
Calvary Health Care Bethlehem, Caulfield South, 3162, Australia
Austin Health, Heidelberg, 3084, Australia
Gold Coast Hospital, Southport, 4215, Australia
Belgium, Other
AZ Sint-Lucas Gent, Gent, 9000, Belgium
UZ Leuven, Leuven, 3000, Belgium
France, Other
CHU de Bordeaux, Bordeaux Cedex, 33076, France
Hôpital Neurologique Pierre Wertheimer, Bron Cedex, 69677, France
CHU Gabriel Montpied, Clermont-Ferrand, 63000, France
CHRU Lille - Hôpital Roger Salengro, Lille Cedex, 59037, France
CHU Dupuytren 1 Limoges, Limoges, 87000, France
CHU Gui de Chauliac (Pharmacie Saint-Eloi & Gui de Chauliac, Hopital Saint-Eloi), Montpellier, 34295, France
CRMR SLA - MNM du CHU de Nice, Nice, 06200, France
Germany, Other
Charite - Universitatsmedizin - Berlin, Berlin, 13353, Germany
Hannover Medical School, Hannover, 30625, Germany
University Hospital Jena, Jena, 7747, Germany
Universitaetsklinikum Schleswig-Holstein (UKSH) Campus Luebeck, Klinik fuer Neurologie, Praezisionsn, Lubeck, 23538, Germany
University of Ulm, Dept. of Neurology, Ulm, 89081, Germany
Italy, Other
Centro Clinico Nemo Brescia, Brescia, 25123, Italy
Istituti Clinici Scientifici Maugeri IRCCS, Milano, 20138, Italy
Istituto Auxolgoico Italiano, Milano, 20149, Italy
Azienda Ospedaliero Universitaria di Modena, Modena, 41126, Italy
Maggiore della Carita University Hospital, Neurology department, ALS center, Novara, 28100, Italy
ALS Clinical Research Center, University Hospital Policlinico "P Giaccone", Palermo, 90127, Italy
IRCCS Fondazione Mondino - Reparto Neuroncologia/Neuroinfiammazione, Pavia, 27100, Italy
Policlinico Umberto I, Roma, 00161, Italy
AOU Citta Della Salute e Scienza, Torino, 10126, Italy
Japan, Other
National Hospital Organization Matsumoto Medical Center, Neurology department, Matsumoto, 399-8701, Japan
Netherlands, Other
UMC Utrecht, Utrecht, 3584 CW, Netherlands
Other, Other
University hospital Brno, Department of Neurology, Brno, 62500, Other
FORBELI s.r.o., Prague 6, 160 00, Other
Poland, Other
City Clinic Research Sp. Z o.o, Warsaw, 02-473, Poland
Centrum Medyczne Neuro Protect, Warszawa, 01-684, Poland
Spain, Other
Hospital Universitario Vall d'Hebron ALS Unit., Barcelona, 08035, Spain
Unidad Neuromuscular. Servicio de Neurologia Hospital Universitari Vall d'Hebron, Barcelona, 08035, Spain
H. St Pau, Barcelona, 08041, Spain
Hospital Universitario y Politecnico La Fe, Valencia, 46026, Spain
Sweden, Other
Skanes universitetssjukhus, VE Neurologi, Malmo, SE-211 24, Sweden
Norrlands universitetssjukhus Neurologens Forskningsavdelning, Umea, 90185, Sweden
United States, California
UC Irvine Health ALS and Neuromuscular Center, Orange, CA, 92868, United States
Forbes Norris MDA/ALS Research Center at California Pacific Medical Center, San Francisco, CA, 94109, United States
United States, Florida
Phil Smith Neuroscience Institute, Holy Cross Hospital, Fort Lauderdale, FL, 33308, United States
Holy Cross Hospital, Phil Smith Neuroscience Institute, Fort Lauderdale, FL, 33308, United States
Intercoastal Medical Group, Inc., Sarasota, FL, 34239, United States
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare, Tampa, FL, 33612, United States
United States, Georgia
Augusta University, Augusta, GA, 30912, United States
United States, Kansas
University of Kansas Medical Center (KUMC) - Landon Center on Aging, Kansas City, KS, 66205, United States
United States, Michigan
Henry Ford Health System Department of Neurology, Detroit, MI, 48202, United States
United States, Nebraska
Neurology Associates, P.C. / Somnos Clinical Research, Lincoln, NE, 68506, United States
University of Nebraska Medical Center, Omaha, NE, 68198-8440, United States
United States, Oregon
Providence Brain and Spine Institute, Portland, OR, 97213, United States
United States, Pennsylvania
Lewis Katz School of Medicine at Temple Universtiy, Philadelphia, PA, 19140, United States
United States, Texas
National Neuromuscular Research Institute, Austin, TX, 78759, United States
Nerve and Muscle Center of Texas, Houston, TX, 77030, United States
United States, Wisconsin
Medical College of Wisconsin, Milwaukee, WI, 53226, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥60% (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
≤18 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Open Label
Update Notes
No significant updates
No significant updates
Site updates
No significant updates
No significant updates
No significant updates
No significant updates
Protocol updates, new sites recruiting
Inclusion criteria updates
Study recruiting

Other Information

This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.
Key Inclusion Criteria:
- 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 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.
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
Participants will be randomized to 1 of 2 treatment groups: PTC857 or matching placebo. 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.
Trial Protocol as Published on
NCT05349721 (First Published: 4/21/2022)