Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (CARDINALS)
Amyotrophic Lateral Sclerosis
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis
Eligibility Criteria
Key Inclusion Criteria:
- Males or females aged between 18 and 70 years with a body mass index between 18 and 35 kilograms/meters squared
ALS with preserved function, defined as:
- Onset of the first symptom leading to the diagnosis of ALS ≥7 and ≤18 months at the time of the initial Screening Visit
- 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%
- All concomitant medications (both prescription and over the counter) 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, with the exception of prohibited medications
- 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
- Edaravone 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
Sites / Locations
- UC Irvine Health ALS and Neuromuscular CenterRecruiting
- Forbes Norris MDA/ALS Research Center at California Pacific Medical CenterRecruiting
- Holy Cross Hospital, Phil Smith Neuroscience Institute
- University of South Florida - Carol and Frank Morsani Center for Advanced HealthcareRecruiting
- Augusta UniversityRecruiting
- University of Kansas Medical Center (KUMC) - Landon Center on Aging
- Henry Ford Health System Department of Neurology
- Neurology Associates, P.C. / Somnos Clinical ResearchRecruiting
- University of Nebraska Medical CenterRecruiting
- Providence Brain and Spine Institute
- Lewis Katz School of Medicine at Temple Universtiy
- National Neuromuscular Research InstituteRecruiting
- Nerve and Muscle Center of TexasRecruiting
- Iadin Srl.Recruiting
- Royal Brisbane and Women's HospitalRecruiting
- Gold Coast HospitalRecruiting
- Calvary Health Care Bethlehem
- Austin HealthRecruiting
- AZ Sint-Lucas Gent
- UZ LeuvenRecruiting
- FORBELI s.r.o.Recruiting
- CHU de BordeauxRecruiting
- Hôpital Neurologique Pierre WertheimerRecruiting
- CHRU Lille - Hôpital Roger SalengroRecruiting
- CHU Dupuytren 1 LimogesRecruiting
- CRMR SLA - MNM du CHU de Nice
- Hannover Medical SchoolRecruiting
- Universitaetsklinikum Schleswig-Holstein (UKSH) Campus Luebeck, Klinik fuer Neurologie, PraezisionsneurologieRecruiting
- Centro Clinico Nemo Brescia
- Istituti Clinici Scientifici Maugeri IRCCSRecruiting
- Istituto Auxolgoico ItalianoRecruiting
- Maggiore della Carita University Hospital, Neurology department, ALS centerRecruiting
- ALS Clinical Research Center, University Hospital Policlinico "P Giaccone"
- Policlinico Umberto I
- AOU Citta Della Salute e ScienzaRecruiting
- UMC UtrechtRecruiting
- City Clinic Research Sp. Z o.oRecruiting
- Centrum Medyczne Neuro ProtectRecruiting
- Unidad Neuromuscular. Servicio de Neurologia Hospital Universitari Vall d'HebronRecruiting
- H. St Pau
- Hospital Universitario y Politecnico La FeRecruiting
- Skanes universitetssjukhus, VE Neurologi
Arms of the Study
Arm 1
Arm 2
Experimental
Active Comparator
PTC857
Placebo
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.
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.