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Quick Info
Trial Type
Treatment Type
Drug Trials
Not Available
Start Date
AL-S Pharma
Contact Information
Belgium, Other
Department of Neurology, University Hospitals, Leuven, 3000, Belgium
Canada, Alberta
ALS clinic at the Kaye Edmonton Clinic, University of Alberta, Edmonton, AB, AB T6G 1Z1, Canada
Contact: 780-248-1089
Canada, Ontario
London Health Sciences Centre - Victoria Hospital, London, ON, ON N6A 5W9, Canada
Contact: 519-663-3597
ALS Research Sunnybrook Health Sciences Centre, Toronto, ON, M4N 3M5, Canada
Canada, Quebec
Montreal Neurological Institute and Hospital / Dr Genge, Montréal, QC, H3A 2B4, Canada
Contact: 514-398-8551
Germany, Other
Charité, Berlin, 13353, Germany
Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE), Bonn, 53127, Germany
Hannover Medical School, Hanover, 30625, Germany
Ulm University Hospital, Ulm, 89081, Germany
Other, Other
Hanyang University Medical Center, Seoul, 04763, Other
Contact: +82-2-2290-8371
Sweden, Other
Studieenheten Akademiskt specialistcentrum, SLSO, Stockholm, 113 61, Sweden
Contact: +46851771231
Norrlands universitetssjukhus/ University Hospital of Northern Sweden (NUS), Umeå, SE- 901 85, Sweden
Contact: +46725487410
United States, California
UC San Diego, ACTRI, La Jolla, CA, 92037, United States
Contact: 858-243-1319
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
50% (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
≤24 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
Contact info and completion date updated
Updates to outcome measures
New site added
Protocol/Site updates
New sites recruiting
No significant updates
Sites added
Study start, protocol updates, sites added

Other Information

The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
Inclusion Criteria:
- All participants must adhere to contraception restrictions - Female participants of childbearing potential must adhere to contraception restrictions - Have possible, clinically probable, clinically probable-laboratory supported or definite familial or sporadic ALS in accordance with the El-Escorial criteria or who have a diagnosis of ALS as defined by the Gold Coast Criteria; progressive motor impairment documented by history or repeated clinical examination, preceded by normal motor development, and presence of upper and lower motor neuron dysfunction in at least 1 body region or lower motor neuron dysfunction in at least 2 body regions and investigations excluding other conditions - In familial ALS participants, a confirmed pathogenic superoxide dismutase 1 (SOD1) mutation - Onset of symptoms (i.e, weakness) within past 24 months prior to screening, at the time of obtaining informed consent - Have slow vital capacity (SVC) of greater than or equal to (> or =) 50 percentage (%) of predicted values. Participants with SVC of <50% of predicted values may be permitted to enter the open-label extension, based on the opinion of the investigator - Absence of bilevel positive airway pressure (BiPAP)/proportional assist ventilation (PAV) > 4 hours for symptoms attributable to ALS. Use of a CPAP for pre-existing conditions will be allowed - If on riluzole, must be on a stable dose.
- If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study - Able to provide informed consent which includes compliance with the requirements and restrictions - Have venous access sufficient to allow for blood sampling - Have clinical laboratory test results within the normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant by the investigator Exclusion Criteria:
- Have participated or currently participating in another clinical trial within 12 weeks of baseline (Day 1) - Have undergone a tracheostomy for ALS symptoms - Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day for the treatment of ALS related symptoms - Have other causes of neuromuscular weakness - Have cognitive impairment, severe disease in the cardiovascular, hematological, renal system, neurodegenerative disease, pulmonary disorder, or psychiatric illness - Pregnant or nursing women - Have been exposed to any antisense treatment targeting SOD1 within 6 months of the baseline visit - Have undergone stem cell therapy
  • AL-S Pharma
Trial Protocol as Published on
NCT05039099 (First Published: 9/1/2021)