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Quick Info
Status
Currently Recruiting
Phase
2
Trial Type
Interventional
Treatment Type
Drug Trials
Randomization
Not Available
Enrollment
63
Start Date
9/2/2021
Sponsor
AL-S Pharma
Contact Information
    Contact information unknown.
Locations
Canada, Alberta
ALS clinic at the Kaye Edmonton Clinic, University of Alberta, Edmonton, AB, AB T6G 1Z1, Canada
Contact: 780-248-1089   wendyj@ualberta.ca
Canada, Ontario
London Health Sciences Centre - Victoria Hospital, London, ON, ON N6A 5W9, Canada
Contact: 519-663-3597   Christen.Shoesmith@lhsc.on.ca
Canada, Quebec
Montreal Neurological Institute and Hospital / Dr Genge, Montréal, QC, H3A 2B4, Canada
Contact: 514-398-3868   maximeberube@sympatico.ca
Sweden, Other
Studieenheten Akademiskt specialistcentrum, SLSO, Stockholm, 113 61, Sweden
Contact: +46725487410   caroline.ingre@sll.se
University Hospital of Northern Sweden (NUS), Umeå, SE- 901 85, Sweden
Contact: +46907852372   peter.andersen@umu.se
United States, California
UC San Diego, ACTRI, La Jolla, CA, 92037, United States
Contact: 858-243-1319   jravits@ucsd.edu
United States, New York
NYC - New York Medical College /Hospital for Special Surgery (HSS), New York, NY, 10021, United States
Contact: 646-797-8917   LangeD@HSS.EDU
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?
No
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
N/A
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Yes
Open Label
No
Update Notes
Study start, protocol updates, sites added
9/22/2021

Other Information

Purpose
The purpose of this Multiple Ascending Dose (MAD) study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
Eligibility
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 - Absence of bilevel positive airway pressure (BiPAP)/proportional assist ventilation (PAV) 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
Details
Collaborator(s)
  • AL-S Pharma
Trial Protocol as Published on Clinicaltrials.gov
NCT05039099 (First Published: 9/1/2021)