A Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate Safety, Tolerability, Pharmacodynamic Markers, and Pharmacokinetics of AP-101 in Patients With Familial Amyotrophic Lateral Sclerosis (fALS) and Sporadic Amyotrophic L

Study 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.


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:



Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

AL-S Pharma SA / .(JavaScript must be enabled to view this email address) / 317-651-7036
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Study Sponsor:

AL-S Pharma

Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Min Vital Capacity (% predicted normal):


    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    Can participants use Riluzole?


    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

  • Site Contact Information

    UC San Diego
    John Ravits / .(JavaScript must be enabled to view this email address) / 858-243-1319
    La Jolla, California 92037
    United States

    Hospital for Special Surgery (HSS)
    Dale Lange / .(JavaScript must be enabled to view this email address) / 646-797-8917
    New York, New York 10021
    United States

    University of Alberta
    Wendy Johnston / .(JavaScript must be enabled to view this email address) / 780-248-1089
    Edmonton, Alberta

    London Health Sciences Centre
    Christen Shoesmith / .(JavaScript must be enabled to view this email address) / 519-663-3597
    London, Ontario

    Montreal Neurological Institute and Hospital
    Maxime Bérubé / .(JavaScript must be enabled to view this email address) / 514-398-3868
    Montréal, Quebec,

    Studieenheten Akademiskt specialistcentrum, SLSO
    Caroline Ingre / .(JavaScript must be enabled to view this email address) / +46725487410

    University Hospital of Northern Sweden
    .(JavaScript must be enabled to view this email address) / +46907852372