for people ages 18 years and up (full criteria)
at La Jolla, California and other locations
study started
completion around
Principal Investigator
by John Ravits
Headshot of John Ravits
John Ravits



The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

Official Title

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 Lateral Sclerosis (sALS)


Amyotrophic Lateral Sclerosis, Familial Amyotrophic Lateral Sclerosis, Sporadic Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Sclerosis


You can join if…

Open to people ages 18 years and up

  • 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

You CAN'T join if...

  • 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


  • UC San Diego, ACTRI accepting new patients
    La Jolla California 92037 United States
  • ALS clinic at the Kaye Edmonton Clinic, University of Alberta accepting new patients
    Edmonton Alberta AB T6G 1Z1 Canada

Lead Scientist at UCSD

  • John Ravits
    Professor Of Clinical, Neurosciences, Vc-health Sciences-schools. Authored (or co-authored) 114 research publications. Research interests: ALS neuroanatomic propagation · ALS molecular neuropathology · ALS genomics and transcriptome · C9orf72 ALS · Antisense oligonucleotide therapies · ALS clinical trials


accepting new patients
Start Date
Completion Date
AL-S Pharma
Phase 2 Amyotropic Lateral Sclerosis (ALS) Research Study
Study Type
Expecting 63 study participants
Last Updated