A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Full Study Summary:

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  18

  Maximum Age:

  N/A

  Min Vital Capacity (% predicted normal):

  60

  Time since Symptom Onset:

  <18 months

  Time since Diagnosis:

  Can participants use Riluzole?

  Yes

  
  

  Inclusion Criteria:

  • At least 18 years of age
  • Sporadic ALS diagnosed as definite, probable, or laboratory-supported probable as defined by the revised El Escorial criteria
  • Slow vital capacity (SVC) ≥60% of the predicted value at screening
  • Onset of ALS symptoms within 72 weeks (18 months) prior to screening
  • Total ALSFRS-R score of ≥30 at screening
  • Have vaccination within 5 years against Streptococcus pneumoniae, Neisseria meningitidis (types A, C, W, Y, and B), and Haemophilus influenzae (type B) or agree to receive vaccination

  Exclusion Criteria:

  • Confirmed or suspected other causes of neuromuscular weakness
  • Diagnosed with another neurodegenerative disease (examples include Parkinson's disease and Huntington's disease)
  • Significant pulmonary disorder not attributed to ALS (eg, chronic obstructive pulmonary disease, pulmonary fibrosis, cystic fibrosis, pulmonary arterial hypertension)
  • If taking riluzole, participant must be on a stable dose for 30 days prior to the start of the screening period. Use of riluzole is not required for participation.
  • If taking edaravone, participant must be on a stable dose for 60 days prior to the start of the screening period. Use of edaravone is not required for participation.
  • Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation
  • Use of any other complement inhibitor within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation

• Site Contact Information

  University of South Florida
  Tampa, Florida 33612
  United States

  Indiana University
  Indianapolis, Indiana 46202
  United States

  Johns Hopkins
  Baltimore, Maryland 21205
  United States

  The Berman Center
  Minneapolis, Minnesota 55415
  United States

  Austin Neuromuscular Center
  Austin, Texas 78756
  United States