This is a 24-month, Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)
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