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)

Study Purpose:

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)

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

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

Clinicaltrials.gov ID (11 digit #):

NCT04579666

Neals Affiliated?

No

Coordinating Center Contact Information


Apellis Clinical Trial Information Line / .(JavaScript must be enabled to view this email address) / 617-977-5700
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Study Sponsor:

Apellis Pharmaceuticals, Inc.

Participant Duration:

Estimated Enrollment:

228

Estimated Study Start Date:

09/30/2020

Estimated Study Completion Date:

10/31/2023

Posting Last Modified Date:

10/13/2020

Date Study Added to alsconsortium.org:

10/13/2020
  • 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

    Austin Neuromuscular Center
    Austin, Texas 78756
    United States