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:

03/23/2021

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

    University of South Florida
    Tampa, Florida 33612
    United States

    Augusta University
    Augusta, GA 30912
    United States

    Indiana University
    Indianapolis, Indiana 46202
    United States

    Johns Hopkins
    Baltimore, Maryland 21205
    United States

    The Berman Center
    Minneapolis, Minnesota 55415
    United States

    Hospital for Special Surgery
    New York City, NY 10021
    United States

    Austin Neuromuscular Center
    Austin, Texas 78756
    United States

    University of Vermont Medical Center
    Burlington, Vermont 05401
    United States