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:

11/01/2021

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

    Cedars-Sinai Medical Center
    Los Angeles, California 90048
    United States

    University of Colorado
    Aurora, Colorado 80045
    United States

    University of South Florida
    Tampa, Florida 33612
    United States

    Augusta University
    Augusta, Georgia 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, New York 10021
    United States

    Austin Neuromuscular Center
    Austin, Texas 78756
    United States

    University of Vermont Medical Center
    Burlington, Vermont 05401
    United States

    Brain and Mind Centre
    Camperdown, New South Wales
    Australia

    Central Coast Neurosciences Research
    Erina, New South Wales
    Australia

    Royal Brisbane and Women's Hospital
    Herston, Queensland
    Australia

    Gold Coast University Hospital
    Southport, Queensland
    Australia

    Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS)
    Heidelberg, Victoria
    Australia

    AZ Sint-Lucas & Volkskliniek
    Gent,
    Belgium

    Universitaire Ziekenhuizen Leuven (UZ Leuven)
    Leuven,
    Belgium

    Vseobecna fakultni nemocnice v Praze
    Prague 2,
    Czechia

    FORBELI s.r.o.
    Prague 6,
    Czechia

    Hopital Pellegrin
    Bordeaux,
    France

    Hôpital Neurologique Pierre Wertheimer
    Bron,
    France

    CHU Gabriel Montpied
    Clermont-Ferrand,
    France

    Hôpital Roger Salengro
    Lille,
    France

    CHU de Limoges Dupuytren 1
    Limoges,
    France

    CHU de Nice Hôpital Pasteur
    Nice,
    France

    Charité - Universitätsmedizin Berlin
    Berlin,
    Germany

    Medizinische Hochschule Hannover Klinik für Neurologie
    Hannover,
    Germany

    Universitätsklinikum Jena
    Jena,
    Germany

    Universitätsmedizin Rostock, Klinik und Poliklinik für Neurologie
    Rostock,
    Germany

    University of Ulm
    Ulm,
    Germany

    Beaumont Hospital
    Dublin,
    Ireland

    Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus
    Milano,
    Italy

    Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena
    Modena,
    Italy

    AOUP "P. Giaccone"
    Palermo,
    Italy

    Azienda Ospedaliera Universitaria di Torino - Città della Salute e della Scienza di Torino
    Torino,
    Italy

    National Hospital Organization Higashinagoya National Hospital
    Aichi,
    Japan

    National Hospital Organization Omuta National Hospital
    Fukuoka,
    Japan

    National Hospital Organization Asahikawa Medical Center
    Hokkaido,
    Japan

    National Hospital Organization Hyogo-Chuo National Hospital
    Hyōgo,
    Japan

    National Hospital Organization Iou National Hospital
    Ishikawa,
    Japan

    National Hospital Organization Matsumoto Medical Center
    Matsumoto,
    Japan

    Niigata National Hospital National Hospital Organization
    Niigata,
    Japan

    National Hospital Organization Okinawa National Hospital
    Okinawa,
    Japan

    National Hospital Organization Higashisaitama National Hospital
    Saitama,
    Japan

    Shizuoka Institute of Epilepsy and Neurological Disorders
    Shizuoka,
    Japan

    Juntendo University Hospital
    Tokyo,
    Japan

    Tokyo Medical University Hospital
    Tokyo,
    Japan

    University Medical Center Utrecht
    Utrecht,
    Netherlands

    Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii
    Olsztyn,
    Poland

    Centrum Medyczne NeuroProtect
    Warsaw,
    Poland

    City Clinic Sp. z o.o.
    Warsaw,
    Poland

    Bellvitge University Hospital
    Barcelona,
    Spain

    Hospital Universitari Vall d'Hebron
    Barcelona,
    Spain

    Hospital Universitari I Politecnic La Fe
    Valencia,
    Spain

    SI Institute of Neurology
    Kharkiv,
    Ukraine

    Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University
    Odessa,
    Ukraine

    Zaporizhzhya Regional Clinical Hospital
    Zaporizhzhya,
    Ukraine

    University Hospitals Sussex NHS Foundation Trust
    Brighton,
    United Kingdom

    Maurice Wohl Clinical Neuroscience Institute, King's College London
    London,
    United Kingdom

    St George's University Hospitals NHS Foundation Trust
    London,
    United Kingdom