A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.

Study Status:

Not recruiting

Disease:

Amyotrophic Lateral Sclerosis , ALS

Study Type:

Interventional

Type of Intervention:

Drug, Biological

Intervention Name:

Placebo, Ravulizumab

Placebo:

Yes

Phase:

Phase 3

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

N/A

Clinicaltrials.gov ID:

NCT04248465

Neals Affiliated?

No

Coordinating Center Contact Information

United States

Study Sponsor:

Alexion Pharmaceuticals

Estimated Enrollment:

382

Estimated Study Start Date:

03 / 30 / 2020

Estimated Study Completion Date:

10 / 17 / 2021

Posting Last Modified Date:

11 / 30 / 2021

Date Study Added to neals.org:

01 / 30 / 2020

Minimum Age:

18 Years

Maximum Age:

N/A

Can participants use Riluzole?

Yes

Key Inclusion Criteria:

1. A diagnosis of sporadic or familial ALS, defined by the El Escorial criteria (possible, laboratory-supported probable, probable, or definite ALS).

2. ALS onset ≤ 36 months from Screening.

3. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.

4. Upright slow vital capacity ≥ 65% predicted at Screening.

5. If on riluzole, participant must be on a stable dose for 30 days; if on edaravone, participant must be on a stable dose for 60 days (2 cycles).

6. Body weight ≥ 40 kilograms at Screening.

7. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Key Exclusion Criteria:

1. History of Neisseria meningitidis infection.

2. Human immunodeficiency virus (HIV) infection (evidenced by HIV 1 or HIV 2 antibody titer).

3. Dependence on invasive or non-invasive mechanical ventilation.

4. Previously or currently treated with a complement inhibitor.

5. Exposure to an investigational drug or device within 30 days of Screening or 5 half lives of the study drug, whichever is greater.

Barrow Neurological Institute

Phoenix, Arizona 85013
United States

HonorHealth Research Institute

Scottsdale, Arizona 85251
United States

Neuromuscular Research Center and Clinic

Phoenix, Arizona 85028
United States

University of California San Diego Medical Center

San Diego, California 92093-0949
United States

University of California San Francisco Medical Center

San Francisco, California 94143
United States

Norris MDA/ALS Center

San Francisco, California 94115
United States

Stanford University Medical Center

Palo Alto, California 94304
United States

University of California-Irvine

Orange, California 92868
United States

University of Southern California

Los Angeles, California 90033
United States

Loma Linda University Medical Center

Loma Linda, California 92354
United States

University of Colorado Anschutz Medical Campus School of Medicine

Aurora, Colorado 80045
United States

University of Florida at Shands Jacksonville

Jacksonville, Florida 32209
United States

University of South Florida

Tampa, Florida 33612
United States

University of Chicago Medical Center

Chicago, Illinois 60637
United States

Indiana University Medical Center

Indianapolis, Indiana 46202
United States

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas 66160
United States

University of Kentucky

Lexington, Kentucky 40536
United States

Johns Hopkins University School Of Medicine

Baltimore, Maryland 21205
United States

Massachusetts General Hospital

Boston, Massachusetts 02114
United States

Las Vegas Clinic

Las Vegas, Nevada 89145
United States

Beth Israel Medical Center - PRIME

New York, New York 10003
United States

Hospital for Special Surgery

New York, New York 10021
United States

Atrium Health Neuroscience Institute

Charlotte, North Carolina 28207
United States

The Cleveland Clinic Foundation

Cleveland, Ohio 44195
United States

The Ohio State University

Columbus, Ohio 43221
United States

Allegheny Neurological Associates

Pittsburgh, Pennsylvania 15212
United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232
United States

Austin Neuromuscular Center

Austin, Texas 78756
United States

Houston Methodist Neurological Institute-Movement Disorders Clinic

Houston, Texas 77030
United States

Nerve & Muscle Center of Texas

Houston, Texas 77030
United States

University of Vermont Medical Center

Burlington, Vermont 05401
United States

Virginia Commonwealth University, Neurology Clinical and Translational Research Office

Richmond, Virginia 23298
United States

Sentara Neurology Specialists

Virginia Beach, Virginia 23456
United States

Swedish Neuroscience Institute

Seattle, Washington 98122-4470
United States

Brain and Mind Centre

Camperdown, New South Wales 2050
Australia

Royal Brisbane and Women's Hospital

Herston, Queensland 4029
Australia

Perron Institute for Neurological and Translational Science

Nedlands, Western Australia 6009
Australia

Westmead Hospital

Westmead, New South Wales 2145
Australia

UZ Leuven

Leuven 3000
Belgium

University Hospital of Quebec-Universite Laval

Québec, Quebec G1J 1Z4
Canada

Royal University Hospital

Saskatoon, Saskatchewan S7K 0M7
Canada

Montreal Neurological Institute and Hospital

Montreal, Quebec H3A 2B4
Canada

Toronto Sunnybrook Hospital

Toronto, Ontario M4N 3M5
Canada

LHSC - University Hospital

London, Ontario N6A 5A5
Canada

Stan Cassidy Center for Rehabilitation

Fredericton, New Brunswick E3B 0C7
Canada

Heritage Medical Research Centre (HMRC)

Edmonton, Alberta T6G2S2
Canada

Ålborg Universitets Hospital

Aalborg 9100
Denmark

Aarhus University Hospital Department of Neurology

Aarhus 8200
Denmark

Bispebjerg Hospital

Copenhagen 2400
Denmark

Hopital Roger Salengro - CHU Lille

Lille 59037
France

Groupe Hospitalier Pitie-Salpetriere

Paris cedex 13 75651
France

Hôpital de la Timone

Marseille 13385
France

CHU de Nice Hôpital Pasteur 2

Nice Cedex 1, Alpes Maritimes 06001
France

Hopital Neurologique Pierre Wertheimer

Bron cedex, Rhone 69677
France

CHU Tours - Hôpital Bretonneau

Tours Cedex 9, Indre Et Loire 37044
France

Hopital Gui de Chauliac

Montpellier, Herault 34295
France

CHU de Limoges - Hôpital Dupuytren

Limoges cedex, Haute Vienne 87042
France

Universitaetsklinikum Ulm

Ulm, Baden Wuerttemberg 89081
Germany

Klinikum rechts der Isar der TU Muenchen

Muenchen, Bayern 81675
Germany

Universitaetsmedizin Goettingen

Goettigen, Niedersachsen 37099
Germany

Medizinische Hochschule Hannover

Hannover, Niedersachsen 30625
Germany

Universitaetsklinikum Jena

Jena, Thueringen 07749
Germany

Beaumont Hospital

Dublin DUBLIN 9
Ireland

Tel Aviv Sourasky Medical Center

Tel Aviv 6423906
Israel

Hadassah University Hospital - Ein Kerem

Jerusalem 91120
Israel

Rambam Health Care Center

Haifa 91096
Israel

ICS Maugeri IRCCS

Milano 20138
Italy

Istituto Auxologico Italiano

Milano 20149
Italy

Azienda Ospedaliero-Universitaria di Modena - Ospedale Civile di Baggiovara

Modena 1355 - 41126
Italy

Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone

Palermo 90127
Italy

Azienda Ospedaliero Universitaria Pisana

Pisa 56126
Italy

University of Turin

Torino 10126
Italy

Ospedale San Raffaele

Milano 20132
Italy

Shiga University of Medical Science Hospital

Ōtsu, Shiga-Ken 520-2192
Japan

Keio University Hospital

Shinjuku-Ku, Tokyo-To 160-8582
Japan

Toho University Omori Medical Center

Ota-ku, Tokyo-To 143-8541
Japan

Tokushima University Hospital

Tokushima-shi, Tokushima-Ken 770-8503
Japan

Medical Hospital, Tokyo Medical and Dental University

Bunkyo-ku, Tokyo-To 113-8519
Japan

Niigata University Medical & Dental Hospital

Niigata-shi, Niigata-Ken 951-8520
Japan

Yoshino Neurology Clinic

Ichikawa-shi, Chiba-Ken 272-0827
Japan

Chiba University Hospital

Chiba-shi, Chiba-Ken 260-8677
Japan

Nagoya University Hospital

Nagoya-shi, Aichi-Ken 466-8560
Japan

Tohoku University Hospital

Sendai-shi, Miyagi-Ken 980-8574
Japan

University Medical Centre Utrecht

Utrecht 3508 GA
Netherlands

CityClinic

Warszawa 02-473
Poland

Hospital San Rafael

Madrid 28016
Spain

Hospital Universitari i Politecnic La Fe

Valencia 46026
Spain

Hospital de la Santa Creu i Sant Pau

Barcelona 08025
Spain

Hospital Universitari Vall d'Hebron

Barcelona 08035
Spain

Hospital Universitari de Bellvitge

L'Hospitalet De Llobregat, Barcelona 08907
Spain

Karolinska Trial Alliance (KTA)

Huddinge 141 86
Sweden

Norrlands universitetssjukhus

Umeå 90185
Sweden

Kantonsspital St. Gallen

Saint Gallen 9007
Switzerland

The National Hospital for Neurology & Neurosurgery

London, Greater London WC1N 3BG
United Kingdom

Royal Hallamshire Hospital

Sheffield, West Midlands S10 2JF
United Kingdom