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An Efficacy and Safety Study of Ravulizumab in ALS Participants

Sponsor
Alexion Pharmaceuticals (Industry)
Overall Status
Terminated
CT.gov ID
NCT04248465
Collaborator
(none)
382
Enrollment
95
Locations
2
Arms
18.6
Actual Duration (Months)
4
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • Drug: Placebo
  • Biological: Ravulizumab
 Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
382 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
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)
Actual Study Start Date :
Mar 30, 2020
Actual Primary Completion Date :
Oct 17, 2021
Actual Study Completion Date :
Oct 17, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: Ravulizumab

Participants will receive ravulizumab for the duration of the study.

Biological: Ravulizumab
Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
Other Names:
  • ALXN1210
  • Ultomiris

    		•
    Placebo Comparator: Placebo

    Participants will receive placebo during the 50-week Randomized Controlled Period of the study, after which they will enter the Open-label Extension Period of the study and switch to receive ravulizumab.

    Drug: Placebo
    Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.

    Biological: Ravulizumab
    Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
    Other Names:
  • ALXN1210
  • Ultomiris

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score [Baseline, Week 50]

    Secondary Outcome Measures

    1. Time To Ventilator Assistance-free Survival [Up to Week 50]

    2. Change From Baseline In Slow Vital Capacity [Baseline, Week 50]

    3. Incidence Of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, And TEAEs Leading To Study Drug Discontinuation [Baseline through Week 156]

    4. Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry [Baseline, Week 50]

    5. Change From Baseline In Serum Neurofilament Light Chain [Baseline, Week 50]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Barrow Neurological Institute Phoenix Arizona United States 85013
    2 Neuromuscular Research Center and Clinic Phoenix Arizona United States 85028
    3 HonorHealth Research Institute Scottsdale Arizona United States 85251
    4 Loma Linda University Medical Center Loma Linda California United States 92354
    5 University of Southern California Los Angeles California United States 90033
    6 University of California-Irvine Orange California United States 92868
    7 Stanford University Medical Center Palo Alto California United States 94304
    8 University of California San Diego Medical Center San Diego California United States 92093-0949
    9 Norris MDA/ALS Center San Francisco California United States 94115
    10 University of California San Francisco Medical Center San Francisco California United States 94143
    11 University of Colorado Anschutz Medical Campus School of Medicine Aurora Colorado United States 80045
    12 University of Florida at Shands Jacksonville Jacksonville Florida United States 32209
    13 University of South Florida Tampa Florida United States 33612
    14 University of Chicago Medical Center Chicago Illinois United States 60637
    15 Indiana University Medical Center Indianapolis Indiana United States 46202
    16 University of Kansas Medical Center Research Institute, Inc. Kansas City Kansas United States 66160
    17 University of Kentucky Lexington Kentucky United States 40536
    18 Johns Hopkins University School Of Medicine Baltimore Maryland United States 21205
    19 Massachusetts General Hospital Boston Massachusetts United States 02114
    20 Las Vegas Clinic Las Vegas Nevada United States 89145
    21 Beth Israel Medical Center - PRIME New York New York United States 10003
    22 Hospital for Special Surgery New York New York United States 10021
    23 Atrium Health Neuroscience Institute Charlotte North Carolina United States 28207
    24 The Cleveland Clinic Foundation Cleveland Ohio United States 44195
    25 The Ohio State University Columbus Ohio United States 43221
    26 Allegheny Neurological Associates Pittsburgh Pennsylvania United States 15212
    27 Vanderbilt University Medical Center Nashville Tennessee United States 37232
    28 Austin Neuromuscular Center Austin Texas United States 78756
    29 Houston Methodist Neurological Institute-Movement Disorders Clinic Houston Texas United States 77030
    30 Nerve & Muscle Center of Texas Houston Texas United States 77030
    31 University of Vermont Medical Center Burlington Vermont United States 05401
    32 Virginia Commonwealth University, Neurology Clinical and Translational Research Office Richmond Virginia United States 23298
    33 Sentara Neurology Specialists Virginia Beach Virginia United States 23456
    34 Swedish Neuroscience Institute Seattle Washington United States 98122-4470
    35 Brain and Mind Centre Camperdown New South Wales Australia 2050
    36 Westmead Hospital Westmead New South Wales Australia 2145
    37 Royal Brisbane and Women's Hospital Herston Queensland Australia 4029
    38 Perron Institute for Neurological and Translational Science Nedlands Western Australia Australia 6009
    39 UZ Leuven Leuven Belgium 3000
    40 Heritage Medical Research Centre (HMRC) Edmonton Alberta Canada T6G2S2
    41 Stan Cassidy Center for Rehabilitation Fredericton New Brunswick Canada E3B 0C7
    42 LHSC - University Hospital London Ontario Canada N6A 5A5
    43 Toronto Sunnybrook Hospital Toronto Ontario Canada M4N 3M5
    44 Montreal Neurological Institute and Hospital Montreal Quebec Canada H3A 2B4
    45 University Hospital of Quebec-Universite Laval Québec Quebec Canada G1J 1Z4
    46 Royal University Hospital Saskatoon Saskatchewan Canada S7K 0M7
    47 Ålborg Universitets Hospital Aalborg Denmark 9100
    48 Aarhus University Hospital Department of Neurology Aarhus Denmark 8200
    49 Bispebjerg Hospital Copenhagen Denmark 2400
    50 CHU de Nice Hôpital Pasteur 2 Nice Cedex 1 Alpes Maritimes France 06001
    51 CHU de Limoges - Hôpital Dupuytren Limoges cedex Haute Vienne France 87042
    52 Hopital Gui de Chauliac Montpellier Herault France 34295
    53 CHU Tours - Hôpital Bretonneau Tours Cedex 9 Indre Et Loire France 37044
    54 Hopital Neurologique Pierre Wertheimer Bron cedex Rhone France 69677
    55 Hopital Roger Salengro - CHU Lille Lille France 59037
    56 Hôpital de la Timone Marseille France 13385
    57 Groupe Hospitalier Pitie-Salpetriere Paris cedex 13 France 75651
    58 Universitaetsklinikum Ulm Ulm Baden Wuerttemberg Germany 89081
    59 Klinikum rechts der Isar der TU Muenchen Muenchen Bayern Germany 81675
    60 Universitaetsmedizin Goettingen Goettigen Niedersachsen Germany 37099
    61 Medizinische Hochschule Hannover Hannover Niedersachsen Germany 30625
    62 Universitaetsklinikum Jena Jena Thueringen Germany 07749
    63 Beaumont Hospital Dublin Ireland DUBLIN 9
    64 Rambam Health Care Center Haifa Israel 91096
    65 Hadassah University Hospital - Ein Kerem Jerusalem Israel 91120
    66 Tel Aviv Sourasky Medical Center Tel Aviv Israel 6423906
    67 Ospedale San Raffaele Milano Italy 20132
    68 ICS Maugeri IRCCS Milano Italy 20138
    69 Istituto Auxologico Italiano Milano Italy 20149
    70 Azienda Ospedaliero-Universitaria di Modena - Ospedale Civile di Baggiovara Modena Italy 1355 - 41126
    71 Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone Palermo Italy 90127
    72 Azienda Ospedaliero Universitaria Pisana Pisa Italy 56126
    73 University of Turin Torino Italy 10126
    74 Nagoya University Hospital Nagoya-shi Aichi-Ken Japan 466-8560
    75 Chiba University Hospital Chiba-shi Chiba-Ken Japan 260-8677
    76 Yoshino Neurology Clinic Ichikawa-shi Chiba-Ken Japan 272-0827
    77 Tohoku University Hospital Sendai-shi Miyagi-Ken Japan 980-8574
    78 Niigata University Medical & Dental Hospital Niigata-shi Niigata-Ken Japan 951-8520
    79 Shiga University of Medical Science Hospital Ōtsu Shiga-Ken Japan 520-2192
    80 Tokushima University Hospital Tokushima-shi Tokushima-Ken Japan 770-8503
    81 Medical Hospital, Tokyo Medical and Dental University Bunkyo-ku Tokyo-To Japan 113-8519
    82 Toho University Omori Medical Center Ota-ku Tokyo-To Japan 143-8541
    83 Keio University Hospital Shinjuku-Ku Tokyo-To Japan 160-8582
    84 University Medical Centre Utrecht Utrecht Netherlands 3508 GA
    85 CityClinic Warszawa Poland 02-473
    86 Hospital Universitari de Bellvitge L'Hospitalet De Llobregat Barcelona Spain 08907
    87 Hospital de la Santa Creu i Sant Pau Barcelona Spain 08025
    88 Hospital Universitari Vall d'Hebron Barcelona Spain 08035
    89 Hospital San Rafael Madrid Spain 28016
    90 Hospital Universitari i Politecnic La Fe Valencia Spain 46026
    91 Karolinska Trial Alliance (KTA) Huddinge Sweden 141 86
    92 Norrlands universitetssjukhus Umeå Sweden 90185
    93 Kantonsspital St. Gallen Saint Gallen Switzerland 9007
    94 The National Hospital for Neurology & Neurosurgery London Greater London United Kingdom WC1N 3BG
    95 Royal Hallamshire Hospital Sheffield West Midlands United Kingdom S10 2JF

    Sponsors and Collaborators

    • Alexion Pharmaceuticals

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Alexion Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT04248465
    Other Study ID Numbers:
    • ALXN1210-ALS-308
    • 2019-004619-30
    First Posted:
    Jan 30, 2020
    Last Update Posted:
    Nov 30, 2021
    Last Verified:
    Nov 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Alexion Pharmaceuticals
    Ravulizumab
    ALXN1210
    Ultomiris
    Motor Neuron Disease
    Amyotrophic Lateral Sclerosis
    ALS
    Additional relevant MeSH terms:
    Motor Neuron Disease
    Amyotrophic Lateral Sclerosis
    Sclerosis
    Ravulizumab

    Study Results

    No Results Posted as of Nov 30, 2021