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An Efficacy and Safety Study of Ravulizumab in ALS Participants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04248465
Recruitment Status : Terminated (The IDMC recommended the study be discontinued due to lack of efficacy with ravulizumab.)
First Posted : January 30, 2020
Last Update Posted : November 30, 2021
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Description
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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
Amyotrophic Lateral Sclerosis ALS Drug: Placebo Biological: Ravulizumab Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
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 : March 30, 2020
Actual Primary Completion Date : October 17, 2021
Actual Study Completion Date : October 17, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Ravulizumab

Arms and Interventions
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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
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Primary Outcome Measures :
  1. Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score [ Time Frame: Baseline, Week 50 ]

Secondary Outcome Measures :
  1. Time To Ventilator Assistance-free Survival [ Time Frame: Up to Week 50 ]
  2. Change From Baseline In Slow Vital Capacity [ Time Frame: Baseline, Week 50 ]
  3. Incidence Of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, And TEAEs Leading To Study Drug Discontinuation [ Time Frame: Baseline through Week 156 ]
  4. Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry [ Time Frame: Baseline, Week 50 ]
  5. Change From Baseline In Serum Neurofilament Light Chain [ Time Frame: Baseline, Week 50 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04248465


Locations
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Sponsors and Collaborators
Alexion Pharmaceuticals
More Information
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04248465    
Other Study ID Numbers: ALXN1210-ALS-308
2019-004619-30 ( EudraCT Number )
First Posted: January 30, 2020    Key Record Dates
Last Update Posted: November 30, 2021
Last Verified: November 2021

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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:
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Ravulizumab
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
 Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs