Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04248465
Recruitment Status : Recruiting
First Posted : January 30, 2020
Last Update Posted : November 9, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 354 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
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : October 2024


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




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 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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.

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


Contacts
Layout table for location contacts
Contact: Alexion Pharmaceuticals Inc. 855-752-2356 clinicaltrials@alexion.com

Locations
Show Show 61 study locations
Sponsors and Collaborators
Alexion Pharmaceuticals
Layout table for additonal information
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 9, 2020
Last Verified: November 2020

Layout table for additional information
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:
Layout table for MeSH terms
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
Ravulizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs