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MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)

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ClinicalTrials.gov Identifier: NCT04579666
Recruitment Status : Recruiting
First Posted : October 8, 2020
Last Update Posted : May 24, 2021
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Brief Summary:
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)

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Motor Neuron Disease Drug: Pegcetacoplan (APL-2) Other: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 228 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : September 30, 2020
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : October 2023


Arm Intervention/treatment
Experimental: 1,080 mg pegcetacoplan (APL-2)
administered subcutaneously twice weekly
Drug: Pegcetacoplan (APL-2)
Complement (C3) Inhibitor

Placebo Comparator: Placebo administered subcutaneously twice weekly Other: Placebo
Sterile solution of equal volume to active arm




Primary Outcome Measures :
  1. Combined Assessment of Function and Survival (CAFS) [ Time Frame: Week 52 ]
    Efficacy


Secondary Outcome Measures :
  1. Incidence and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Up to Week 104 ]
    Safety

  2. Number of participants with positive responses (yes) to the Columbia Suicide Severity Rating Scale (C-SSRS) [ Time Frame: Up to Week 104 ]
  3. Change from Baseline in the Revised ALS Functional Rating scale (ALSFRS-R) score [ Time Frame: Baseline, Week 52 ]
    Minimum score of 0 and maximum of 48, with higher values representing better function

  4. Change from Baseline in percentage of slow vital capacity (%SVC) [ Time Frame: Baseline, Week 52 ]
  5. Change from Baseline in muscle strength [ Time Frame: Baseline, Week 52 ]
  6. Time to death, permanent tracheostomy, or permanent assisted ventilation [ Time Frame: Up to Week 52 ]


Information from the National Library of Medicine

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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

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

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): NCT04579666


Contacts
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Contact: Apellis Clinical Trial Information Line 617-977-5700 clinicaltrials@apellis.com

Locations
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United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
United States, Indiana
Indiana University Recruiting
Indianapolis, Indiana, United States, 46202
United States, Maryland
Johns Hopkins Recruiting
Baltimore, Maryland, United States, 21205
United States, Minnesota
The Berman Center Recruiting
Minneapolis, Minnesota, United States, 55415
United States, Texas
Austin Neuromuscular Center Recruiting
Austin, Texas, United States, 78756
United States, Vermont
University of Vermont Medical Center Recruiting
Burlington, Vermont, United States, 05401
Poland
Centrum Medyczne NeuroProtect Recruiting
Warsaw, Poland, 01-684
City Clinic Sp. z o.o. Recruiting
Warsaw, Poland, 02-473
Spain
Bellvitge University Hospital Recruiting
Barcelona, Spain, 08907
Hospital Universitari I Politecnic La Fe Recruiting
Valencia, Spain, 46026
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.
Additional Information:
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Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04579666    
Other Study ID Numbers: APL2-ALS-206
First Posted: October 8, 2020    Key Record Dates
Last Update Posted: May 24, 2021
Last Verified: May 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 Apellis Pharmaceuticals, Inc.:
Amyotrophic Lateral Sclerosis
ALS
Motor Neuron Disease
APL-2
APL2
Pegcetacoplan
Additional relevant MeSH terms:
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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