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

Primary Purpose

Amyotrophic Lateral Sclerosis, Motor Neuron Disease

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Pegcetacoplan (APL-2)
Placebo
Sponsored by
Apellis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring Amyotrophic Lateral Sclerosis, ALS, Motor Neuron Disease, APL-2, APL2, Pegcetacoplan

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Cedars-Sinai Medical Center
  • University of Colorado
  • University of South Florida
  • Augusta University
  • Indiana University
  • Johns Hopkins
  • The Berman Center
  • Hospital for Special Surgery
  • Austin Neuromuscular Center
  • University of Vermont Medical Center
  • Brain and Mind Centre
  • Central Coast Neurosciences Research
  • Royal Brisbane and Women's Hospital
  • Gold Coast University Hospital
  • Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health
  • AZ Sint-Lucas & Volkskliniek
  • Universitaire Ziekenhuizen Leuven (UZ Leuven)
  • Vseobecna fakultni nemocnice v Praze
  • FORBELI s.r.o.
  • Hopital Pellegrin
  • Hôpital Neurologique Pierre Wertheimer
  • CHU Gabriel Montpied
  • Hôpital Roger Salengro
  • CHU de Limoges Dupuytren 1
  • CHU de Nice Hôpital Pasteur
  • Charité - Universitätsmedizin Berlin
  • Medizinische Hochschule Hannover Klinik für Neurologie
  • Universitätsklinikum Jena
  • Universitätsmedizin Rostock, Klinik und Poliklinik für Neurologie
  • University of Ulm
  • Beaumont Hospital
  • Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus
  • Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena
  • AOUP "P. Giaccone"
  • Azienda Ospedaliera Universitaria di Torino - Città della Salute e della Scienza di Torino
  • National Hospital Organization Higashinagoya National Hospital
  • National Hospital Organization Omuta National Hospital
  • National Hospital Organization Asahikawa Medical Center
  • National Hospital Organization Hyogo-Chuo National Hospital
  • National Hospital Organization Iou National Hospital
  • National Hospital Organization Matsumoto Medical Center
  • Niigata National Hospital National Hospital Organization
  • National Hospital Organization Okinawa National Hospital
  • National Hospital Organization Higashisaitama National Hospital
  • Shizuoka Institute of Epilepsy and Neurological Disorders
  • Juntendo University Hospital
  • Tokyo Medical University Hospital
  • University Medical Center Utrecht
  • Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii
  • Centrum Medyczne NeuroProtect
  • City Clinic Sp. z o.o.
  • Hospital Universitari Vall d'Hebron
  • Bellvitge University Hospital
  • Hospital Universitari I Politecnic La Fe
  • SI Institute of Neurology, Psychiatry and Narcology of NAMSU
  • Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University
  • Zaporizhzhya Regional Clinical Hospital
  • University Hospitals Sussex NHS Foundation Trust
  • Maurice Wohl Clinical Neuroscience Institute, King's College London
  • St George's University Hospitals NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

1,080 mg pegcetacoplan (APL-2)

Placebo administered subcutaneously twice weekly

Arm Description

administered subcutaneously twice weekly

Outcomes

Primary Outcome Measures

Combined Assessment of Function and Survival (CAFS)
Efficacy

Secondary Outcome Measures

Incidence and severity of treatment-emergent adverse events (TEAEs)
Safety
Number of participants with positive responses (yes) to the Columbia Suicide Severity Rating Scale (C-SSRS)
Change from Baseline in the Revised ALS Functional Rating scale (ALSFRS-R) score
Minimum score of 0 and maximum of 48, with higher values representing better function
Change from Baseline in percentage of slow vital capacity (%SVC)
Change from Baseline in muscle strength
Time to death, permanent tracheostomy, or permanent assisted ventilation

Full Information

First Posted
September 22, 2020
Last Updated
May 27, 2022
Sponsor
Apellis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04579666
Brief Title
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 30, 2020 (Actual)
Primary Completion Date
March 2023 (Anticipated)
Study Completion Date
March 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Apellis Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Keywords
Amyotrophic Lateral Sclerosis, ALS, Motor Neuron Disease, APL-2, APL2, Pegcetacoplan

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
249 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1,080 mg pegcetacoplan (APL-2)
Arm Type
Experimental
Arm Description
administered subcutaneously twice weekly
Arm Title
Placebo administered subcutaneously twice weekly
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Pegcetacoplan (APL-2)
Intervention Description
Complement (C3) Inhibitor
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Sterile solution of equal volume to active arm
Primary Outcome Measure Information:
Title
Combined Assessment of Function and Survival (CAFS)
Description
Efficacy
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
Incidence and severity of treatment-emergent adverse events (TEAEs)
Description
Safety
Time Frame
Up to Week 104
Title
Number of participants with positive responses (yes) to the Columbia Suicide Severity Rating Scale (C-SSRS)
Time Frame
Up to Week 104
Title
Change from Baseline in the Revised ALS Functional Rating scale (ALSFRS-R) score
Description
Minimum score of 0 and maximum of 48, with higher values representing better function
Time Frame
Baseline, Week 52
Title
Change from Baseline in percentage of slow vital capacity (%SVC)
Time Frame
Baseline, at Week 52
Title
Change from Baseline in muscle strength
Time Frame
Baseline, Week 52
Title
Time to death, permanent tracheostomy, or permanent assisted ventilation
Time Frame
Up to Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Augusta University
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Johns Hopkins
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
The Berman Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55415
Country
United States
Facility Name
Hospital for Special Surgery
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Austin Neuromuscular Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78756
Country
United States
Facility Name
University of Vermont Medical Center
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States
Facility Name
Brain and Mind Centre
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Central Coast Neurosciences Research
City
Erina
State/Province
New South Wales
ZIP/Postal Code
2250
Country
Australia
Facility Name
Royal Brisbane and Women's Hospital
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Gold Coast University Hospital
City
Southport
State/Province
Queensland
ZIP/Postal Code
4215
Country
Australia
Facility Name
Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health
City
Heidelberg
State/Province
Victoria
ZIP/Postal Code
3084
Country
Australia
Facility Name
AZ Sint-Lucas & Volkskliniek
City
Gent
ZIP/Postal Code
B-9000
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven (UZ Leuven)
City
Leuven
ZIP/Postal Code
B-3000
Country
Belgium
Facility Name
Vseobecna fakultni nemocnice v Praze
City
Prague 2
ZIP/Postal Code
128 21
Country
Czechia
Facility Name
FORBELI s.r.o.
City
Prague 6
ZIP/Postal Code
160 00
Country
Czechia
Facility Name
Hopital Pellegrin
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Facility Name
Hôpital Neurologique Pierre Wertheimer
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
CHU Gabriel Montpied
City
Clermont-Ferrand
ZIP/Postal Code
63000
Country
France
Facility Name
Hôpital Roger Salengro
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
CHU de Limoges Dupuytren 1
City
Limoges
ZIP/Postal Code
87042
Country
France
Facility Name
CHU de Nice Hôpital Pasteur
City
Nice
ZIP/Postal Code
6300
Country
France
Facility Name
Charité - Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
D-13353
Country
Germany
Facility Name
Medizinische Hochschule Hannover Klinik für Neurologie
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Facility Name
Universitätsklinikum Jena
City
Jena
ZIP/Postal Code
07747
Country
Germany
Facility Name
Universitätsmedizin Rostock, Klinik und Poliklinik für Neurologie
City
Rostock
ZIP/Postal Code
18147
Country
Germany
Facility Name
University of Ulm
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Facility Name
Beaumont Hospital
City
Dublin
ZIP/Postal Code
DO9 V2NO
Country
Ireland
Facility Name
Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus
City
Milano
ZIP/Postal Code
20162
Country
Italy
Facility Name
Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena
City
Modena
ZIP/Postal Code
41126
Country
Italy
Facility Name
AOUP "P. Giaccone"
City
Palermo
ZIP/Postal Code
90129
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria di Torino - Città della Salute e della Scienza di Torino
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
National Hospital Organization Higashinagoya National Hospital
City
Aichi
ZIP/Postal Code
465-8620
Country
Japan
Facility Name
National Hospital Organization Omuta National Hospital
City
Fukuoka
ZIP/Postal Code
837-0911
Country
Japan
Facility Name
National Hospital Organization Asahikawa Medical Center
City
Hokkaido
ZIP/Postal Code
070-8644
Country
Japan
Facility Name
National Hospital Organization Hyogo-Chuo National Hospital
City
Hyōgo
ZIP/Postal Code
669-1592
Country
Japan
Facility Name
National Hospital Organization Iou National Hospital
City
Ishikawa
ZIP/Postal Code
920-0192
Country
Japan
Facility Name
National Hospital Organization Matsumoto Medical Center
City
Matsumoto
ZIP/Postal Code
399-8701
Country
Japan
Facility Name
Niigata National Hospital National Hospital Organization
City
Niigata
ZIP/Postal Code
945-8585
Country
Japan
Facility Name
National Hospital Organization Okinawa National Hospital
City
Okinawa
ZIP/Postal Code
901-2214
Country
Japan
Facility Name
National Hospital Organization Higashisaitama National Hospital
City
Saitama
ZIP/Postal Code
349-0196
Country
Japan
Facility Name
Shizuoka Institute of Epilepsy and Neurological Disorders
City
Shizuoka
ZIP/Postal Code
420-8688
Country
Japan
Facility Name
Juntendo University Hospital
City
Tokyo
ZIP/Postal Code
113-8431
Country
Japan
Facility Name
Tokyo Medical University Hospital
City
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
University Medical Center Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii
City
Olsztyn
ZIP/Postal Code
10-082
Country
Poland
Facility Name
Centrum Medyczne NeuroProtect
City
Warsaw
ZIP/Postal Code
01-684
Country
Poland
Facility Name
City Clinic Sp. z o.o.
City
Warsaw
ZIP/Postal Code
02-473
Country
Poland
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Bellvitge University Hospital
City
Barcelona
ZIP/Postal Code
08907
Country
Spain
Facility Name
Hospital Universitari I Politecnic La Fe
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
SI Institute of Neurology, Psychiatry and Narcology of NAMSU
City
Kharkiv
ZIP/Postal Code
61068
Country
Ukraine
Facility Name
Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University
City
Odessa
ZIP/Postal Code
65062
Country
Ukraine
Facility Name
Zaporizhzhya Regional Clinical Hospital
City
Zaporizhzhya
ZIP/Postal Code
69600
Country
Ukraine
Facility Name
University Hospitals Sussex NHS Foundation Trust
City
Brighton
ZIP/Postal Code
BN2 5BE
Country
United Kingdom
Facility Name
Maurice Wohl Clinical Neuroscience Institute, King's College London
City
London
ZIP/Postal Code
SE5 9RX
Country
United Kingdom
Facility Name
St George's University Hospitals NHS Foundation Trust
City
London
ZIP/Postal Code
SW17 0WT
Country
United Kingdom

12. IPD Sharing Statement

Links:
URL
http://MERIDIANTrial.com
Description
Study Website

Learn more about this trial

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

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