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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04579666




Registration number
NCT04579666
Ethics application status
Date submitted
22/09/2020
Date registered
8/10/2020
Date last updated
14/02/2024

Titles & IDs
Public title
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
Scientific 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)
Secondary ID [1] 0 0
APL2-ALS-206
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Motor Neuron Disease 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan (APL-2)
Other interventions - Placebo

Experimental: 1,080 mg pegcetacoplan (APL-2) - administered subcutaneously twice weekly

Placebo comparator: Placebo administered subcutaneously twice weekly -


Treatment: Drugs: Pegcetacoplan (APL-2)
Complement (C3) Inhibitor

Other interventions: Placebo
Sterile solution of equal volume to active arm
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Combined Assessment of Function and Survival (CAFS)
Timepoint [1] 0 0
Week 52
Secondary outcome [1] 0 0
Incidence and severity of treatment-emergent adverse events (TEAEs)
Timepoint [1] 0 0
Up to Week 104
Secondary outcome [2] 0 0
Number of participants with positive responses (yes) to the Columbia Suicide Severity Rating Scale (C-SSRS)
Timepoint [2] 0 0
Up to Week 104
Secondary outcome [3] 0 0
Change from Baseline in the Revised ALS Functional Rating scale (ALSFRS-R) score
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Change from Baseline in percentage of slow vital capacity (%SVC)
Timepoint [4] 0 0
Baseline, at Week 52
Secondary outcome [5] 0 0
Change from Baseline in muscle strength
Timepoint [5] 0 0
Baseline, Week 52
Secondary outcome [6] 0 0
Time to death, permanent tracheostomy, or permanent assisted ventilation
Timepoint [6] 0 0
Up to Week 52

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/07/2023
Sample size
Target
Accrual to date
Final
249
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Brain and Mind Centre - Camperdown
Recruitment hospital [2] 0 0
Central Coast Neurosciences Research - Erina
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [4] 0 0
Gold Coast University Hospital - Southport
Recruitment hospital [5] 0 0
Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health - Heidelberg
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2250 - Erina
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
4215 - Southport
Recruitment postcode(s) [5] 0 0
3084 - Heidelberg
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Vermont
Country [11] 0 0
Belgium
State/province [11] 0 0
Gent
Country [12] 0 0
Belgium
State/province [12] 0 0
Leuven
Country [13] 0 0
Czechia
State/province [13] 0 0
Prague 2
Country [14] 0 0
Czechia
State/province [14] 0 0
Prague 6
Country [15] 0 0
France
State/province [15] 0 0
Bordeaux
Country [16] 0 0
France
State/province [16] 0 0
Bron
Country [17] 0 0
France
State/province [17] 0 0
Clermont-Ferrand
Country [18] 0 0
France
State/province [18] 0 0
Lille
Country [19] 0 0
France
State/province [19] 0 0
Limoges
Country [20] 0 0
France
State/province [20] 0 0
Nice
Country [21] 0 0
Germany
State/province [21] 0 0
Berlin
Country [22] 0 0
Germany
State/province [22] 0 0
Hannover
Country [23] 0 0
Germany
State/province [23] 0 0
Jena
Country [24] 0 0
Germany
State/province [24] 0 0
Rostock
Country [25] 0 0
Germany
State/province [25] 0 0
Ulm
Country [26] 0 0
Ireland
State/province [26] 0 0
Dublin
Country [27] 0 0
Italy
State/province [27] 0 0
Milano
Country [28] 0 0
Italy
State/province [28] 0 0
Modena
Country [29] 0 0
Italy
State/province [29] 0 0
Palermo
Country [30] 0 0
Italy
State/province [30] 0 0
Torino
Country [31] 0 0
Japan
State/province [31] 0 0
Aichi
Country [32] 0 0
Japan
State/province [32] 0 0
Fukuoka
Country [33] 0 0
Japan
State/province [33] 0 0
Hokkaido
Country [34] 0 0
Japan
State/province [34] 0 0
Hyogo
Country [35] 0 0
Japan
State/province [35] 0 0
Ishikawa
Country [36] 0 0
Japan
State/province [36] 0 0
Matsumoto
Country [37] 0 0
Japan
State/province [37] 0 0
Niigata
Country [38] 0 0
Japan
State/province [38] 0 0
Okinawa
Country [39] 0 0
Japan
State/province [39] 0 0
Saitama
Country [40] 0 0
Japan
State/province [40] 0 0
Shizuoka
Country [41] 0 0
Japan
State/province [41] 0 0
Tokyo
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Netherlands
State/province [42] 0 0
Utrecht
Country [43] 0 0
Poland
State/province [43] 0 0
Olsztyn
Country [44] 0 0
Poland
State/province [44] 0 0
Warsaw
Country [45] 0 0
Spain
State/province [45] 0 0
Barcelona
Country [46] 0 0
Spain
State/province [46] 0 0
Valencia
Country [47] 0 0
Ukraine
State/province [47] 0 0
Kharkiv
Country [48] 0 0
Ukraine
State/province [48] 0 0
Odessa
Country [49] 0 0
Ukraine
State/province [49] 0 0
Zaporizhzhya
Country [50] 0 0
United Kingdom
State/province [50] 0 0
Brighton
Country [51] 0 0
United Kingdom
State/province [51] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Apellis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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)
Trial website
https://clinicaltrials.gov/study/NCT04579666
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04579666