Clinical trial NCT04579666: 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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Keywords: Amyotrophic Lateral Sclerosis
ALS
Motor Neuron Disease
APL-2
APL2
Pegcetacoplan
Lead sponsor: Apellis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2020-10-08
Enrollment: 249
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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)


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

Primary outcomes:
measure: RTP: Combined Assessment of Function and Survival (CAFS) Rank Score (Joint-Rank Score) at Week 52
timeFrame: Week 52
measure: RTP: Number of Subjects With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
timeFrame: From first dose of study drug (Day 1) up to 56 days post last dose of study drug, approximately 60 weeks
measure: OLP: Number of Participants With Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events
timeFrame: From first dose of study drug (Week 52) up to 56 days post last dose of study drug, approximately 60 weeks
measure: RTP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale (C-SSRS) up to Week 52
timeFrame: Baseline (Day 1) up to Week 52
measure: OLP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale up to Week 104
timeFrame: From Baseline (Week 52) up to Week 104
Secondary outcomes:
measure: RTP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score at Week 52
timeFrame: Baseline (Day 1) and Week 52
measure: RTP: Change From Baseline in Percent Predicted Slow Vital Capacity (%SVC) at Week 52
timeFrame: Baseline (Day 1) and Week 52
measure: RTP: Change From Baseline in Muscle Strength at Week 52
timeFrame: Baseline (Day 1) and Week 52
measure: RTP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 52
timeFrame: Baseline (Day 1) up to Week 52
measure: RTP: Change From Baseline in ALS Assessment Questionnaire (ALSAQ)-40 at Week 52
timeFrame: Baseline (Day 1) and Week 52
measure: OLP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Score at Week 104
timeFrame: Baseline (Week 52) and Week 104
measure: OLP: Change From Baseline in Percent Predicted Slow Vital Capacity at Week 104
timeFrame: Baseline (Week 52) and Week 104
measure: OLP: Change From Baseline in Muscle Strength at Week 104
timeFrame: Baseline (Week 52) and Week 104
measure: OLP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 104
timeFrame: Baseline (Week 52) and Week 104
measure: OLP: Change From Baseline in ALS Assessment Questionnaire-40 at Week 104
timeFrame: Baseline (Week 52) and Week 104
measure: Number of Subjects With an Event of Death During the Study
timeFrame: RTP: Baseline (Day 1) up to Week 52; OLP: Baseline (Week 52) up to Week 104
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Minneapolis
country: United States
facility: The Berman Center
state: Minnesota
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Camperdown
country: Australia
facility: Brain and Mind Centre
state: New South Wales
city: Erina
country: Australia
facility: Central Coast Neurosciences Research
state: New South Wales
city: Herston
country: Australia
facility: Royal Brisbane and Women's Hospital
state: Queensland
city: Southport
country: Australia
facility: Gold Coast University Hospital
state: Queensland
city: Heidelberg
country: Australia
facility: Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health
state: Victoria
city: Gent
country: Belgium
facility: AZ Sint-Lucas & Volkskliniek
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven (UZ Leuven)
city: Prague 2
country: Czechia
facility: Vseobecna fakultni nemocnice v Praze
city: Prague 6
country: Czechia
facility: FORBELI s.r.o.
city: Bordeaux
country: France
facility: Hopital Pellegrin
city: Bron
country: France
facility: Hôpital Neurologique Pierre Wertheimer
city: Clermont-Ferrand
country: France
facility: CHU Gabriel Montpied
city: Lille
country: France
facility: Hôpital Roger Salengro
city: Limoges
country: France
facility: CHU de Limoges Dupuytren 1
city: Nice
country: France
facility: CHU de Nice Hôpital Pasteur
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover Klinik für Neurologie
city: Jena
country: Germany
facility: Universitätsklinikum Jena
city: Rostock
country: Germany
facility: Universitätsmedizin Rostock, Klinik und Poliklinik für Neurologie
city: Ulm
country: Germany
facility: University of Ulm
city: Dublin
country: Ireland
facility: Beaumont Hospital
city: Milano
country: Italy
facility: Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus
city: Modena
country: Italy
facility: Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena
city: Palermo
country: Italy
facility: AOUP "P. Giaccone"
city: Torino
country: Italy
facility: Azienda Ospedaliera Universitaria di Torino - Città della Salute e della Scienza di Torino
city: Aichi
country: Japan
facility: National Hospital Organization Higashinagoya National Hospital
city: Fukuoka
country: Japan
facility: National Hospital Organization Omuta National Hospital
city: Hokkaido
country: Japan
facility: National Hospital Organization Asahikawa Medical Center
city: Hyōgo
country: Japan
facility: National Hospital Organization Hyogo-Chuo National Hospital
city: Ishikawa
country: Japan
facility: National Hospital Organization Iou National Hospital
city: Matsumoto
country: Japan
facility: National Hospital Organization Matsumoto Medical Center
city: Niigata
country: Japan
facility: Niigata National Hospital National Hospital Organization
city: Okinawa
country: Japan
facility: National Hospital Organization Okinawa National Hospital
city: Saitama
country: Japan
facility: National Hospital Organization Higashisaitama National Hospital
city: Shizuoka
country: Japan
facility: Shizuoka Institute of Epilepsy and Neurological Disorders
city: Tokyo
country: Japan
facility: Juntendo University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Utrecht
country: Netherlands
facility: University Medical Center Utrecht
city: Olsztyn
country: Poland
facility: Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii
city: Warsaw
country: Poland
facility: Centrum Medyczne NeuroProtect
city: Warsaw
country: Poland
facility: City Clinic Sp. z o.o.
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Barcelona
country: Spain
facility: Bellvitge University Hospital
city: Valencia
country: Spain
facility: Hospital Universitari I Politecnic La Fe
city: Kharkiv
country: Ukraine
facility: SI Institute of Neurology, Psychiatry and Narcology of NAMSU
city: Odessa
country: Ukraine
facility: Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University
city: Zaporizhzhya
country: Ukraine
facility: Zaporizhzhya Regional Clinical Hospital
city: Brighton
country: United Kingdom
facility: University Hospitals Sussex NHS Foundation Trust
city: London
country: United Kingdom
facility: Maurice Wohl Clinical Neuroscience Institute, King's College London
city: London
country: United Kingdom
facility: St George's University Hospitals NHS Foundation Trust
Study officials:
References: