MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
- Conditions
- Amyotrophic Lateral SclerosisMotor Neuron Disease
- Interventions
- Drug: Pegcetacoplan (APL-2)Other: Placebo
- Registration Number
- NCT04579666
- Lead Sponsor
- 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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 249
- 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
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description 1,080 mg pegcetacoplan (APL-2) Pegcetacoplan (APL-2) administered subcutaneously twice weekly Placebo administered subcutaneously twice weekly Placebo -
- Primary Outcome Measures
Name Time Method Combined Assessment of Function and Survival (CAFS) Week 52 Efficacy
- Secondary Outcome Measures
Name Time Method Incidence and severity of treatment-emergent adverse events (TEAEs) Up to Week 104 Safety
Change from Baseline in percentage of slow vital capacity (%SVC) Baseline, at Week 52 Time to death, permanent tracheostomy, or permanent assisted ventilation Up to Week 52 Number of participants with positive responses (yes) to the Columbia Suicide Severity Rating Scale (C-SSRS) Up to Week 104 Change from Baseline in muscle strength Baseline, Week 52 Change from Baseline in the Revised ALS Functional Rating scale (ALSFRS-R) score Baseline, Week 52 Minimum score of 0 and maximum of 48, with higher values representing better function
Trial Locations
- Locations (60)
Brain and Mind Centre
🇦🇺Camperdown, New South Wales, Australia
Johns Hopkins
🇺🇸Baltimore, Maryland, United States
Hospital for Special Surgery
🇺🇸New York, New York, United States
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
The Berman Center
🇺🇸Minneapolis, Minnesota, United States
Augusta University
🇺🇸Augusta, Georgia, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Austin Neuromuscular Center
🇺🇸Austin, Texas, United States
University of Vermont Medical Center
🇺🇸Burlington, Vermont, United States
Central Coast Neurosciences Research
🇦🇺Erina, New South Wales, Australia
Royal Brisbane and Women's Hospital
🇦🇺Herston, Queensland, Australia
Gold Coast University Hospital
🇦🇺Southport, Queensland, Australia
Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health
🇦🇺Heidelberg, Victoria, Australia
AZ Sint-Lucas & Volkskliniek
🇧🇪Gent, Belgium
Universitaire Ziekenhuizen Leuven (UZ Leuven)
🇧🇪Leuven, Belgium
Hopital Pellegrin
🇫🇷Bordeaux, France
FORBELI s.r.o.
🇨🇿Prague 6, Czechia
CHU Gabriel Montpied
🇫🇷Clermont-Ferrand, France
Vseobecna fakultni nemocnice v Praze
🇨🇿Prague 2, Czechia
Hôpital Neurologique Pierre Wertheimer
🇫🇷Bron, France
Hôpital Roger Salengro
🇫🇷Lille, France
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany
CHU de Nice Hôpital Pasteur
🇫🇷Nice, France
Medizinische Hochschule Hannover Klinik für Neurologie
🇩🇪Hannover, Germany
Universitätsklinikum Jena
🇩🇪Jena, Germany
Universitätsmedizin Rostock, Klinik und Poliklinik für Neurologie
🇩🇪Rostock, Germany
University of Ulm
🇩🇪Ulm, Germany
Beaumont Hospital
🇮🇪Dublin, Ireland
Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus
🇮🇹Milano, Italy
Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena
🇮🇹Modena, Italy
AOUP "P. Giaccone"
🇮🇹Palermo, Italy
Azienda Ospedaliera Universitaria di Torino - Città della Salute e della Scienza di Torino
🇮🇹Torino, Italy
National Hospital Organization Higashinagoya National Hospital
🇯🇵Aichi, Japan
National Hospital Organization Omuta National Hospital
🇯🇵Fukuoka, Japan
National Hospital Organization Asahikawa Medical Center
🇯🇵Hokkaido, Japan
National Hospital Organization Iou National Hospital
🇯🇵Ishikawa, Japan
National Hospital Organization Matsumoto Medical Center
🇯🇵Matsumoto, Japan
National Hospital Organization Hyogo-Chuo National Hospital
🇯🇵Hyōgo, Japan
Niigata National Hospital National Hospital Organization
🇯🇵Niigata, Japan
National Hospital Organization Okinawa National Hospital
🇯🇵Okinawa, Japan
National Hospital Organization Higashisaitama National Hospital
🇯🇵Saitama, Japan
Shizuoka Institute of Epilepsy and Neurological Disorders
🇯🇵Shizuoka, Japan
Juntendo University Hospital
🇯🇵Tokyo, Japan
Tokyo Medical University Hospital
🇯🇵Tokyo, Japan
Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii
🇵🇱Olsztyn, Poland
Centrum Medyczne NeuroProtect
🇵🇱Warsaw, Poland
City Clinic Sp. z o.o.
🇵🇱Warsaw, Poland
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University
🇺🇦Odessa, Ukraine
Zaporizhzhya Regional Clinical Hospital
🇺🇦Zaporizhzhya, Ukraine
Bellvitge University Hospital
🇪🇸Barcelona, Spain
SI Institute of Neurology, Psychiatry and Narcology of NAMSU
🇺🇦Kharkiv, Ukraine
Hospital Universitari I Politecnic La Fe
🇪🇸Valencia, Spain
Maurice Wohl Clinical Neuroscience Institute, King's College London
🇬🇧London, United Kingdom
University Hospitals Sussex NHS Foundation Trust
🇬🇧Brighton, United Kingdom
CHU de Limoges Dupuytren 1
🇫🇷Limoges, France
St George's University Hospitals NHS Foundation Trust
🇬🇧London, United Kingdom
University of Colorado
🇺🇸Aurora, Colorado, United States
University of South Florida
🇺🇸Tampa, Florida, United States
University Medical Center Utrecht
🇳🇱Utrecht, Netherlands