Amyotrophic Lateral Sclerosis Clinical Trial
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)
Verified date | February 2024 |
Source | Apellis Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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)
Status | Terminated |
Enrollment | 249 |
Est. completion date | July 13, 2023 |
Est. primary completion date | March 31, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
Australia | Brain and Mind Centre | Camperdown | New South Wales |
Australia | Central Coast Neurosciences Research | Erina | New South Wales |
Australia | Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health | Heidelberg | Victoria |
Australia | Royal Brisbane and Women's Hospital | Herston | Queensland |
Australia | Gold Coast University Hospital | Southport | Queensland |
Belgium | AZ Sint-Lucas & Volkskliniek | Gent | |
Belgium | Universitaire Ziekenhuizen Leuven (UZ Leuven) | Leuven | |
Czechia | Vseobecna fakultni nemocnice v Praze | Prague 2 | |
Czechia | FORBELI s.r.o. | Prague 6 | |
France | Hopital Pellegrin | Bordeaux | |
France | Hôpital Neurologique Pierre Wertheimer | Bron | |
France | CHU Gabriel Montpied | Clermont-Ferrand | |
France | Hôpital Roger Salengro | Lille | |
France | CHU de Limoges Dupuytren 1 | Limoges | |
France | CHU de Nice Hôpital Pasteur | Nice | |
Germany | Charité - Universitätsmedizin Berlin | Berlin | |
Germany | 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 | |
Ireland | Beaumont Hospital | Dublin | |
Italy | 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 | |
Japan | 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 Hyogo-Chuo National Hospital | Hyogo | |
Japan | National Hospital Organization Iou National Hospital | Ishikawa | |
Japan | National Hospital Organization Matsumoto Medical Center | Matsumoto | |
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 | |
Netherlands | University Medical Center Utrecht | Utrecht | |
Poland | Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii | Olsztyn | |
Poland | Centrum Medyczne NeuroProtect | Warsaw | |
Poland | City Clinic Sp. z o.o. | Warsaw | |
Spain | Bellvitge University Hospital | Barcelona | |
Spain | Hospital Universitari Vall d'Hebron | Barcelona | |
Spain | Hospital Universitari I Politecnic La Fe | Valencia | |
Ukraine | SI Institute of Neurology, Psychiatry and Narcology of NAMSU | Kharkiv | |
Ukraine | Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University | Odessa | |
Ukraine | Zaporizhzhya Regional Clinical Hospital | Zaporizhzhya | |
United Kingdom | University Hospitals Sussex NHS Foundation Trust | Brighton | |
United Kingdom | Maurice Wohl Clinical Neuroscience Institute, King's College London | London | |
United Kingdom | St George's University Hospitals NHS Foundation Trust | London | |
United States | Augusta University | Augusta | Georgia |
United States | University of Colorado | Aurora | Colorado |
United States | Austin Neuromuscular Center | Austin | Texas |
United States | Johns Hopkins | Baltimore | Maryland |
United States | University of Vermont Medical Center | Burlington | Vermont |
United States | Indiana University | Indianapolis | Indiana |
United States | Cedars-Sinai Medical Center | Los Angeles | California |
United States | The Berman Center | Minneapolis | Minnesota |
United States | Hospital for Special Surgery | New York | New York |
United States | University of South Florida | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
Apellis Pharmaceuticals, Inc. |
United States, Australia, Belgium, Czechia, France, Germany, Ireland, Italy, Japan, Netherlands, Poland, Spain, Ukraine, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Combined Assessment of Function and Survival (CAFS) | Efficacy | Week 52 | |
Secondary | Incidence and severity of treatment-emergent adverse events (TEAEs) | Safety | Up to Week 104 | |
Secondary | Number of participants with positive responses (yes) to the Columbia Suicide Severity Rating Scale (C-SSRS) | Up to Week 104 | ||
Secondary | 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 | Baseline, Week 52 | |
Secondary | Change from Baseline in percentage of slow vital capacity (%SVC) | Baseline, at Week 52 | ||
Secondary | Change from Baseline in muscle strength | Baseline, Week 52 | ||
Secondary | Time to death, permanent tracheostomy, or permanent assisted ventilation | Up to Week 52 |
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