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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT04579666
Other study ID # APL2-ALS-206
Secondary ID
Status Terminated
Phase Phase 2
First received
Last updated
Start date September 30, 2020
Est. completion date July 13, 2023

Study information

Verified date February 2024
Source Apellis Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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)


Recruitment information / eligibility

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

Study Design


Intervention

Drug:
Pegcetacoplan (APL-2)
Complement (C3) Inhibitor
Other:
Placebo
Sterile solution of equal volume to active arm

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Apellis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Czechia,  France,  Germany,  Ireland,  Italy,  Japan,  Netherlands,  Poland,  Spain,  Ukraine,  United Kingdom, 

Outcome

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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