MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04579666
• Other study ID #: APL2-ALS-206
• Status: Terminated
• Phase: Phase 2
• 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
• 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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

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

External Links

•   Study Website