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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04579666
Registration number
NCT04579666
Ethics application status
Date submitted
22/09/2020
Date registered
8/10/2020
Date last updated
24/04/2025
Titles & IDs
Public title
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
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Scientific 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)
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Secondary ID [1]
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APL2-ALS-206
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis
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Motor Neuron Disease
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Condition category
Condition code
Neurological
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Neurodegenerative diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan (APL-2)
Other interventions - Placebo
Experimental: 1,080 mg pegcetacoplan (APL-2) - administered subcutaneously twice weekly
Placebo comparator: Placebo administered subcutaneously twice weekly -
Treatment: Drugs: Pegcetacoplan (APL-2)
Complement (C3) Inhibitor
Other interventions: Placebo
Sterile solution of equal volume to active arm
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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RTP: Combined Assessment of Function and Survival (CAFS) Rank Score (Joint-Rank Score) at Week 52
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Assessment method [1]
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The CAFS scale is a combined endpoint ranking subjects' clinical outcomes based on ALS Functional Rating Scale-Revised (ALSFRS-R-described below) and survival time. For ALSFRS-R, 12 functions were rated on 5-point ordinal rating scales (0 to 4) with a total score range of 0-48 (sum of all 12 items); higher score indicated better functioning. For survival time, longer the subject survives indicated better outcome. Each subject's outcome was compared to every other subject outcome in trial in series of pairwise comparisons, summed scores (sum of comparisons \[+1 {better}, 0 {tie}, -1 {worse}\]) were ranked and ranged from 001-247 (number of subjects in modified \[m\]ITT population).Reported values are the least squares mean rank scores in each group for the composite endpoint. Higher rank indicated better outcome.
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Timepoint [1]
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Week 52
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Primary outcome [2]
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RTP: Number of Subjects With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
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Assessment method [2]
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An AE was any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An SAE was any AE or suspected adverse reaction that, in the view of the investigator, resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or was a congenital anomaly or birth defect. TEAEs were AEs that started on or after first dose of study drug or started before first dose of study drug but increased in severity on or after the first dose of study drug up to 56 days post last dose of study drug.
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Timepoint [2]
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From first dose of study drug (Day 1) up to 56 days post last dose of study drug, approximately 60 weeks
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Primary outcome [3]
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OLP: Number of Participants With Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events
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Assessment method [3]
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An AE was any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An SAE was any AE or suspected adverse reaction that, in the view of the investigator, resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or was a congenital anomaly or birth defect. TEAEs were AEs that started on or after first dose of study drug or started before first dose of study drug but increased in severity on or after the first dose of study drug up to 56 days post last dose of study drug.
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Timepoint [3]
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From first dose of study drug (Week 52) up to 56 days post last dose of study drug, approximately 60 weeks
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Primary outcome [4]
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RTP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale (C-SSRS) up to Week 52
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Assessment method [4]
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The C-SSRS is a measure used to identify and assess individuals at risk for suicide and included "yes" or "no" responses for assessment of suicidal ideation (SI) and suicidal behavior (SB).C-SSRS SI items are classified on 5-item scale:1 (wish to be dead),2 (non-specific active suicidal thoughts),3 (active SI with any methods without intent to act),4 (active SI with some intent to act, without specific plan) and 5 (active SI with a specific plan and intent).C-SSRS SB items are classified on 5-item scale: 1 (preparatory acts or behavior), 2 (aborted attempt), 3 (interrupted attempt), 4 (actual attempt \[non-fatal\]) and 5 (completed suicide). Numeric ratings were provided for SI (1 to 5) and SB (1 to 5) with 5 being more severe for each. Number of subjects with a response of 'yes' to SI only, SB only \& SI and SB are reported. Baseline: last available, non-missing observation prior to first study drug administration.
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Timepoint [4]
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Baseline (Day 1) up to Week 52
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Primary outcome [5]
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OLP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale up to Week 104
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Assessment method [5]
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The C-SSRS is a measure used to identify and assess individuals at risk for suicide and included "yes" or "no" responses for assessment of SI and SB.C-SSRS SI items are classified on 5-item scale:1 (wish to be dead),2 (non-specific active suicidal thoughts),3 (active SI with any methods without intent to act),4 (active SI with some intent to act, without specific plan) and 5 (active SI with a specific plan and intent).C-SSRS SB items are classified on 5-item scale:1 (preparatory acts or behavior),2 (aborted attempt), 3 (interrupted attempt), 4 (actual attempt \[non-fatal\]) and 5 (completed suicide). Numeric ratings were provided for SI (1 to 5) and SB (1 to 5) with 5 being more severe for each. Number of subjects with a response of 'yes' to SI only, SB only \& SI and SB are reported. Baseline: last available, non-missing observation prior to first study drug administration in OLP.
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Timepoint [5]
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From Baseline (Week 52) up to Week 104
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Secondary outcome [1]
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RTP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score at Week 52
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Assessment method [1]
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The ALSFRS-R included 12 items for assessment of functional status: speech, salivation, swallowing, handwriting, cutting food and handling utensils, dressing and hygiene, turning in bed and adjusting bed clothes, walking, climbing stairs, dyspnea, orthopnea, and respiratory insufficiency. Each item ranged from 0 (no ability) to 4 (normal ability). Individual item scores were summed to produce a total score between 0 (worst) and 48 (best) with higher scores meaning better outcome. Least squares mean is presented here. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [1]
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Baseline (Day 1) and Week 52
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Secondary outcome [2]
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RTP: Change From Baseline in Percent Predicted Slow Vital Capacity (%SVC) at Week 52
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Assessment method [2]
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SVC is a pulmonary function test and predictor of functional loss in ALS. It was conducted at clinic visits with the clinic spirometer which reflected the maximum amount of air that could be exhaled slowly. %SVC is the actual volume exhaled in the first 1 second, divided by the normal value for that actual value for a person of that age, gender, height and weight. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [2]
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Baseline (Day 1) and Week 52
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Secondary outcome [3]
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RTP: Change From Baseline in Muscle Strength at Week 52
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Assessment method [3]
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Muscle strength was measured using handheld dynamometry (HHD) and assessed the following muscles: first dorsal interosseous, wrist extension, elbow extension, elbow flexion, shoulder flexion, knee extension, knee flexion, and ankle dorsiflexion, on both sides of the body. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [3]
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Baseline (Day 1) and Week 52
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Secondary outcome [4]
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RTP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 52
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Assessment method [4]
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Subjects with an event (that is, either death or permanent tracheostomy or permanent assisted ventilation) in RTP are reported.
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Timepoint [4]
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Baseline (Day 1) up to Week 52
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Secondary outcome [5]
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RTP: Change From Baseline in ALS Assessment Questionnaire (ALSAQ)-40 at Week 52
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Assessment method [5]
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The ALSAQ-40 was a 40-item validated questionnaire designed to assess health related quality of life (QoL) over the previous 2 weeks in subjects with ALS. It represented 5 dimensions of health status; each scored from 0 (never, or best) to 4 (always, or worst). 5 dimensions evaluated were: physical mobility (10 items: 1-10; possible score of 0-40); activities of daily living/independence (10 items: 11-20; possible score of 0-40); eating and drinking (3 items: 21-23; possible score of 0-12); communication (7 items: 24-30; possible score: 0-28) and emotional functioning (10 items: 31-40; possible score: 0-40). The total score 0 (no impairment) to 160 (severe impairment) was calculated by adding the 5 dimension scores; least squares mean is presented here. Higher scores indicated worse QoL.
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Timepoint [5]
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Baseline (Day 1) and Week 52
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Secondary outcome [6]
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OLP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Score at Week 104
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Assessment method [6]
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The ALSFRS-R included 12 items for assessment of functional status: speech, salivation, swallowing, handwriting, cutting food and handling utensils, dressing and hygiene, turning in bed and adjusting bed clothes, walking, climbing stairs, dyspnea, orthopnea, and respiratory insufficiency. Each item ranged from 0 (no ability) to 4 (normal ability). Individual item scores were summed to produce a total score between 0 (worst) and 48 (best) with higher scores meaning better outcome. Mean is presented here. Baseline was defined as the last observed value for the efficacy assessment prior to taking the first dose of study drug in OLP.
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Timepoint [6]
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Baseline (Week 52) and Week 104
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Secondary outcome [7]
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OLP: Change From Baseline in Percent Predicted Slow Vital Capacity at Week 104
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Assessment method [7]
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SVC is a pulmonary function test and predictor of functional loss in ALS. It was planned to be conducted at clinic visits with the clinic spirometer which reflected the maximum amount of air that could be exhaled slowly. %SVC is the actual volume exhaled in the first 1 second, divided by the normal value for that actual value for a person of that age, gender, height and weight. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [7]
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Baseline (Week 52) and Week 104
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Secondary outcome [8]
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OLP: Change From Baseline in Muscle Strength at Week 104
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Assessment method [8]
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Muscle strength was planned to be measured using HHD and assessed the following muscles: first dorsal interosseous, wrist extension, elbow extension, elbow flexion, shoulder flexion, knee extension, knee flexion, and ankle dorsiflexion, on both sides of the body. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [8]
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Baseline (Week 52) and Week 104
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Secondary outcome [9]
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OLP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 104
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Assessment method [9]
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Subjects with an event of death are reported. Subjects were planned to be assessed for permanent tracheostomy or permanent assisted ventilation in OLP; however, that data was not collected as study was terminated early.
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Timepoint [9]
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Baseline (Week 52) and Week 104
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Secondary outcome [10]
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OLP: Change From Baseline in ALS Assessment Questionnaire-40 at Week 104
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Assessment method [10]
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The ALSAQ-40 was a 40-item validated questionnaire designed to assess health related QoL over the previous 2 weeks in subjects with ALS. It represented 5 dimensions of health status; each scored from 0 (never, or best) to 4 (always, or worst). 5 dimensions evaluated were: physical mobility (10 items: 1-10; possible score of 0-40); activities of daily living/independence (10 items: 11-20; possible score of 0-40); eating and drinking (3 items: 21-23; possible score of 0-12); communication (7 items: 24-30; possible score: 0-28) and emotional functioning (10 items: 31-40; possible score: 0-40). The total score 0 (no impairment) to 160 (severe impairment) was planned to be calculated by adding the 5 dimension scores. Higher scores would have indicated worse QoL.
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Timepoint [10]
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Baseline (Week 52) and Week 104
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Secondary outcome [11]
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Number of Subjects With an Event of Death During the Study
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Assessment method [11]
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Total number of subjects who died in the study are reported.
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Timepoint [11]
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RTP: Baseline (Day 1) up to Week 52; OLP: Baseline (Week 52) up to Week 104
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/09/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
13/07/2023
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Sample size
Target
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Accrual to date
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Final
249
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Brain and Mind Centre - Camperdown
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Recruitment hospital [2]
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Central Coast Neurosciences Research - Erina
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Recruitment hospital [3]
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Royal Brisbane and Women's Hospital - Herston
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Recruitment hospital [4]
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Gold Coast University Hospital - Southport
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Recruitment hospital [5]
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Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health - Heidelberg
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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2250 - Erina
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Recruitment postcode(s) [3]
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4029 - Herston
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Recruitment postcode(s) [4]
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4215 - Southport
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Recruitment postcode(s) [5]
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3084 - Heidelberg
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Recruitment outside Australia
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United States of America
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California
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Colorado
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Indiana
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Maryland
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Minnesota
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Vermont
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Belgium
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Gent
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Leuven
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Czechia
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Prague 2
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Prague 6
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Bordeaux
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Bron
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Clermont-Ferrand
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Lille
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France
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Limoges
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France
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Nice
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Germany
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Berlin
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Hannover
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Jena
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Rostock
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Germany
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Ulm
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Milano
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Modena
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Italy
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Palermo
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Italy
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Torino
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Aichi
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Fukuoka
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Hokkaido
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Ishikawa
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Matsumoto
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Niigata
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Okinawa
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Saitama
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Shizuoka
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Tokyo
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Netherlands
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Utrecht
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Poland
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Olsztyn
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Warsaw
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Barcelona
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Spain
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Valencia
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Ukraine
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Kharkiv
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Ukraine
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Odessa
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Ukraine
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Zaporizhzhya
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United Kingdom
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Brighton
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Apellis Pharmaceuticals, Inc.
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Address
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Ethics approval
Ethics application status
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Summary
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)
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Trial website
https://clinicaltrials.gov/study/NCT04579666
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Contact person for public queries
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/66/NCT04579666/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/66/NCT04579666/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04579666
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