Amyotrophic Lateral SclerosisPhase 2NCT04579666
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing Pegcetacoplan (APL-2) in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Apellis Pharmaceuticals, Inc. at 60 sites in 14 countries.
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) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- Pegcetacoplan (APL-2)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 249 participants
- Where
- 60 sitesin 14 countries
- Study dates
- 30 Sep 2020to 13 Jul 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
60 sites in 14 countries.
60 sites in 14 countries
Japan12 sites
National Hospital Organization Higashinagoya National Hospital
Aichi, 465-8620
National Hospital Organization Omuta National Hospital
Fukuoka, 837-0911
National Hospital Organization Asahikawa Medical Center
Hokkaido, 070-8644
National Hospital Organization Hyogo-Chuo National Hospital
Hyōgo, 669-1592
National Hospital Organization Iou National Hospital
Ishikawa, 920-0192
National Hospital Organization Matsumoto Medical Center
Matsumoto, 399-8701
Niigata National Hospital National Hospital Organization
Niigata, 945-8585
National Hospital Organization Okinawa National Hospital
Okinawa, 901-2214
National Hospital Organization Higashisaitama National Hospital
Saitama, 349-0196
Shizuoka Institute of Epilepsy and Neurological Disorders
Shizuoka, 420-8688
Juntendo University Hospital
Tokyo, 113-8431
Tokyo Medical University Hospital
Tokyo, 160-0023
United States10 sites
Augusta University
Augusta, Georgia, 30912
University of Colorado
Aurora, Colorado, 80045
Austin Neuromuscular Center
Austin, Texas, 78756
Johns Hopkins
Baltimore, Maryland, 21205
University of Vermont Medical Center
Burlington, Vermont, 05401
Indiana University
Indianapolis, Indiana, 46202
Cedars-Sinai Medical Center
Los Angeles, California, 90048
The Berman Center
Minneapolis, Minnesota, 55415
Hospital for Special Surgery
New York, New York, 10021
University of South Florida
Tampa, Florida, 33612
France6 sites
Hopital Pellegrin
Bordeaux, 33076
Hôpital Neurologique Pierre Wertheimer
Bron, 69677
CHU Gabriel Montpied
Clermont-Ferrand, 63000
Hôpital Roger Salengro
Lille, 59037
CHU de Limoges Dupuytren 1
Limoges, 87042
CHU de Nice Hôpital Pasteur
Nice, 6300
Australia5 sites
Brain and Mind Centre
Camperdown, New South Wales, 2050
Central Coast Neurosciences Research
Erina, New South Wales, 2250
Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health
Heidelberg, Victoria, 3084
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
Gold Coast University Hospital
Southport, Queensland, 4215
Germany5 sites
Charité - Universitätsmedizin Berlin
Berlin, D-13353
Medizinische Hochschule Hannover Klinik für Neurologie
Hanover, 30625
Universitätsklinikum Jena
Jena, 07747
Universitätsmedizin Rostock, Klinik und Poliklinik für Neurologie
Rostock, 18147
University of Ulm
Ulm, 89081
Italy4 sites
Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus
Milan, 20162
Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena
Modena, 41126
AOUP "P. Giaccone"
Palermo, 90129
Azienda Ospedaliera Universitaria di Torino - Città della Salute e della Scienza di Torino
Torino, 10126
Poland3 sites
Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii
Olsztyn, 10-082
Centrum Medyczne NeuroProtect
Warsaw, 01-684
City Clinic Sp. z o.o.
Warsaw, 02-473
Spain3 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Bellvitge University Hospital
Barcelona, 08907
Hospital Universitari I Politecnic La Fe
Valencia, 46026
Ukraine3 sites
SI Institute of Neurology, Psychiatry and Narcology of NAMSU
Kharkiv, 61068
Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University
Odesa, 65062
Zaporizhzhya Regional Clinical Hospital
Zaporizhzhya, 69600
United Kingdom3 sites
University Hospitals Sussex NHS Foundation Trust
Brighton, BN2 5BE
Maurice Wohl Clinical Neuroscience Institute, King's College London
London, SE5 9RX
St George's University Hospitals NHS Foundation Trust
London, SW17 0WT
Belgium2 sites
AZ Sint-Lucas & Volkskliniek
Ghent, B-9000
Universitaire Ziekenhuizen Leuven (UZ Leuven)
Leuven, B-3000
Czechia2 sites
Vseobecna fakultni nemocnice v Praze
Prague, 128 21
FORBELI s.r.o.
Prague, 160 00
About this study
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)
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)
- Being tested
- Pegcetacoplan (APL-2), Placebo
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, Motor Neuron Disease, APL-2, APL2, Pegcetacoplan
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pegcetacoplan (APL-2)Drug
Complement (C3) Inhibitor
PlaceboOther
Sterile solution of equal volume to active arm
| Study group | Role | What they receive |
|---|---|---|
| 1,080 mg pegcetacoplan (APL-2) | Experimental | administered subcutaneously twice weekly Receives: Drug: Pegcetacoplan (APL-2) |
| Placebo administered subcutaneously twice weekly | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
RTP: Combined Assessment of Function and Survival (CAFS) Rank Score (Joint-Rank Score) at Week 52
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.
Measured: Week 52
RTP: Number of Subjects With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
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.
Measured: From first dose of study drug (Day 1) up to 56 days post last dose of study drug, approximately 60 weeks
OLP: Number of Participants With Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events
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.
Measured: From first dose of study drug (Week 52) up to 56 days post last dose of study drug, approximately 60 weeks
RTP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale (C-SSRS) up to Week 52
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.
Measured: Baseline (Day 1) up to Week 52
OLP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale up to Week 104
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.
Measured: From Baseline (Week 52) up to Week 104
Secondary outcomes (11)
RTP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score at Week 52
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.
Measured: Baseline (Day 1) and Week 52
RTP: Change From Baseline in Percent Predicted Slow Vital Capacity (%SVC) at Week 52
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.
Measured: Baseline (Day 1) and Week 52
RTP: Change From Baseline in Muscle Strength at Week 52
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.
Measured: Baseline (Day 1) and Week 52
RTP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 52
Subjects with an event (that is, either death or permanent tracheostomy or permanent assisted ventilation) in RTP are reported.
Measured: Baseline (Day 1) up to Week 52
RTP: Change From Baseline in ALS Assessment Questionnaire (ALSAQ)-40 at Week 52
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.
Measured: Baseline (Day 1) and Week 52
OLP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Score at Week 104
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.
Measured: Baseline (Week 52) and Week 104
OLP: Change From Baseline in Percent Predicted Slow Vital Capacity at Week 104
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.
Measured: Baseline (Week 52) and Week 104
OLP: Change From Baseline in Muscle Strength at Week 104
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.
Measured: Baseline (Week 52) and Week 104
OLP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 104
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.
Measured: Baseline (Week 52) and Week 104
OLP: Change From Baseline in ALS Assessment Questionnaire-40 at Week 104
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.
Measured: Baseline (Week 52) and Week 104
Number of Subjects With an Event of Death During the Study
Total number of subjects who died in the study are reported.
Measured: RTP: Baseline (Day 1) up to Week 52; OLP: Baseline (Week 52) up to Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2020
- Study start30 Sep 2020
- Primary completion31 Mar 2023
- Study completion13 Jul 2023
Why it stopped: The study did not meet primary endpoint at Week 52. The study also did not meet key secondary efficacy endpoints. Pegcetacoplan (APL-2) was well tolerated in the study, and the data were consistent with the established safety profile.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 249 participants (actual)
Sponsor and investigators
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04579666
- Sponsor's protocol ID
- APL2-ALS-206
- Status last verified
- Apr 2025
- First submitted
- 22 Sep 2020
- Last updated
- 24 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04579666
- Acronym / sponsor ID
- APL2-ALS-206
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, Motor Neuron Disease, APL-2, APL2, Pegcetacoplan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study did not meet primary endpoint at Week 52. The study also did not meet key secondary efficacy endpoints. Pegcetacoplan (APL-2) was well tolerated in the study, and the data were consistent with the established safety profile.
- Enrolment
- 249 participants (actual)
- Lead sponsor
- Apellis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Sep 2020 (actual)
- Primary completion
- 31 Mar 2023 (actual)
- Study completion
- 13 Jul 2023 (actual)
- First posted
- 8 Oct 2020 (actual)
- Results
- Posted 24 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1,080 mg pegcetacoplan (APL-2) | Experimental | Drug: Pegcetacoplan (APL-2)administered subcutaneously twice weekly |
| Placebo administered subcutaneously twice weekly | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pegcetacoplan (APL-2)Complement (C3) Inhibitor | Drug | 1,080 mg pegcetacoplan (APL-2) |
| PlaceboSterile solution of equal volume to active arm | Other | Placebo administered subcutaneously twice weekly |
Primary outcome
RTP: Combined Assessment of Function and Survival (CAFS) Rank Score (Joint-Rank Score) at Week 52
Time frame: Week 52
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.
RTP: Number of Subjects With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: From first dose of study drug (Day 1) up to 56 days post last dose of study drug, approximately 60 weeks
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.
OLP: Number of Participants With Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events
Time frame: From first dose of study drug (Week 52) up to 56 days post last dose of study drug, approximately 60 weeks
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.
RTP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale (C-SSRS) up to Week 52
Time frame: Baseline (Day 1) up to Week 52
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.
OLP: Number of Subjects With Positive Response to Columbia-Suicide Severity Rating Scale up to Week 104
Time frame: From Baseline (Week 52) up to Week 104
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.
11 secondary outcomes
RTP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score at Week 52
Time frame: Baseline (Day 1) and Week 52
RTP: Change From Baseline in Percent Predicted Slow Vital Capacity (%SVC) at Week 52
Time frame: Baseline (Day 1) and Week 52
RTP: Change From Baseline in Muscle Strength at Week 52
Time frame: Baseline (Day 1) and Week 52
RTP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 52
Time frame: Baseline (Day 1) up to Week 52
RTP: Change From Baseline in ALS Assessment Questionnaire (ALSAQ)-40 at Week 52
Time frame: Baseline (Day 1) and Week 52
OLP: Change From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Score at Week 104
Time frame: Baseline (Week 52) and Week 104
OLP: Change From Baseline in Percent Predicted Slow Vital Capacity at Week 104
Time frame: Baseline (Week 52) and Week 104
OLP: Change From Baseline in Muscle Strength at Week 104
Time frame: Baseline (Week 52) and Week 104
OLP: Number of Subjects With an Event of Death or Permanent Tracheostomy or Permanent Assisted Ventilation at Week 104
Time frame: Baseline (Week 52) and Week 104
OLP: Change From Baseline in ALS Assessment Questionnaire-40 at Week 104
Time frame: Baseline (Week 52) and Week 104
Number of Subjects With an Event of Death During the Study
Time frame: RTP: Baseline (Day 1) up to Week 52; OLP: Baseline (Week 52) up to Week 104
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
60 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pegcetacoplan (APL-2) in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Apellis Pharmaceuticals, Inc. at 60 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 60 sites in Japan, United States, France, Australia, Germany and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
RTP: Combined Assessment of Function and Survival (CAFS) Rank Score (Joint-Rank Score) at Week 52 (time frame: Week 52). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2025.