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An Efficacy and Safety Study of Ravulizumab in ALS Participants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04248465
Recruitment Status : Terminated (The IDMC recommended the study be discontinued due to lack of efficacy with ravulizumab.)
First Posted : January 30, 2020
Results First Posted : January 10, 2023
Last Update Posted : January 10, 2023
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Amyotrophic Lateral Sclerosis
ALS
Interventions Drug: Placebo
Biological: Ravulizumab
Enrollment 382
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ravulizumab/Ravulizumab Placebo/Ravulizumab
Hide Arm/Group Description Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then once every 8 weeks (q8w) up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 milligrams (mg) dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment. Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Period Title: Randomized-Controlled Period
Started 255 127
Received at Least 1 Dose of Study Drug 255 127
Completed 15 5
Not Completed 240 122
Reason Not Completed
Death             12             5
Adverse Event             2             0
Study Terminated by Sponsor             194             99
Withdrawal by Subject             30             17
Physician Decision             1             1
Lost to Follow-up             1             0
Period Title: Open-label Extension Period
Started 14 5
Received at Least 1 Dose of Study Drug 14 5
Completed 0 0
Not Completed 14 5
Reason Not Completed
Study Terminated by Sponsor             14             4
Withdrawal by Subject             0             1
Arm/Group Title Ravulizumab Placebo Total
Hide Arm/Group Description Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment. Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment. Total of all reporting groups
Overall Number of Baseline Participants 255 127 382
Hide Baseline Analysis Population Description
Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 255 participants 127 participants 382 participants
58.6  (10.57) 58.0  (11.03) 58.4  (10.72)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 255 participants 127 participants 382 participants
Female
94
  36.9%
58
  45.7%
152
  39.8%
Male
161
  63.1%
69
  54.3%
230
  60.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 255 participants 127 participants 382 participants
Hispanic or Latino
22
   8.6%
12
   9.4%
34
   8.9%
Not Hispanic or Latino
205
  80.4%
105
  82.7%
310
  81.2%
Unknown or Not Reported
28
  11.0%
10
   7.9%
38
   9.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 255 participants 127 participants 382 participants
American Indian or Alaska Native
0
   0.0%
2
   1.6%
2
   0.5%
Asian
22
   8.6%
12
   9.4%
34
   8.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   0.4%
0
   0.0%
1
   0.3%
White
202
  79.2%
103
  81.1%
305
  79.8%
More than one race
4
   1.6%
0
   0.0%
4
   1.0%
Unknown or Not Reported
26
  10.2%
10
   7.9%
36
   9.4%
1.Primary Outcome
Title Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score
Hide Description The ALSFRS-Revised is a validated instrument for evaluating the levels of the functional status of participants with amyotrophic lateral sclerosis (ALS) in 4 areas, including bulbar, gross motor activity, fine motor activity, and respiratory functions. The scale included 12 functional items and each item is rated on a 0 to 4 scale, with a maximum total score of 48. A higher score indicated greater retention of function. Baseline was defined as last non-missing value on or before first study drug administration.
Time Frame Baseline, Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 32 14
Mean (Standard Deviation)
Unit of Measure: units on a scale
-11.9  (7.30) -10.6  (6.05)
2.Secondary Outcome
Title Time To Ventilator Assistance-free Survival
Hide Description Ventilation Assistance-Free Survival (VAFS) is a composite endpoint of survival and severe and irreversible respiratory decline. The use of VAFS allowed for the collection of survival data that was not impacted by survival prolongation from noninvasive or permanent ventilatory interventions which could prolong life without impacting underlying disease progression.
Time Frame Up to Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 255 127
Median (Full Range)
Unit of Measure: months
6.05
(0.79 to 11.10)
7.69
(4.83 to 9.53)
3.Secondary Outcome
Title Change From Baseline In Percent Predicted Slow Vital Capacity
Hide Description Slow vital capacity measures slow and gradual expulsion of air from the lungs using a spirometer.
Time Frame Baseline, Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 19 9
Mean (Standard Deviation)
Unit of Measure: percentage of predicted volume
-20.9  (19.74) -21.3  (13.90)
4.Secondary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, and TEAEs Leading To Study Drug Discontinuation
Hide Description An adverse event (AE) was defined as any unfavorable and unintended sign (for example, including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product or procedure, whether or not considered related to the medicinal product or procedure, which occurred during the course of the clinical study. TEAEs were defined as AEs that occurred on or after the date and time of study drug administration, or those that first occurred before dosing but worsened in frequency or severity after study drug administration. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time Frame Baseline up to Week 156
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set included all participants who received at least 1 dose of study drug grouped by treatment actually received (for reporting exposure and safety data).
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 255 127
Measure Type: Count of Participants
Unit of Measure: Participants
TEAEs
204
  80.0%
108
  85.0%
Treatment Emergent Serious AEs
41
  16.1%
24
  18.9%
TEAE Leading to Study Drug Discontinuation
2
   0.8%
0
   0.0%
5.Secondary Outcome
Title Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry
Hide Description Handheld dynamometry (HHD) is a procedure for quantitative strength testing. Muscle strength testing was performed on prespecified muscles in the upper and lower extremities bilaterally and the force measurements were recorded. Force of measurement is reported in megascores (lower, upper, total). The total megascore is defined as the average of the non-missing ratios over baseline for all the muscles involved. The megascore at baseline is always 100. The range of a potential megascore can not be determined in advance. A megascore >100 indicates more strength compared to baseline.
Time Frame Baseline, Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 26 13
Mean (Standard Deviation)
Unit of Measure: % (as the unit of megascore)
-46.5  (27.57) -53.4  (20.28)
6.Secondary Outcome
Title Change From Baseline In Serum Neurofilament Light Chain
Hide Description [Not Specified]
Time Frame Baseline, Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 14 7
Mean (Standard Deviation)
Unit of Measure: picograms/milliliter
91.5  (40.40) 73.1  (27.82)
7.Secondary Outcome
Title Change From Baseline in Serum Ravulizumab Concentration Over the Study Duration
Hide Description [Not Specified]
Time Frame Baseline, Predose at Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic Analysis Set (PKAS) included all participants who received at least 1 dose of the study drug and had at least 1 postdose pharmacokinetic (PK) sample. This endpoint was planned to be reported for Ravulizumab arm only.
Arm/Group Title Ravulizumab
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 14
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: micrograms/milliliter
634
(29.1%)
8.Secondary Outcome
Title Change From Baseline in Serum Free Complement Component 5 (C5) Concentration Over the Study Duration
Hide Description [Not Specified]
Time Frame Baseline, Predose at Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic analysis set (PDAS) included all participants who received at least 1 dose of the study drug and had at least 1 postdose pharmacodynamics (PD) sample. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure. There were no participants with evaluable C5 data in the Placebo arm at Week 50.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 13 0
Mean (Standard Deviation)
Unit of Measure: micrograms/milliliter
-155.2  (24.42)
9.Secondary Outcome
Title Number of Participants With Positive Antidrug Antibodies (ADAs) to ALXN1210
Hide Description Blood samples were collected to evaluate antibody response through development of ADAs.
Time Frame Week 50
Hide Outcome Measure Data
Hide Analysis Population Description
PDAS included all participants who received at least 1 dose of the study drug and had at least 1 postdose PD sample. Here, Number of Participants analyzed signifies those participants who were evaluable at Week 50. There were no participants with evaluable C5 data in the Placebo arm at Week 50.
Arm/Group Title Ravulizumab Placebo
Hide Arm/Group Description:
Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
Overall Number of Participants Analyzed 23 0
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
Time Frame Baseline up to Week 156
Adverse Event Reporting Description Safety Set included all participants who received at least 1 dose of study drug grouped by treatment actually received (for reporting exposure and safety data). "All-Cause Mortality" reports all deaths that occurred during the study, including the deaths that led to Study Discontinuation.
 
Arm/Group Title Randomized Controlled Period: Ravulizumab Randomized Controlled Period: Placebo Open Label Extension Period: Ravulizumab Open Label Extension Period: Placebo
Hide Arm/Group Description Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive). Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive). Participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment. Participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
All-Cause Mortality
Randomized Controlled Period: Ravulizumab Randomized Controlled Period: Placebo Open Label Extension Period: Ravulizumab Open Label Extension Period: Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   15/255 (5.88%)      6/127 (4.72%)      0/14 (0.00%)      0/5 (0.00%)    
Hide Serious Adverse Events
Randomized Controlled Period: Ravulizumab Randomized Controlled Period: Placebo Open Label Extension Period: Ravulizumab Open Label Extension Period: Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   41/255 (16.08%)      24/127 (18.90%)      2/14 (14.29%)      0/5 (0.00%)    
Cardiac disorders         
Cardio-respiratory arrest * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Gastrointestinal disorders         
Abdominal pain * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Abdominal pain upper * 1  1/255 (0.39%)  1 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Colitis ulcerative * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Dysphagia * 1  1/255 (0.39%)  1 2/127 (1.57%)  2 0/14 (0.00%)  0 0/5 (0.00%)  0
Pancreatitis * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Pancreatitis relapsing * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Constipation * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
General disorders         
Death * 1  2/255 (0.78%)  2 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Complication associated with device * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Pyrexia * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Hepatobiliary disorders         
Cholelithiasis * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Cholecystitis * 1  0/255 (0.00%)  0 0/127 (0.00%)  0 1/14 (7.14%)  1 0/5 (0.00%)  0
Immune system disorders         
Infusion related hypersensitivity reaction * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Infections and infestations         
Pneumonia * 1  3/255 (1.18%)  3 5/127 (3.94%)  6 0/14 (0.00%)  0 0/5 (0.00%)  0
Pneumonia aspiration * 1  2/255 (0.78%)  2 2/127 (1.57%)  2 0/14 (0.00%)  0 0/5 (0.00%)  0
Bronchitis * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Pneumonia bacterial * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Pyelonephritis * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Respiratory tract infection * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Appendicitis * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
COVID-19 * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 1/14 (7.14%)  1 0/5 (0.00%)  0
COVID-19 pneumonia * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Injury, poisoning and procedural complications         
Femoral neck fracture * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Femur fracture * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Gastrostomy failure * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Gastrostomy tube site complication * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Hip fracture * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Rib fracture * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Skin laceration * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Infusion related reaction * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Investigations         
Myocardial necrosis marker increased * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Weight decreased * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
SARS-CoV-2 test positive * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Metabolism and nutrition disorders         
Dehydration * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Euglycaemic diabetic ketoacidosis * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Malnutrition * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Nervous system disorders         
Amyotrophic lateral sclerosis * 1  1/255 (0.39%)  1 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Headache * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Subarachnoid haemorrhage * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Transient ischaemic attack * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Haemorrhage intracranial * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Syncope * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Psychiatric disorders         
Completed suicide * 1  2/255 (0.78%)  2 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Assisted suicide * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Mental status changes * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Respiratory failure * 1  8/255 (3.14%)  8 4/127 (3.15%)  4 0/14 (0.00%)  0 0/5 (0.00%)  0
Dyspnoea * 1  3/255 (1.18%)  3 2/127 (1.57%)  2 0/14 (0.00%)  0 0/5 (0.00%)  0
Atelectasis * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Pneumonitis aspiration * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Pulmonary embolism * 1  1/255 (0.39%)  1 4/127 (3.15%)  4 0/14 (0.00%)  0 0/5 (0.00%)  0
Respiratory disorder * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Respiratory distress * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Aspiration * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Cough * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
Social circumstances         
Feeding tube user * 1  1/255 (0.39%)  1 0/127 (0.00%)  0 0/14 (0.00%)  0 0/5 (0.00%)  0
Surgical and medical procedures         
Euthanasia * 1  0/255 (0.00%)  0 1/127 (0.79%)  1 0/14 (0.00%)  0 0/5 (0.00%)  0
1
Term from vocabulary, MedDRA v24.1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Randomized Controlled Period: Ravulizumab Randomized Controlled Period: Placebo Open Label Extension Period: Ravulizumab Open Label Extension Period: Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   196/255 (76.86%)      106/127 (83.46%)      3/14 (21.43%)      1/5 (20.00%)    
Gastrointestinal disorders         
Constipation * 1  26/255 (10.20%)  29 5/127 (3.94%)  5 0/14 (0.00%)  0 0/5 (0.00%)  0
Nausea * 1  23/255 (9.02%)  28 10/127 (7.87%)  10 0/14 (0.00%)  0 0/5 (0.00%)  0
Diarrhoea * 1  4/255 (1.57%)  6 9/127 (7.09%)  10 0/14 (0.00%)  0 0/5 (0.00%)  0
General disorders         
Fatigue * 1  22/255 (8.63%)  40 11/127 (8.66%)  11 0/14 (0.00%)  0 0/5 (0.00%)  0
Hepatobiliary disorders         
Cholelithiasis * 1  0/255 (0.00%)  0 0/127 (0.00%)  0 1/14 (7.14%)  1 0/5 (0.00%)  0
Infections and infestations         
Urinary tract infection * 1  12/255 (4.71%)  12 11/127 (8.66%)  12 0/14 (0.00%)  0 0/5 (0.00%)  0
Hordeolum * 1  0/255 (0.00%)  0 0/127 (0.00%)  0 0/14 (0.00%)  0 1/5 (20.00%)  1
Injury, poisoning and procedural complications         
Fall * 1  54/255 (21.18%)  81 36/127 (28.35%)  65 1/14 (7.14%)  1 1/5 (20.00%)  1
Contusion * 1  11/255 (4.31%)  13 8/127 (6.30%)  13 0/14 (0.00%)  0 0/5 (0.00%)  0
Ankle fracture * 1  0/255 (0.00%)  0 0/127 (0.00%)  0 0/14 (0.00%)  0 1/5 (20.00%)  1
Musculoskeletal and connective tissue disorders         
Back Pain * 1  21/255 (8.24%)  31 5/127 (3.94%)  7 0/14 (0.00%)  0 0/5 (0.00%)  0
Arthralgia * 1  18/255 (7.06%)  23 4/127 (3.15%)  5 0/14 (0.00%)  0 0/5 (0.00%)  0
Nervous system disorders         
Headache * 1  42/255 (16.47%)  62 22/127 (17.32%)  29 1/14 (7.14%)  1 0/5 (0.00%)  0
Psychiatric disorders         
Insomnia * 1  13/255 (5.10%)  14 8/127 (6.30%)  8 0/14 (0.00%)  0 0/5 (0.00%)  0
Skin and subcutaneous tissue disorders         
Dermatitis contact * 1  0/255 (0.00%)  0 0/127 (0.00%)  0 1/14 (7.14%)  1 0/5 (0.00%)  0
1
Term from vocabulary, MedDRA v24.1
*
Indicates events were collected by non-systematic assessment
The IDMC recommended the study be discontinued due to lack of efficacy with ravulizumab.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
Phone: +1 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04248465    
Other Study ID Numbers: ALXN1210-ALS-308
2019-004619-30 ( EudraCT Number )
First Submitted: January 27, 2020
First Posted: January 30, 2020
Results First Submitted: September 22, 2022
Results First Posted: January 10, 2023
Last Update Posted: January 10, 2023