Trial Outcomes & Findings for Study of Eteplirsen in DMD Patients (NCT NCT02255552)
NCT ID: NCT02255552
Last Updated: 2021-01-25
Results Overview
6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 96 was reported.
COMPLETED
PHASE3
109 participants
Baseline, Week 96
2021-01-25
Participant Flow
The study was conducted at 40 sites in the United States.
A total of 109 participants were enrolled in the study. Only 79 participants were treated and the remaining participants were not applicable for treatment as those participants assessed under "Untreated" arm.
Participant milestones
| Measure |
Eteplirsen 30 mg/kg
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
79
|
30
|
|
Overall Study
COMPLETED
|
78
|
15
|
|
Overall Study
NOT COMPLETED
|
1
|
15
|
Reasons for withdrawal
| Measure |
Eteplirsen 30 mg/kg
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Overall Study
Participants enrolled into another study
|
0
|
11
|
|
Overall Study
Withdrawal by Subject
|
1
|
4
|
Baseline Characteristics
Study of Eteplirsen in DMD Patients
Baseline characteristics by cohort
| Measure |
Eteplirsen 30 mg/kg
n=79 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=30 Participants
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
Total
n=109 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
9.1 Years
STANDARD_DEVIATION 2.04 • n=5 Participants
|
8.8 Years
STANDARD_DEVIATION 1.76 • n=7 Participants
|
9.0 Years
STANDARD_DEVIATION 1.96 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
79 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
109 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
71 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
95 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
67 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
79 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
109 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 96Population: Primary efficacy set: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure.
6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 96 was reported.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=65 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=9 Participants
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96
|
-117.91 meters
Standard Deviation 128.488
|
-133.56 meters
Standard Deviation 129.333
|
SECONDARY outcome
Timeframe: Baseline, Week 96Population: Analysis Set consisted of a subset of participants who received at least 1 dose of eteplirsen and had both baseline and 1 post-dose muscle biopsy samples evaluable for dystrophin expression at Week 96. Here, "Overall Number of Participants Analyzed" signifies number of participants who were evaluable for this outcome measure. Data for this outcome measure was not planned to be collected and analyzed for the Untreated Control group.
Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=16 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96
|
0.516 Percent Normal Dystrophin Protein Level
Standard Deviation 0.7236
|
—
|
SECONDARY outcome
Timeframe: Week 96Population: Primary efficacy set: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure.
NSAA is a clinician-administered scale that rates participant performance on 17-items and included assessments of abilities such as 10-meter walk/run, rising from a sit to stand, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. For all activities, participants were graded as follows: 0 = unable to achieve goal independently; 1 = modified method but achieves goal independent of physical assistance from another and 2 = normal, no obvious modification of activity. Number of participants having ability to rise independently from the floor indicated by a NSAA Rise from floor sub score greater than 0 (unable to achieve goal independently) was reported.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=61 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=9 Participants
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96
|
33 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Up to Week 96Population: Primary efficacy set consisted of all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive.
Number of participants who lost ambulation (LOA) by Week 96 was reported. Participant were considered non-ambulatory if each of the 3 conditions below were met: NSAA walk subscore was "0" (unable to achieve goal independently) on 2 consecutive days within a visit or NSAA was not done due to reason related to non-ambulation; 6MWT was not done with any reason related to permanent non-ambulation; and no later data showing this participant was still ambulatory. This was not required if non ambulatory status occurred at the time of early withdrawal or at the end of Week 96 assessment. NSAA is a 17-item scale to assess the participant's abilities; total score range from 0 (if all the activities are failed) to 34 (if all the activities are achieved) with higher scores indicating better performance on the assessment/ fully-independent function.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=67 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=20 Participants
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Number of Participants Who Lost Ambulation (LOA) by Week 96
|
12 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 96Population: Primary efficacy set: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure.
FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry; and is the most important measurement of lung function. This test requires participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate participant's bronchial (breathing) tubes. Percent of predicted FVC = (observed value) / (predicted value) \* 100%.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=66 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=9 Participants
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96
|
-3.413 Percentage of predicted FVC
Standard Deviation 12.4011
|
-2.461 Percentage of predicted FVC
Standard Deviation 9.6000
|
SECONDARY outcome
Timeframe: Baseline, Week 96Population: Primary efficacy: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure.
NSAA is a clinician-administered scale that rates participant performance on 17-items and included assessments of abilities such as 10-meter walk/run, rising from a sit to stand, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participant were graded as follows: 0 = unable to achieve goal independently; 1 = modified method but achieves goal independent of physical assistance from another and 2 = normal, no obvious modification of activity. NSAA total score was derived by summing the scores for all the individual items and range from 0 (if all the activities are failed) to 34 (if all the activities are achieved) with higher scores indicating better performance on the assessment/ fully-independent function.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=61 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=9 Participants
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96
|
-7.23 Unit on scale
Standard Deviation 5.173
|
-8.44 Unit on scale
Standard Deviation 9.812
|
SECONDARY outcome
Timeframe: Baseline, Week 96Population: Analysis Set consisted of a subset of participants who received at least 1 dose of eteplirsen and had both baseline and 1 post-dose muscle biopsy samples evaluable for dystrophin expression at Week 96. Here, "Overall Number of Participants Analyzed" signifies number of participants who were evaluable for this outcome measure. Data for this outcome measure was not planned to be collected and analyzed for the Untreated Control group.
Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=16 Participants
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96
|
0.030 Percent dystrophin positive fibers
Standard Deviation 0.0360
|
—
|
Adverse Events
Eteplirsen 30 mg/kg
Untreated Control Group (Non-exon 51 Amenable Participants)
Serious adverse events
| Measure |
Eteplirsen 30 mg/kg
n=79 participants at risk
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=30 participants at risk
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Femoral neck fracture
|
2.5%
2/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Femur fracture
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Hip fracture
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Pneumonitis chemical
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Cardiac disorders
Arrhythmia
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Cardiac disorders
Myocarditis
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Nervous system disorders
Loss of consciousness
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Gait disturbance
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Abasia
|
0.00%
0/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Psychiatric disorders
Aggression
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Psychiatric disorders
Major depression
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Catheter site infection
|
1.3%
1/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Lymphadenitis viral
|
0.00%
0/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
Other adverse events
| Measure |
Eteplirsen 30 mg/kg
n=79 participants at risk
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks.
|
Untreated Control Group (Non-exon 51 Amenable Participants)
n=30 participants at risk
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks.
|
|---|---|---|
|
Vascular disorders
Flushing
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
6.3%
5/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Immune system disorders
Seasonal allergy
|
17.7%
14/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Pyrexia
|
25.3%
20/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
General disorders
Non-cardiac chest pain
|
11.4%
9/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Fatigue
|
10.1%
8/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Catheter site pain
|
8.9%
7/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Peripheral swelling
|
6.3%
5/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Infusion site bruising
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
General disorders
Infusion site pain
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Psychiatric disorders
Anxiety
|
7.6%
6/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Psychiatric disorders
Insomnia
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Procedural pain
|
31.6%
25/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Contusion
|
30.4%
24/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Fall
|
27.8%
22/79 • From start of study drug administration to Week 144
|
20.0%
6/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
26.6%
21/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
15.2%
12/79 • From start of study drug administration to Week 144
|
10.0%
3/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
12.7%
10/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Muscle strain
|
8.9%
7/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Laceration
|
5.1%
4/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Limb injury
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Injury, poisoning and procedural complications
Torus fracture
|
0.00%
0/79 • From start of study drug administration to Week 144
|
6.7%
2/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
45.6%
36/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
31.6%
25/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
27.8%
22/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
25.3%
20/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
13.9%
11/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
8.9%
7/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
5.1%
4/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Nervous system disorders
Headache
|
50.6%
40/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Nervous system disorders
Dizziness
|
11.4%
9/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Nervous system disorders
Migraine
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Ear and labyrinth disorders
Ear pain
|
5.1%
4/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Vomiting
|
49.4%
39/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Diarrhoea
|
25.3%
20/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Nausea
|
21.5%
17/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Abdominal pain upper
|
17.7%
14/79 • From start of study drug administration to Week 144
|
10.0%
3/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Abdominal pain
|
10.1%
8/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Constipation
|
10.1%
8/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
6.3%
5/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.1%
4/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Renal and urinary disorders
Proteinuria
|
10.1%
8/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Skin and subcutaneous tissue disorders
Rash
|
21.5%
17/79 • From start of study drug administration to Week 144
|
6.7%
2/30 • From start of study drug administration to Week 144
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.6%
6/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
36.7%
29/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
32.9%
26/79 • From start of study drug administration to Week 144
|
6.7%
2/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
20.3%
16/79 • From start of study drug administration to Week 144
|
6.7%
2/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
11.4%
9/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
7.6%
6/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
7.6%
6/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
5.1%
4/79 • From start of study drug administration to Week 144
|
6.7%
2/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Nasopharyngitis
|
36.7%
29/79 • From start of study drug administration to Week 144
|
10.0%
3/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Upper respiratory tract infection
|
31.6%
25/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Ear infection
|
13.9%
11/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Influenza
|
10.1%
8/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Gastroenteritis
|
8.9%
7/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Gastroenteritis viral
|
8.9%
7/79 • From start of study drug administration to Week 144
|
0.00%
0/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Pharyngitis streptococcal
|
7.6%
6/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
|
Infections and infestations
Sinusitis
|
7.6%
6/79 • From start of study drug administration to Week 144
|
3.3%
1/30 • From start of study drug administration to Week 144
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER