Trial Outcomes & Findings for Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy (NCT NCT02420379)
NCT ID: NCT02420379
Last Updated: 2021-01-25
Results Overview
Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug \[up to 100 weeks\]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events.
COMPLETED
PHASE2
33 participants
Baseline up to 100 weeks
2021-01-25
Participant Flow
The study was conducted at 13 sites in the United States.
A total of 33 participants were enrolled in the study treatment.
Participant milestones
| Measure |
Eteplirsen 30 mg/kg
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
26
|
7
|
|
Overall Study
COMPLETED
|
25
|
3
|
|
Overall Study
NOT COMPLETED
|
1
|
4
|
Reasons for withdrawal
| Measure |
Eteplirsen 30 mg/kg
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
3
|
|
Overall Study
Unable to complete due to schooling
|
0
|
1
|
|
Overall Study
Transitioned to commercial drug
|
1
|
0
|
Baseline Characteristics
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
Total
n=33 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
5.0 years
STANDARD_DEVIATION 0.82 • n=5 Participants
|
5.0 years
STANDARD_DEVIATION 1.00 • n=7 Participants
|
5.0 years
STANDARD_DEVIATION 0.85 • 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
|
26 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
21 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=5 Participants
|
7 participants
n=7 Participants
|
33 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 100 weeksPopulation: Full set included all participants who were enrolled in the eteplirsen group and received at least 1 dose of eteplirsen as well as all participants who were enrolled in the untreated group and had at least 1 assessment post-enrollment assessment.
Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug \[up to 100 weeks\]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
Participants with TEAEs
|
26 Participants
|
5 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
Participants with Serious TEAEs
|
4 Participants
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
Participants with TEAEs leading to discontinuation
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 100 weeksPopulation: Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
SC: Blood creatine phosphokinase increased
|
1 Participants
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
Hematology: Anaemia
|
1 Participants
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
Hematology: Iron deficiency Anaemia
|
1 Participants
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
Urinalysis: chromaturia
|
3 Participants
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
Urinalysis: pollakiuria
|
1 Participants
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
Coagulation: Thrombocytopenia
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 100 weeksPopulation: Full set included all participants who were enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
Pyrexia
|
11 Participants
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
Pulse pressure increased
|
1 Participants
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
Tachycardia
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 100 weeksPopulation: Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment postenrollment assessment.
Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Number of participants with at least one abnormal physical examination findings were reported. Abnormality in physical examinations was based on Investigator's discretion.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Number of Participants With at Least One Abnormal Physical Examination Finding
|
23 Participants
|
4 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 96 weeksPopulation: Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at 1 assessment post-enrollment assessment.
Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECGs were reported as TEAEs.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 96 weeksPopulation: Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECHO were reported as TEAEs.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=26 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 Participants
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 48 and 96Population: Muscle Biopsy Set included all participants who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable participants). Here, number of participants analyzed signifies participants who were evaluable at specific time point.
Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 48 and 96 was reported. 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=25 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96
Change at Week 48
|
0.102 Percent Normal Dystrophin Protein Level
Standard Deviation 0.0896
|
—
|
|
Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96
Change at Week 96
|
0.321 Percent Normal Dystrophin Protein Level
Standard Deviation 0.4863
|
—
|
SECONDARY outcome
Timeframe: Baseline, Week 48 and 96Population: Muscle Biopsy Set included all participants who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable participants). Here, number of participants analyzed signifies participants who were evaluable at specific time point.
Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry at Week 48 and 96 was reported.
Outcome measures
| Measure |
Eteplirsen 30 mg/kg
n=25 Participants
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96
Change at Week 48
|
0.004 Percent dystrophin positive fibers
Standard Deviation 0.0096
|
—
|
|
Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96
Change at Week 96
|
0.015 Percent dystrophin positive fibers
Standard Deviation 0.0175
|
—
|
Adverse Events
Eteplirsen 30 mg/kg
Control Group (Untreated) (Non-exon 51 Amenable Participants)
Serious adverse events
| Measure |
Eteplirsen 30 mg/kg
n=26 participants at risk
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 participants at risk
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Foreign body
|
3.8%
1/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Coxsackie viral infection
|
3.8%
1/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Influenza
|
3.8%
1/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Rhinovirus infection
|
3.8%
1/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
3.8%
1/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
Other adverse events
| Measure |
Eteplirsen 30 mg/kg
n=26 participants at risk
Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
|
Control Group (Untreated) (Non-exon 51 Amenable Participants)
n=7 participants at risk
Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
61.5%
16/26 • Baseline up to 100 weeks
|
28.6%
2/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
42.3%
11/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Ear infection
|
30.8%
8/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Influenza
|
19.2%
5/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Otitis media
|
7.7%
2/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Pharyngitis streptococcal
|
11.5%
3/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Rhinitis
|
11.5%
3/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Conjunctivitis
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Gastroenteritis viral
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Staphylococcal infection
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Infections and infestations
Impetigo
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
88.5%
23/26 • Baseline up to 100 weeks
|
42.9%
3/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
50.0%
13/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
38.5%
10/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
19.2%
5/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Vomiting
|
42.3%
11/26 • Baseline up to 100 weeks
|
28.6%
2/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
26.9%
7/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
19.2%
5/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Abdominal pain upper
|
19.2%
5/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Constipation
|
11.5%
3/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Nausea
|
7.7%
2/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Gastrointestinal disorders
Dental caries
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
34.6%
9/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Contusion
|
19.2%
5/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Procedural pain
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Scratch
|
11.5%
3/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Head injury
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Sunburn
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
30.8%
8/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
23.1%
6/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
13/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
11.5%
3/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Skin and subcutaneous tissue disorders
Keloid scar
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
General disorders
Pyrexia
|
42.3%
11/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
General disorders
Catheter site pain
|
11.5%
3/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
General disorders
Catheter site bruise
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Nervous system disorders
Headache
|
38.5%
10/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Nervous system disorders
Migraine
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Ear and labyrinth disorders
Ear pain
|
26.9%
7/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Investigations
Cardiac murmur
|
15.4%
4/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Psychiatric disorders
Aggression
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Renal and urinary disorders
Chromaturia
|
11.5%
3/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
7.7%
2/26 • Baseline up to 100 weeks
|
0.00%
0/7 • Baseline up to 100 weeks
|
|
Surgical and medical procedures
Adenoidectomy
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Surgical and medical procedures
Myringotomy
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
|
Surgical and medical procedures
Orchidopexy
|
0.00%
0/26 • Baseline up to 100 weeks
|
14.3%
1/7 • Baseline up to 100 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The most restrictive relevant agreement provides that the PI can only publish the study results with the approval of Sponsor.
- Publication restrictions are in place
Restriction type: OTHER