Trial Outcomes & Findings for Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) (NCT NCT04060199)
NCT ID: NCT04060199
Last Updated: 2024-12-11
Results Overview
The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients. TTSTAND was assessed as the time it takes the participant to go from lying flat on the floor to standing. The time measured for TTSTAND was converted to a velocity expressed as rise per second.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
77 participants
Primary outcome timeframe
baseline, Week 13, 25, 37, 49
Results posted on
2024-12-11
Participant Flow
Participants were enrolled in the study from April 14, 2020 to October 19, 2023 at 30 sites in 17 countries, predominantly in Europe, Asia, North America, and South America
Eighty-three patients were screened. Six patients were screen failed due to failure to satisfy inclusion/exclusion criteria. Analysis Populations for all randomized patients was 77 patients.
Participant milestones
| Measure |
Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Viltolarsen: IV infusion
|
Placebo
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Placebo: IV infusion
|
|---|---|---|
|
Overall Study
STARTED
|
38
|
39
|
|
Overall Study
COMPLETED
|
36
|
38
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Baseline characteristics by cohort
| Measure |
Viltolarsen
n=38 Participants
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Viltolarsen: IV infusion
|
Placebo
n=39 Participants
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Placebo: IV infusion
|
Total
n=77 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
5.5 years
STANDARD_DEVIATION 1.20 • n=5 Participants
|
5.7 years
STANDARD_DEVIATION 1.16 • n=7 Participants
|
5.6 years
STANDARD_DEVIATION 1.18 • 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
|
38 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
77 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
36 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
69 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 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
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
21 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
27 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
53 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
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Weight
|
21.12 kg
STANDARD_DEVIATION 4.354 • n=5 Participants
|
20.70 kg
STANDARD_DEVIATION 4.277 • n=7 Participants
|
20.91 kg
STANDARD_DEVIATION 4.292 • n=5 Participants
|
|
Height
|
110.9 cm
STANDARD_DEVIATION 9.78 • n=5 Participants
|
111.8 cm
STANDARD_DEVIATION 7.78 • n=7 Participants
|
111.4 cm
STANDARD_DEVIATION 8.78 • n=5 Participants
|
|
Body Mass Index (BMI)
|
17.053 kg/m^2
STANDARD_DEVIATION 1.9427 • n=5 Participants
|
16.439 kg/m^2
STANDARD_DEVIATION 2.1105 • n=7 Participants
|
16.742 kg/m^2
STANDARD_DEVIATION 2.0396 • n=5 Participants
|
PRIMARY outcome
Timeframe: baseline, Week 13, 25, 37, 49Population: The modified ITT (mITT) Population was set as the analysis population. The mITT Population was defined as all randomized patients who received at least 1 dose of IP and had a baseline assessment and at least 1 post baseline efficacy assessment. The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients using a mixed-effect model for repeated measures (MMRM) analysis.
The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients. TTSTAND was assessed as the time it takes the participant to go from lying flat on the floor to standing. The time measured for TTSTAND was converted to a velocity expressed as rise per second.
Outcome measures
| Measure |
Viltolarsen
n=37 Participants
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Viltolarsen: IV infusion
|
Placebo
n=38 Participants
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Placebo: IV infusion
|
|---|---|---|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity
Week 13
|
0.026 rise/s
Standard Error 0.0103
|
0.013 rise/s
Standard Error 0.0104
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity
Week 25
|
0.027 rise/s
Standard Error 0.0100
|
0.014 rise/s
Standard Error 0.0100
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity
Week 37
|
0.027 rise/s
Standard Error 0.0107
|
0.027 rise/s
Standard Error 0.0108
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity
Week 49
|
0.009 rise/s
Standard Error 0.0096
|
0.013 rise/s
Standard Error 0.0096
|
Adverse Events
Viltolarsen
Serious events: 2 serious events
Other events: 33 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 37 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Viltolarsen
n=38 participants at risk
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Viltolarsen: IV infusion
|
Placebo
n=39 participants at risk
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Placebo: IV infusion
|
|---|---|---|
|
Infections and infestations
Influenza
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Pharyngitis
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
0.00%
0/39 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Adverse drug reaction
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
0.00%
0/39 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Nervous system disorders
Epilepsy
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
Other adverse events
| Measure |
Viltolarsen
n=38 participants at risk
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Viltolarsen: IV infusion
|
Placebo
n=39 participants at risk
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Placebo: IV infusion
|
|---|---|---|
|
Infections and infestations
COVID-19
|
31.6%
12/38 • Number of events 13 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
30.8%
12/39 • Number of events 13 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Upper respiratory tract infection
|
26.3%
10/38 • Number of events 18 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
23.1%
9/39 • Number of events 12 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Nasopharyngitis
|
23.7%
9/38 • Number of events 25 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
17.9%
7/39 • Number of events 16 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Rhinitis
|
15.8%
6/38 • Number of events 10 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
7.7%
3/39 • Number of events 5 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Gastroenteritis
|
7.9%
3/38 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
10.3%
4/39 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Influenza
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
10.3%
4/39 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Urinary tract infection
|
5.3%
2/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Bronchitis
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Hordeolum
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
7.7%
3/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Pharyngitis
|
5.3%
2/38 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
0.00%
0/39 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Pneumonia
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Varicella
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Pyrexia
|
26.3%
10/38 • Number of events 12 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
33.3%
13/39 • Number of events 19 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Fatigue
|
7.9%
3/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Influenza-like illness
|
5.3%
2/38 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Administration site extravasation
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Diarrhea
|
21.1%
8/38 • Number of events 8 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
17.9%
7/39 • Number of events 13 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Vomiting
|
10.5%
4/38 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
28.2%
11/39 • Number of events 12 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Abdominal pain
|
10.5%
4/38 • Number of events 10 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
15.4%
6/39 • Number of events 11 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Nausea
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
7.7%
3/39 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.3%
2/38 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Constipation
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Feces soft
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
31.6%
12/38 • Number of events 14 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
12.8%
5/39 • Number of events 7 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
15.8%
6/38 • Number of events 7 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
7.9%
3/38 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Blood triglycerides increased
|
7.9%
3/38 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Protein urine present
|
5.3%
2/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
7.7%
3/39 • Number of events 5 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Blood cholesterol increased
|
2.6%
1/38 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Urine protein:creatinine ratio increased
|
5.3%
2/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Beta 2 microglobulin urine increased
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Crystal urine present
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Injury, poisoning and procedural complications
Fall
|
13.2%
5/38 • Number of events 6 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
10.3%
4/39 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Injury, poisoning and procedural complications
Contusion
|
5.3%
2/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Renal and urinary disorders
Hematuria
|
5.3%
2/38 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Renal and urinary disorders
Urine abnormality
|
7.9%
3/38 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
0.00%
0/39 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Renal and urinary disorders
Pyelocaliectasis
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
7.7%
3/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Skin and subcutaneous tissue disorders
Eczema
|
7.9%
3/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
0.00%
0/39 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
5.3%
2/38 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
0.00%
0/39 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.9%
3/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Nervous system disorders
Headache
|
7.9%
3/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
12.8%
5/39 • Number of events 8 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Cardiac disorders
Tachycardia
|
7.9%
3/38 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Metabolism and nutrition disorders
Hypercholesterolemia
|
2.6%
1/38 • Number of events 1 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Ear and labyrinth disorders
Ear pain
|
7.9%
3/38 • Number of events 4 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
2.6%
1/39 • Number of events 3 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
|
Vascular disorders
Hematoma
|
0.00%
0/38 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
5.1%
2/39 • Number of events 2 • Up to 48 weeks
Incidence of Adverse Events as assessed by CTCAE v4.03
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The most restrictive relevant agreement on the PI provides that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is not less than 30 days from the time submitted to the sponsor for review.
- Publication restrictions are in place
Restriction type: OTHER