Trial Outcomes & Findings for Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (NCT NCT02606136)
NCT ID: NCT02606136
Last Updated: 2024-08-27
Results Overview
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted FVC is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Analysis was done using a random coefficient model (RCM), which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
Baseline, Week 104
Results posted on
2024-08-27
Participant Flow
All participants who completed the main portion of the study for a minimum of 104 weeks (2 years) were rolled over to an open-label extension (OLE) for up to an additional 208 weeks (4 years). Some participants remained in the main study for up to 206 weeks before rolling over to the OLE.
Participant milestones
| Measure |
Pamrevlumab
Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
|
|---|---|
|
Main Study (104 Weeks)
STARTED
|
21
|
|
Main Study (104 Weeks)
Received at Least 1 Dose of Study Drug
|
21
|
|
Main Study (104 Weeks)
COMPLETED
|
15
|
|
Main Study (104 Weeks)
NOT COMPLETED
|
6
|
|
OLE (Up to 208 Weeks)
STARTED
|
15
|
|
OLE (Up to 208 Weeks)
Received at Least 1 Dose of Study Drug
|
15
|
|
OLE (Up to 208 Weeks)
COMPLETED
|
0
|
|
OLE (Up to 208 Weeks)
NOT COMPLETED
|
15
|
Reasons for withdrawal
| Measure |
Pamrevlumab
Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
|
|---|---|
|
Main Study (104 Weeks)
Participant/Legal Guardian Decision
|
5
|
|
Main Study (104 Weeks)
Withdrawal by Subject
|
1
|
|
OLE (Up to 208 Weeks)
Participant/Legal Guardian Decision
|
1
|
|
OLE (Up to 208 Weeks)
Sponsor Decision to Terminate Study
|
14
|
Baseline Characteristics
'Number analyzed' signifies participants evaluable for this parameter.
Baseline characteristics by cohort
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
|
|---|---|
|
Age, Continuous
|
15.99 years
STANDARD_DEVIATION 3.277 • n=21 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
20 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=21 Participants
|
|
Percent Predicted Forced Vital Capacity (ppFVC)
|
54.15 percentage of predicted FVC
n=21 Participants
|
|
Percent Predicted Peak Expiratory Flow (PEF)
|
54.66 percentage of predicted PEF
n=21 Participants
|
|
Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1)
|
53.815 percentage of predicted FEV1
n=21 Participants
|
|
Left Ventricular Ejection Fraction Percentage (LVEF%)
|
56.992 percentage of LVEF
n=21 Participants
|
|
Performance of Upper Limb (PUL) Total Score
|
24.4 unit on a scale
n=21 Participants
|
|
Pinch Strength, as Measured by Hand Held Myometry (HHM)
Dominant Pinch Best Result
|
17.003 newton
n=21 Participants
|
|
Pinch Strength, as Measured by Hand Held Myometry (HHM)
Non-Dominant Pinch Best Result
|
16.607 newton
n=21 Participants
|
|
Grip Strength by Hand, as Measured by HHM
Dominant Grip Best Result
|
45.861 newton
n=21 Participants
|
|
Grip Strength by Hand, as Measured by HHM
Non-Dominant Grip Best Result
|
41.977 newton
n=21 Participants
|
|
Cardiac Fibrosis Score (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI)
|
24.100 grams
n=20 Participants • 'Number analyzed' signifies participants evaluable for this parameter.
|
|
Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score
|
8.01 unit on a scale
n=12 Participants • 'Number analyzed' signifies participants evaluable for this parameter.
|
|
Fat Fraction Percentage (%F), as Measured by MRI
|
22.07 percentage of fat
n=9 Participants • 'Number analyzed' signifies participants evaluable for this parameter.
|
PRIMARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted FVC is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Analysis was done using a random coefficient model (RCM), which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104
|
-4.17 percentage of predicted FVC
Standard Error 0.655
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Predicted FEV1 is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FEV1= (observed value)/(predicted value) \* 100%. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104
|
-8.32 percentage of predicted FEV1
Standard Error 1.217
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
Percent predicted PEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort-dependent measure of lung function. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104
|
-7.13 percentage of predicted PEF
Standard Error 2.313
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
LVEF% is an important measure of cardiac function. LVEF is a fraction of blood (in percent) pumped out of the left ventricle of the heart (the main pumping chamber). Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104
|
-2.73 percentage of LVEF
Standard Error 1.651
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items: maximum score = 12), elbow (middle, 9 items: maximum score = 17) and wrist/hand (distal, 7 items: maximum score = 13). Higher scores of each item indicate higher level of function. Total score was calculated by adding the 3 level scores, with a maximum global score of 42 (total score range = 0-42; with higher score indicating better outcome). Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104
|
-4.14 unit on a scale
Standard Error 0.651
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104
Dominant Grip Best Result
|
-2.52 newton
Standard Error 3.610
|
|
Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104
Non-Dominant Grip Best Result
|
-1.31 newton
Standard Error 3.591
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study.
The HHM was used to measure distal upper arm strength (pinch strength). Data has been presented by dominant and non-dominant hand. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=21 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Pinch Strength, as Measured by HHM at Week 104
Dominant Pinch Best Result
|
-4.24 newton
Standard Error 1.547
|
|
Change From Baseline in Pinch Strength, as Measured by HHM at Week 104
Non-Dominant Pinch Best Result
|
-3.46 newton
Standard Error 1.378
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Cardiac MRI was used to assess the cardiac fibrosis by detecting the presence of late gadolinium enhancement (LGE), a marker for myocardial fibrosis. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=20 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104
|
3.74 grams
Standard Error 4.475
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
T2-mapping with MRI was conducted to measure upper arm muscle fibrosis and fat in the biceps brachii muscle. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. The visual score for muscle fat and fibrosis will be assessed using a modified 5-point Mercuri score in which 0 = normal muscle appearance and 5 = complete replacement of muscle by connective tissue and fat, where a lower score indicated visually healthier muscle. Change from baseline was calculated as the score at Week 104 - the score at baseline.
Outcome measures
| Measure |
Pamrevlumab
n=12 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104
|
-2.22 unit on scale
Standard Error 1.088
|
SECONDARY outcome
Timeframe: Baseline, Week 104Population: ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.
Outcome measures
| Measure |
Pamrevlumab
n=9 Participants
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
|---|---|
|
Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104
|
4.49 percentage of fat
Standard Error 2.030
|
Adverse Events
Main Study: Pamrevlumab
Serious events: 6 serious events
Other events: 21 other events
Deaths: 0 deaths
OLE: Pamrevlumab
Serious events: 5 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Main Study: Pamrevlumab
n=21 participants at risk
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
OLE: Pamrevlumab
n=15 participants at risk
Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
|
|---|---|---|
|
Gastrointestinal disorders
Food poisoning
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Adverse drug reaction
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Pneumonia
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Concussion
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Skull fracture
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Renal and urinary disorders
Hydronephrosis
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Renal and urinary disorders
Nephrolithiasis
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Vascular disorders
Hypotension
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Eye disorders
Retinal exudates
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
COVID-19
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Catheter site abscess
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Pilonidal disease
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
Other adverse events
| Measure |
Main Study: Pamrevlumab
n=21 participants at risk
Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.
|
OLE: Pamrevlumab
n=15 participants at risk
Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Dyspepsia
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Constipation
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Pyrexia
|
38.1%
8/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Chills
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Fatigue
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Vaccination site pain
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Oedema peripheral
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Pain
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
General disorders
Medical device site rash
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Immune system disorders
Hypersensitivity
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Nasopharyngitis
|
52.4%
11/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Upper respiratory tract infection
|
23.8%
5/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Sinusitis
|
14.3%
3/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Influenza
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Pneumonia
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Cardiac disorders
Palpitations
|
14.3%
3/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Ear and labyrinth disorders
Ear pain
|
14.3%
3/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Eye disorders
Cataract
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Vomiting
|
47.6%
10/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Nausea
|
33.3%
7/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
23.8%
5/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
23.8%
5/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Muscle strain
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Bone contusion
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Contusion
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Fall
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Sunburn
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Investigations
Cystatin C increased
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Investigations
Weight decreased
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Investigations
Bone density decreased
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
38.1%
8/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
23.8%
5/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Nervous system disorders
Headache
|
66.7%
14/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
26.7%
4/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Nervous system disorders
Dizziness
|
14.3%
3/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Nervous system disorders
Migraine
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Nervous system disorders
Sinus headache
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Nervous system disorders
Fine motor skill dysfunction
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Psychiatric disorders
Anxiety
|
14.3%
3/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Psychiatric disorders
Depression
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Renal and urinary disorders
Nephrolithiasis
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
42.9%
9/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
26.7%
4/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
28.6%
6/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
19.0%
4/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
26.7%
4/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
19.0%
4/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
14.3%
3/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Skin discolouration
|
9.5%
2/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
0.00%
0/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
4.8%
1/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Cardiac disorders
Cardiac dysfunction
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Cardiac disorders
Cardiomyopathy
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Ear and labyrinth disorders
Vertigo positional
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Eye disorders
Chalazion
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Hyperaesthesia teeth
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Melaena
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Oral pain
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Gastrointestinal disorders
Haemorrhoidal haemorrhage
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Anal abscess
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
COVID-19
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
26.7%
4/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Ear infection
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Fungal infection
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Fungal skin infection
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Infected cyst
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Pilonidal disease
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Infections and infestations
Skin candida
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Accident
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Investigations
Ejection fraction decreased
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Investigations
SARS-CoV-2 test positive
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
26.7%
4/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Dactylitis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Joint contracture
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Neuromuscular scoliosis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
26.7%
4/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Temporomandibular joint syndrome
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary congestion
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Restrictive pulmonary disease
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
20.0%
3/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Dermal cyst
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
6.7%
1/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
0.00%
0/21 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
13.3%
2/15 • Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The multisite consortium can publish any time after the data is collected and analyzed by FibroGen. The investigator can only publish after the multisite consortium publishes (or tries to publish and fails). FibroGen has 60 days to review a publication and can extend the embargo up to an additional 120 days (or 180 total).
- Publication restrictions are in place
Restriction type: OTHER