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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-000321-80 | EudraCT Number |
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Sponsor Decision
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This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
The study will include a screening period, main study period, open-label extension (OLE) period, and follow-up period 4 weeks after the last dose. All participants who complete the main portion of the study for a minimum of 104 weeks (2 years) will be rolled over to an OLE for up to an additional 208 weeks (4 years).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pamrevlumab | Experimental | Participants will receive pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks. Participants who complete the main study, will continue to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pamrevlumab | Drug | Pamrevlumab, 10 milligrams (mg)/milliliter (mL), single dose vials |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104 | 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. | Baseline, Week 104 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104 | 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. |
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Inclusion Criteria:
Written consent/assent by participant and/or legal guardian as per regional and/or institutional review board (IRB) requirements
Non-ambulatory
Brooke Score for Arms and Shoulders ≤5
Diagnosis of DMD by medical history and confirmed Duchenne mutation in available genetic testing using a validated genetic test
Able to perform spirometry
Able to undergo cardiac and extremity (upper arm) MRI
Percent predicted FVC between 40 and 90, inclusive
At least one historical ppFVC predicted value within 18 months of baseline
Left ventricular ejection fraction ≥ 45% as determined by cardiac MRI at screening or within 3 months prior to Day 0
Participants currently receiving heart failure cardiac medications (for example, angiotensin converting enzyme inhibitors, angiotensin-receptor blockers, and beta-blockers) must achieve a stable regimen for at least 3 months prior to screening
On a stable dose of corticosteroids for a minimum of 6 months prior to screening with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening and no foreseen change in corticosteroid use during the course of study participation
Received pneumococcal vaccine and is receiving annual influenza vaccinations
Adequate renal function: cystatin C ≤1.4 mg/liter (L)
Adequate hematological function
Adequate hepatic function
If sexually active, will use medically accepted contraceptives during participation in the study and for 3 months after the last dose of study drug
Exclusion Criteria:
Requires ≥16 hours continuous ventilation
Prior or ongoing medical condition that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of 156 weeks of treatment and follow-up would be completed, or could impair the assessment of study results
Anticipated spine surgery within 156 weeks
Severe uncontrolled heart disease, including any of the following:
Arrhythmia requiring anti-arrhythmic therapy
Hospitalization due to respiratory failure in the last 6 weeks
Poorly controlled asthma or underlying lung disease such as bronchopulmonary dysplasia
Known or suspected active hepatitis B or C or history of human immunodeficiency virus (HIV)
Body mass index (BMI) ≥40 kilograms (kg)/square meter (m^2) or weight >117 kg
Exposure to another investigational drug or another approved product for DMD (for example, eteplirsen or golodirsen) within 28 days prior to start of study treatment
Exposure to another investigational drug or another approved product for DMD (e.g. eteplirsen) within 28 days prior to start of study treatment (or 5 half-lives of the product whichever is longer) prior to first screening visit with the exception of deflazacort. Use of deflazacort, if regarded by the principal investigator as standard of care, is allowed.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| David Geffen School of Medicine at UCLA | Los Angeles | California | 90095 | United States | ||
| University of California San Francisco - Benioff Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35204752 | Derived | Rayego-Mateos S, Morgado-Pascual JL, Lavoz C, Rodrigues-Diez RR, Marquez-Exposito L, Tejera-Munoz A, Tejedor-Santamaria L, Rubio-Soto I, Marchant V, Ruiz-Ortega M. CCN2 Binds to Tubular Epithelial Cells in the Kidney. Biomolecules. 2022 Feb 3;12(2):252. doi: 10.3390/biom12020252. |
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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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Main Study (104 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 18, 2020 | Jul 31, 2024 |
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| Baseline, Week 104 |
| Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Pinch Strength, as Measured by HHM at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104 | 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. | Baseline, Week 104 |
| Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104 | 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. | Baseline, Week 104 |
| San Francisco |
| California |
| 94143 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Rare Disease Research | Atlanta | Georgia | 30318 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Washington University in St. Louis School of Medicine | St Louis | Missouri | 63110 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Shriner's Hospital for Children - Portland | Portland | Oregon | 97239 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Medical Center Ambulatory Care Pavilion | Dallas | Texas | 75207 | United States |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| OLE (Up to 208 Weeks) |
|
|
Intent-to-treat (ITT) population included all participants who enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Pamrevlumab | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Percent Predicted Forced Vital Capacity (ppFVC) | 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%. | Mean | Full Range | percentage of predicted FVC |
| ||||||||||||||||
| Percent Predicted Peak Expiratory Flow (PEF) | 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. | Mean | Full Range | percentage of predicted PEF |
| ||||||||||||||||
| Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) | 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%. | Mean | Full Range | percentage of predicted FEV1 |
| ||||||||||||||||
| Left Ventricular Ejection Fraction Percentage (LVEF%) | 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). | Mean | Full Range | percentage of LVEF |
| ||||||||||||||||
| Performance of Upper Limb (PUL) Total Score | 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). | Mean | Full Range | unit on a scale |
| ||||||||||||||||
| Pinch Strength, as Measured by Hand Held Myometry (HHM) | The HHM was used to measure distal upper arm strength (pinch strength). Data has been presented by dominant and non-dominant hand. | Mean | Full Range | newton |
| ||||||||||||||||
| Grip Strength by Hand, as Measured by HHM | The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. | Mean | Full Range | newton |
| ||||||||||||||||
| Cardiac Fibrosis Score (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) | 'Number analyzed' signifies participants evaluable for this parameter. | Mean | Full Range | grams |
| ||||||||||||||||
| Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score | T2-mapping with MRI was conducted to measure upper arm muscle fibrosis and fat in the biceps brachii muscle. | 'Number analyzed' signifies participants evaluable for this parameter. | Mean | Full Range | unit on a scale |
| |||||||||||||||
| Fat Fraction Percentage (%F), as Measured by MRI | 'Number analyzed' signifies participants evaluable for this parameter. | Mean | Full Range | percentage of fat |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | percentage of predicted FVC | Baseline, Week 104 |
|
|
| |||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | percentage of predicted FEV1 | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | percentage of predicted PEF | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | percentage of LVEF | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | unit on a scale | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | newton | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pinch Strength, as Measured by HHM at Week 104 | 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. | ITT population included all participants who enrolled in the study. | Posted | Least Squares Mean | Standard Error | newton | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104 | 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. | ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | grams | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104 | 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. | ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | unit on scale | Baseline, Week 104 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104 | 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. | ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | percentage of fat | Baseline, Week 104 |
|
|
Baseline up to Week 210
Safety population included participants who received at least one dose of study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Main Study: Pamrevlumab | Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks. | 0 | 21 | 6 | 21 | 21 | 21 |
| EG001 | OLE: Pamrevlumab | 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. | 0 | 15 | 5 | 15 | 15 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Food poisoning | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Adverse drug reaction | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Concussion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Skull fracture | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Retinal exudates | Eye disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Catheter site abscess | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pilonidal disease | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Medical device site rash | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Bone contusion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Cystatin C increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Bone density decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Sinus headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Fine motor skill dysfunction | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cardiac dysfunction | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Vertigo positional | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Chalazion | Eye disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hyperaesthesia teeth | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Infected cyst | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pilonidal disease | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Skin candida | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Accident | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Ejection fraction decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dactylitis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Joint contracture | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Neuromuscular scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Temporomandibular joint syndrome | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Restrictive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
|
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).
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Information Desk | FibroGen, Inc. | 415-978-1441 | FG3019-079DMDStudy@fibrogen.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 12, 2023 | Jul 31, 2024 | SAP_003.pdf |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C560078 | pamrevlumab |
Not provided
Not provided
Not provided
|
| Unknown or Not Reported |
|
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Non-Dominant Pinch Best Result |
|
|
| Non-Dominant Grip Best Result |
|
|
|
|
|
|
|
|
|
|
|