Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2020-000698-26 | EudraCT Number |
Not provided
Not provided
Study did not meet its primary endpoint.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
This is a global, Phase 3, randomized, double-blind trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy, aged 12 years and older. Approximately 90 male participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic corticosteroid) or Arm B (placebo+ systemic corticosteroid), respectively.
Participants must be fully informed of the potential benefits of approved products and make an informed decision that they prefer to participate in a clinical trial in which they could be randomized to placebo.
This trial has 3 study periods:
In the screening period, participants will be evaluated per the protocol inclusion/exclusion criteria to determine eligibility for participation in this trial.
During the treatment period, each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks.
Participants who complete the 52-week study (either arm) may be eligible for rollover into an open-label extension treatment (OLE) with pamrevlumab + systemic corticosteroids.
Participants who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pamrevlumab | Experimental | Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks |
|
| Placebo | Placebo Comparator | Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pamrevlumab | Drug | Pamrevlumab per dose and schedule specified in the arm description |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items, 12 points), elbow (middle, 9 items, 17 points) and wrist/hand (distal, 7 items, 13 points). Higher scores indicate higher level of function. Total score ranges from 0-42 points and is the sum of the scores for the 3 subscales. Analysis was done using a random coefficient model (RCM), which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline ordinal PUL entry score as covariate. | Baseline, Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry | 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 an RCM, which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline value as covariate. |
Not provided
Inclusion Criteria:
Males at least 12 years of age, non-ambulatory at screening initiation
Written consent by participant and/or legal guardian as per regional/ country and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
Male participants with partners of childbearing potential must use contraception during the conduct of the study, and for 12 weeks after the last dose of study drug.
Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test
Brooke Score for Arms and Shoulders ≤5
Able to undergo MRI test for the upper arm extremities (Biceps Brachii muscle) and cardiac muscle
Able to perform spirometry
Average (of Screening and Day 0) percent predicted forced vital capacity (FVC) between 45 and 85, inclusive
Left ventricular ejection fraction ≥50% as determined by local cardiac MRI read at screening or within 3 months prior to randomization (Day 0)
If participants have a history of cardiomyopathy, then participant must be on a stable dose of cardiomyopathy/ heart failure medications (for example, angiotensin converting enzyme inhibitors, aldosterone receptors blockers, angiotensin-receptor blockers, and betablockers) for at least 1 month prior to screening. If participants have no diagnosis of cardiomyopathy, then no dose of cardiomyopathy/heart failure medication is required for eligibility.
On a stable dose of systemic corticosteroids for a minimum of 6 months, with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening. Corticosteroid dosage should be in compliance with the DMD Care Considerations Working Group recommendations (for example, prednisone or prednisolone 0.75 mg/kg per day or deflazacort 0.9 mg/kg per day) or stable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.
Agreement to receive annual influenza vaccinations during the course of the study.
Adequate renal function: cystatin C ≤1.4 mg/liter (L)
Adequate hematology and electrolytes parameters:
Adequate hepatic function:
Exclusion Criteria:
Previous exposure to pamrevlumab
BMI ≥40 kg/square meter (m^2) or weight >117 kg
History of:
Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (for example, eteplirsen [exondys 51], ataluren, golodirsen [vyondys 53], casimersen [amondys 45]) within 5 half-lives of screening, whichever is longer, with the exception of the systemic corticosteroids, including deflazacort
Severe uncontrolled heart failure (NYHA Classes III-IV), or renal dysfunction, including any of the following:
Arrhythmia requiring anti-arrhythmic therapy
Requires ≥16 hours continuous ventilation
Hospitalization due to respiratory failure within the 8 weeks prior to screening
Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the investigator might impact respiratory function
The Investigator judges that the participant will be unable to fully participate in the study and complete it for any reason, including inability to comply with study procedures and treatment, or any other relevant medical or psychiatric conditions
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's | Little Rock | Arkansas | 72202 | United States | ||
| University of California Los Angeles Medical Center |
Not provided
Not provided
Not provided
Not provided
Not provided
The study included 2 periods: Double-blind (DB) Treatment Period and Open-label Extension (OLE) Period.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Pamrevlumab | Participants received pamrevlumab intravenously (IV) every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| DB Treatment Period (52 Weeks) |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 1, 2022 | Feb 13, 2024 |
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Matching placebo per schedule specified in the arm description |
|
| Corticosteroids | Drug | Systemic deflazacort or equivalent potency of corticosteroids administered orally |
|
| Baseline, Week 52 |
| Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Grip Strength was analyzed using a MMRM with fixed effects for treatment, visit (as a factor), treatment-by-visit interaction, and covariates (baseline values). | Baseline, Week 52 |
| Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI) | 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). The LVEF% was analyzed using an analysis of covariance (ANCOVA) model with treatment and baseline value. | Baseline, Week 52 |
| Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry | The ppPEF 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. The ppFEV1 was analyzed using an RCM including fixed effects of time, treatment, and treatment-by-time interaction, with baseline as covariate. | Baseline, Week 52 |
| Los Angeles |
| California |
| 90045 |
| United States |
| UC Davis Health | Sacramento | California | 95817 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Fairway | Kansas | 66205 | United States |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
| UMASS Med School | Worcester | Massachusetts | 01655 | United States |
| C.S. Mott Children's Hospital | Ann Arbor | Michigan | 48109-4234 | United States |
| Spectrum Health Hospitals Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | United States |
| Washington University School of Medicine in Saint Louis | St Louis | Missouri | 63110 | United States |
| Carolinas HealthCare System Neurosciences Institute-Neurology - Charlotte | Charlotte | North Carolina | 28207 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205-2664 | United States |
| Shriners Hospital for Children | Portland | Oregon | 97239 | United States |
| Penn State Health Children's Hospital | Hershey | Pennsylvania | 17033 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Children's Health Dallas/UTSW | Dallas | Texas | 75207 | United States |
| University of Utah Health | Salt Lake City | Utah | 84108 | United States |
| Children's Specialty Group - Medical Center Office | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Murdoch Children's Research Institute | Parkville | Victoria | 3052 | Australia |
| Klinik Favoriten | Vienna | 1100 | Austria |
| Universitair Ziekenhuis Gent | Ghent | 9000 | Belgium |
| Universitair Ziekenhuis Leuven - Campus Gasthuisberg | Leuven | 3000 | Belgium |
| Centre Hospitalier Régional de la Citadelle | Liège | 4000 | Belgium |
| London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| Children's Hospital of Chongqing Medical University | Chongqing | Chongqing Municipality | 400015 | China |
| West China Second University Hospital, Sichuan University | Chengdu | Sichuan | 610041 | China |
| Peking Union Medical College Hospital, Chinese Academy of Medical Sciences | Beijing | 100730 | China |
| Fakultní Nemocnice Brno - Dětská Nemocnice | Brno | 613 00 | Czechia |
| Klinika dÄ>tské neurologie, Neuromuskulární centrum | Prague | 150 06 | Czechia |
| CHU de Nantes - Hotel Dieu | Nantes | 44093 | France |
| Association Institut de Myologie | Paris | 75012 | France |
| Hopital Hautepierre | Strasbourg | 67098 | France |
| The Chaim Sheba Medical Center | Tel Aviv | 5265601 | Israel |
| The Edith Wolfson Medical Center | Tel Aviv | 5822012 | Israel |
| Istituto di Ricovero e Cura a Carattere Scientifico Eugenio Medea - Lombardia | Lecco | 23842 | Italy |
| IRRCS Ospedale San Raffaele | Milan | 20132 | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli | Rome | 00168 | Italy |
| Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo | Rome | 165 | Italy |
| Radboud Universitair Medisch Centrum | Nijmegen | Gelderland | 6525 | Netherlands |
| Leiden Universitair Medisch Centrum | Leiden | 2333 ZA | Netherlands |
| Hospital General Universitario de Alicante | Alicante | 3010 | Spain |
| Hospital Universitari Vall d'Hebrón | Barcelona | 08035 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| Inselspital Universitätsspital Bern | Bern | 3010 | Switzerland |
| Leeds Teaching Hospitals NHS Trust | Leeds | LS1 3EX | United Kingdom |
| University College London Hospitals NHS Foundation Trust | London | WC1N 3BG | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust | Oxford | OX3 9DU | United Kingdom |
| Placebo |
Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| OLE (Maximum Exposure: 93.4 Weeks) |
|
|
The intent-to-treat (ITT) set included all randomized participants.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Pamrevlumab | Participants received pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks. |
| BG001 | Placebo | Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks. |
| BG002 | Total | Total of all reporting groups |
| 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 |
|
| 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 | Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items, 12 points), elbow (middle, 9 items, 17 points) and wrist/hand (distal, 7 items, 13 points). Higher scores indicate higher level of function. Total score ranges from 0-42 points and is the sum of the scores for the 3 subscales. Analysis was done using a random coefficient model (RCM), which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline ordinal PUL entry score as covariate. | The modified intent-to-treat (mITT) population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline, Week 52 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry | 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 an RCM, which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline value as covariate. | The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | Posted | Least Squares Mean | Standard Error | percentage of predicted FVC | Baseline, Week 52 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Grip Strength was analyzed using a MMRM with fixed effects for treatment, visit (as a factor), treatment-by-visit interaction, and covariates (baseline values). | The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. 'Number analyzed' = participants evaluable for specified category. | Posted | Least Squares Mean | Standard Error | newton | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI) | 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). The LVEF% was analyzed using an analysis of covariance (ANCOVA) model with treatment and baseline value. | The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | Posted | Least Squares Mean | Standard Error | percentage of LVEF | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry | The ppPEF 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. The ppFEV1 was analyzed using an RCM including fixed effects of time, treatment, and treatment-by-time interaction, with baseline as covariate. | The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | Posted | Least Squares Mean | Standard Error | percentage of predicted PEF | Baseline, Week 52 |
|
|
DB treatment period: From first dose of study drug up to 61 weeks OLE period: From first dose of study drug up to study termination by Sponsor and safety follow-up (up to approximately 102 weeks)
The safety analysis set included all participants who received any dose of study medication.
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 | DB Period: Pamrevlumab | Participants received pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. | 1 | 48 | 8 | 48 | 42 | 48 |
| EG001 | DB Period: Placebo | Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. | 0 | 49 | 6 | 49 | 44 | 49 |
| EG002 | OLE Period: Pamrevlumab | After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks. | 0 | 86 | 5 | 86 | 53 | 86 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Myocarditis | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Testicular necrosis | Reproductive system and breast disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Testicular torsion | Reproductive system and breast disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiomyopathy | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Vascular access site bruising | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | 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 | FG3019-093DMDStudy@fibrogen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 23, 2023 | Feb 13, 2024 | SAP_001.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 |
| D000305 | Adrenal Cortex Hormones |
| ID | Term |
|---|---|
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
Not provided
Not provided
| Other than specified |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|