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The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Following primary efficacy endpoints, dosing will continue to week 144 to evaluate the long term effects of eteplirsen.
Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks). Patients in the untreated group will not receive treatment.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.
Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treated Group | Experimental | Approximately 80 patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping will receive 30 mg/kg of eteplirsen weekly for 96 weeks, followed by a safety extension (not to exceed 48 weeks). |
|
| Untreated Group | No Intervention | Approximately 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eteplirsen | Drug | Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 | 6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 96 was reported. | Baseline, Week 96 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96 | Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value. |
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Inclusion Criteria:
Exclusion Criteria:
Other inclusion/exclusion criteria apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neuromuscular Research Center | Phoenix | Arizona | 85028 | United States | ||
| David Geffen School of Medicine at UCLA |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31237898 | Background | Brogna C, Coratti G, Pane M, Ricotti V, Messina S, D'Amico A, Bruno C, Vita G, Berardinelli A, Mazzone E, Magri F, Ricci F, Mongini T, Battini R, Bello L, Pegoraro E, Baranello G, Previtali SC, Politano L, Comi GP, Sansone VA, Donati A, Bertini E, Muntoni F, Goemans N, Mercuri E; on behalf on the International DMD group. Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53. PLoS One. 2019 Jun 25;14(6):e0218683. doi: 10.1371/journal.pone.0218683. eCollection 2019. |
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A total of 109 participants were enrolled in the study. Only 79 participants were treated and the remaining participants were not applicable for treatment as those participants assessed under "Untreated" arm.
The study was conducted at 40 sites in the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eteplirsen 30 mg/kg | Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Jun 2, 2017 | Jun 11, 2020 |
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|
| Baseline, Week 96 |
| Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | NSAA is a clinician-administered scale that rates participant performance on 17-items and included assessments of abilities such as 10-meter walk/run, rising from a sit to stand, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. For all activities, participants were graded as follows: 0 = unable to achieve goal independently; 1 = modified method but achieves goal independent of physical assistance from another and 2 = normal, no obvious modification of activity. Number of participants having ability to rise independently from the floor indicated by a NSAA Rise from floor sub score greater than 0 (unable to achieve goal independently) was reported. | Week 96 |
| Number of Participants Who Lost Ambulation (LOA) by Week 96 | Number of participants who lost ambulation (LOA) by Week 96 was reported. Participant were considered non-ambulatory if each of the 3 conditions below were met: NSAA walk subscore was "0" (unable to achieve goal independently) on 2 consecutive days within a visit or NSAA was not done due to reason related to non-ambulation; 6MWT was not done with any reason related to permanent non-ambulation; and no later data showing this participant was still ambulatory. This was not required if non ambulatory status occurred at the time of early withdrawal or at the end of Week 96 assessment. NSAA is a 17-item scale to assess the participant's abilities; total score range from 0 (if all the activities are failed) to 34 (if all the activities are achieved) with higher scores indicating better performance on the assessment/ fully-independent function. | Up to Week 96 |
| Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 | FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry; and is the most important measurement of lung function. This test requires participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate participant's bronchial (breathing) tubes. Percent of predicted FVC = (observed value) / (predicted value) * 100%. | Baseline, Week 96 |
| Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96 | NSAA is a clinician-administered scale that rates participant performance on 17-items and included assessments of abilities such as 10-meter walk/run, rising from a sit to stand, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participant were graded as follows: 0 = unable to achieve goal independently; 1 = modified method but achieves goal independent of physical assistance from another and 2 = normal, no obvious modification of activity. NSAA total score was derived by summing the scores for all the individual items and range from 0 (if all the activities are failed) to 34 (if all the activities are achieved) with higher scores indicating better performance on the assessment/ fully-independent function. | Baseline, Week 96 |
| Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96 | Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry. | Baseline, Week 96 |
| Los Angeles |
| California |
| 90095 |
| United States |
| University of California, Davis Medical Center | Sacramento | California | 95817 | United States |
| Rady Children's Hospital, U.C. San Diego | San Diego | California | 92130 | United States |
| Stanford University School of Medicine/Medical Center | Stanford | California | 94305 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | United States |
| Children's National Health System | Washington D.C. | District of Columbia | 20010 | United States |
| The University of Florida, Powell Gene Therapy Center | Gainesville | Florida | 32610 | United States |
| NW FL Clinical Research Group, LLC | Gulf Breeze | Florida | 32561 | United States |
| Nemours Children's Hospital | Orlando | Florida | 32827 | United States |
| Rare Disease Research Center | Atlanta | Georgia | 30318 | United States |
| Emory University | Atlanta | Georgia | 30324 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Iowa Children's Hospital | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Dartmouth-Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| University of Rochester Clinical Research Center | Rochester | New York | 14642 | United States |
| Levine Childrens Hospital, Carolinas Medical Center | Charlotte | North Carolina | 28207 | United States |
| Cincinnati Children's Hospital Medical Center (CCHMC) | Cincinnati | Ohio | 45229 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Shriners Hospital for Children | Portland | Oregon | 97239 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Untreated Control Group (Non-exon 51 Amenable Participants) |
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Consisted of all participants who received at least 1 dose of eteplirsen in the eteplirsen-treated group or had at least 1 post-baseline safety assessment in the untreated group.Comparisons between the eteplirsen-treated and the untreated group were not to be performed due to the small number of subjects and differences in the population groups.
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| ID | Title | Description |
|---|---|---|
| BG000 | Eteplirsen 30 mg/kg | Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks. |
| BG001 | Untreated Control Group (Non-exon 51 Amenable Participants) | DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 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 |
| ||||||||||||||||
| Region of Enrollment | 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 6 Minute Walk Test (6MWT) Distance at Week 96 | 6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 96 was reported. | Primary efficacy set: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | meters | Baseline, Week 96 |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96 | Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value. | Analysis Set consisted of a subset of participants who received at least 1 dose of eteplirsen and had both baseline and 1 post-dose muscle biopsy samples evaluable for dystrophin expression at Week 96. Here, "Overall Number of Participants Analyzed" signifies number of participants who were evaluable for this outcome measure. Data for this outcome measure was not planned to be collected and analyzed for the Untreated Control group. | Posted | Mean | Standard Deviation | Percent Normal Dystrophin Protein Level | Baseline, Week 96 |
| ||||||||||||||||||||||||||||||
| Secondary | Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | NSAA is a clinician-administered scale that rates participant performance on 17-items and included assessments of abilities such as 10-meter walk/run, rising from a sit to stand, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. For all activities, participants were graded as follows: 0 = unable to achieve goal independently; 1 = modified method but achieves goal independent of physical assistance from another and 2 = normal, no obvious modification of activity. Number of participants having ability to rise independently from the floor indicated by a NSAA Rise from floor sub score greater than 0 (unable to achieve goal independently) was reported. | Primary efficacy set: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure. | Posted | Count of Participants | Participants | Week 96 |
| |||||||||||||||||||||||||||||||
| Secondary | Number of Participants Who Lost Ambulation (LOA) by Week 96 | Number of participants who lost ambulation (LOA) by Week 96 was reported. Participant were considered non-ambulatory if each of the 3 conditions below were met: NSAA walk subscore was "0" (unable to achieve goal independently) on 2 consecutive days within a visit or NSAA was not done due to reason related to non-ambulation; 6MWT was not done with any reason related to permanent non-ambulation; and no later data showing this participant was still ambulatory. This was not required if non ambulatory status occurred at the time of early withdrawal or at the end of Week 96 assessment. NSAA is a 17-item scale to assess the participant's abilities; total score range from 0 (if all the activities are failed) to 34 (if all the activities are achieved) with higher scores indicating better performance on the assessment/ fully-independent function. | Primary efficacy set consisted of all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. | Posted | Count of Participants | Participants | Up to Week 96 |
| |||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 | FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry; and is the most important measurement of lung function. This test requires participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate participant's bronchial (breathing) tubes. Percent of predicted FVC = (observed value) / (predicted value) * 100%. | Primary efficacy set: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Percentage of predicted FVC | Baseline, Week 96 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96 | NSAA is a clinician-administered scale that rates participant performance on 17-items and included assessments of abilities such as 10-meter walk/run, rising from a sit to stand, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participant were graded as follows: 0 = unable to achieve goal independently; 1 = modified method but achieves goal independent of physical assistance from another and 2 = normal, no obvious modification of activity. NSAA total score was derived by summing the scores for all the individual items and range from 0 (if all the activities are failed) to 34 (if all the activities are achieved) with higher scores indicating better performance on the assessment/ fully-independent function. | Primary efficacy: all participants in the efficacy set (all participants in eteplirsen-treated and untreated control groups who had at least 1 post-baseline functional assessment) who had a Baseline 6MWT distance of 300 to 450 meters, inclusive. Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Unit on scale | Baseline, Week 96 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96 | Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry. | Analysis Set consisted of a subset of participants who received at least 1 dose of eteplirsen and had both baseline and 1 post-dose muscle biopsy samples evaluable for dystrophin expression at Week 96. Here, "Overall Number of Participants Analyzed" signifies number of participants who were evaluable for this outcome measure. Data for this outcome measure was not planned to be collected and analyzed for the Untreated Control group. | Posted | Mean | Standard Deviation | Percent dystrophin positive fibers | Baseline, Week 96 |
|
|
From start of study drug administration to Week 144
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eteplirsen 30 mg/kg | Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping received Eteplirsen as an Intravenous (IV) infusion, at a dose of 30 milligram per kilogram (mg/kg) once weekly for 96 weeks. | 0 | 79 | 11 | 79 | 78 | 79 |
| EG001 | Untreated Control Group (Non-exon 51 Amenable Participants) | DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks. | 0 | 30 | 2 | 30 | 24 | 30 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pneumonitis chemical | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Myocarditis | Cardiac disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Abasia | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Major depression | Psychiatric disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Catheter site infection | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Lymphadenitis viral | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Flushing | Vascular disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (17.1) | Non-systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Infusion site bruising | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Infusion site pain | General disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Torus fracture | Injury, poisoning and procedural complications | MedDRA (17.1) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (17.1) | Non-systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | +1-800-690-2003 | clinicaltrials@sarepta.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 5, 2019 | Jun 11, 2020 | 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 |
|---|---|
| C000611335 | eteplirsen |
Not provided
Not provided
Not provided
| 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 |
|
|
| OG001 |
| Untreated Control Group (Non-exon 51 Amenable Participants) |
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks. |
|
|
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks. |
|
|
|
|
| OG001 |
| Untreated Control Group (Non-exon 51 Amenable Participants) |
DMD participants with mutations amenable to skipping of any exon(s) except exon 51 did not receive any treatment, but completed study assessments up to 96 weeks. |
|
|
|