Trial · NCT02255552
Study of Eteplirsen in DMD Patients
NCT02255552, Phase 3, Completed, Sarepta Therapeutics, Inc.; results first posted 2020-07-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Eteplirsen in DMD Patients. Trials, record NCT02255552. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02255552
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Eteplirsen in DMD Patients
- NCT number
- NCT02255552
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 109 participants, actual
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- eteplirsen
- First posted
- 2014-10-02
- Last updated
- 2021-01-25
- Results first posted
- 2020-07-01
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 | Baseline, 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. |
| Secondary | Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96 | Baseline, 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. |
| Secondary | Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | 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. |
| Secondary | Number of Participants Who Lost Ambulation (LOA) by Week 96 | Up to 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. |
| Secondary | Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 | Baseline, Week 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%. |
| Secondary | Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96 | Baseline, 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. |
| Secondary | Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96 | Baseline, Week 96 | Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 | Baseline, Week 96 | 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. | meters | MEAN |
| SECONDARY | Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96 | Baseline, Week 96 | 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. | Percent Normal Dystrophin Protein Level | MEAN |
| SECONDARY | Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | Week 96 | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Lost Ambulation (LOA) by Week 96 | Up to Week 96 | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 | Baseline, Week 96 | 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. | Percentage of predicted FVC | MEAN |
| SECONDARY | Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96 | Baseline, Week 96 | 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. | Unit on scale | MEAN |
| SECONDARY | Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96 | Baseline, Week 96 | 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. | Percent dystrophin positive fibers | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 | Eteplirsen 30 mg/kg | -117.91 | Standard Deviation 128.488 | meters | |
| Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 | Untreated Control Group (Non-exon 51 Amenable Participants) | -133.56 | Standard Deviation 129.333 | meters | |
| Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96 | Eteplirsen 30 mg/kg | 0.516 | Standard Deviation 0.7236 | Percent Normal Dystrophin Protein Level | |
| Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | Eteplirsen 30 mg/kg | 33 | Participants | ||
| Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | Untreated Control Group (Non-exon 51 Amenable Participants) | 3 | Participants | ||
| Number of Participants Who Lost Ambulation (LOA) by Week 96 | Eteplirsen 30 mg/kg | 12 | Participants | ||
| Number of Participants Who Lost Ambulation (LOA) by Week 96 | Untreated Control Group (Non-exon 51 Amenable Participants) | 1 | Participants | ||
| Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 | Eteplirsen 30 mg/kg | -3.413 | Standard Deviation 12.4011 | Percentage of predicted FVC | |
| Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 | Untreated Control Group (Non-exon 51 Amenable Participants) | -2.461 | Standard Deviation 9.6000 | Percentage of predicted FVC | |
| Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96 | Eteplirsen 30 mg/kg | -7.23 | Standard Deviation 5.173 | Unit on scale |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Eteplirsen 30 mg/kg | 79 | |
| Overall Study | STARTED | Untreated Control Group (Non-exon 51 Amenable Participants) | 30 | |
| Overall Study | COMPLETED | Eteplirsen 30 mg/kg | 78 | |
| Overall Study | COMPLETED | Untreated Control Group (Non-exon 51 Amenable Participants) | 15 | |
| Overall Study | NOT COMPLETED | Eteplirsen 30 mg/kg | 1 | |
| Overall Study | NOT COMPLETED | Untreated Control Group (Non-exon 51 Amenable Participants) | 15 |
Source ClinicalTrials.gov
Adverse events158
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Flushing | Vascular disorders | Eteplirsen 30 mg/kg | 4 | 79 | |
| other | Flushing | Vascular disorders | Untreated Control Group (Non-exon 51 Amenable Participants) | 0 | 30 | |
| other | Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Eteplirsen 30 mg/kg | 5 | 79 | |
| other | Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Untreated Control Group (Non-exon 51 Amenable Participants) | 1 | 30 | |
| other | Seasonal allergy | Immune system disorders | Eteplirsen 30 mg/kg | 14 | 79 | |
| other | Seasonal allergy | Immune system disorders | Untreated Control Group (Non-exon 51 Amenable Participants) | 0 | 30 | |
| other | Pyrexia | General disorders | Eteplirsen 30 mg/kg | 20 | 79 | |
| other | Pyrexia | General disorders | Untreated Control Group (Non-exon 51 Amenable Participants) | 1 | 30 | |
| other | Non-cardiac chest pain | General disorders | Eteplirsen 30 mg/kg | 9 | 79 | |
| other | Non-cardiac chest pain | General disorders | Untreated Control Group (Non-exon 51 Amenable Participants) | 0 | 30 |
1 to 10 of 158Source ClinicalTrials.gov
See all 158 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eteplirsen 30 mg/kg | 0 | 79 |
| Total, all-cause mortality | Untreated Control Group (Non-exon 51 Amenable Participants) | 0 | 30 |
| Total, other adverse events | Eteplirsen 30 mg/kg | 78 | 79 |
| Total, other adverse events | Untreated Control Group (Non-exon 51 Amenable Participants) | 24 | 30 |
| Total, serious adverse events | Eteplirsen 30 mg/kg | 11 | 79 |
| Total, serious adverse events | Untreated Control Group (Non-exon 51 Amenable Participants) | 2 | 30 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 31237898 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
Eteplirsen 30 mg/kg vs Untreated Control Group (Non-exon 51 Amenable Participants): Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 | Trial comparisons |
| Eteplirsen 30 mg/kg vs Untreated Control Group (Non-exon 51 Amenable Participants): Number of Participants Who Lost Ambulation (LOA) by Week 96 | Trial comparisons |
| Eteplirsen 30 mg/kg vs Untreated Control Group (Non-exon 51 Amenable Participants): deaths from any cause | Trial comparisons |
| Eteplirsen 30 mg/kg vs Untreated Control Group (Non-exon 51 Amenable Participants): serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02255552
GET https://conductscience.org/api/trials/v1/NCT02255552/events?page=2