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Study of Eteplirsen in DMD Patients (PROMOVI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02255552
Recruitment Status : Completed
First Posted : October 2, 2014
Results First Posted : July 1, 2020
Last Update Posted : July 1, 2020
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy (DMD)
Intervention Drug: eteplirsen
Enrollment 109
Recruitment Details The study was conducted at 40 sites in the United States.
Pre-assignment Details 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.
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description 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. 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.
Period Title: Overall Study
Started 79 30
Completed 78 15
Not Completed 1 15
Reason Not Completed
Participants enrolled into another study             0             11
Withdrawal by Subject             1             4
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants) Total
Hide Arm/Group Description 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. 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. Total of all reporting groups
Overall Number of Baseline Participants 79 30 109
Hide Baseline Analysis Population Description
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.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 79 participants 30 participants 109 participants
9.1  (2.04) 8.8  (1.76) 9.0  (1.96)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 79 participants 30 participants 109 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
79
 100.0%
30
 100.0%
109
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 79 participants 30 participants 109 participants
Hispanic or Latino
7
   8.9%
6
  20.0%
13
  11.9%
Not Hispanic or Latino
71
  89.9%
24
  80.0%
95
  87.2%
Unknown or Not Reported
1
   1.3%
0
   0.0%
1
   0.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 79 participants 30 participants 109 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
5
   6.3%
1
   3.3%
6
   5.5%
Native Hawaiian or Other Pacific Islander
2
   2.5%
0
   0.0%
2
   1.8%
Black or African American
2
   2.5%
0
   0.0%
2
   1.8%
White
67
  84.8%
26
  86.7%
93
  85.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
3
   3.8%
3
  10.0%
6
   5.5%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 79 participants 30 participants 109 participants
79
 100.0%
30
 100.0%
109
 100.0%
1.Primary Outcome
Title Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96
Hide Description 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.
Time Frame Baseline, Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 65 9
Mean (Standard Deviation)
Unit of Measure: meters
-117.91  (128.488) -133.56  (129.333)
2.Secondary Outcome
Title Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96
Hide Description Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot. Data for this outcome measure was not planned to be collected and analyzed for Untreated Control group.
Time Frame Baseline, Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Eteplirsen 30 mg/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Normal Dystrophin Protein Level
0.516  (0.7236)
3.Secondary Outcome
Title Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96
Hide Description 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.
Time Frame Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 61 9
Measure Type: Count of Participants
Unit of Measure: Participants
33
  54.1%
3
  33.3%
4.Secondary Outcome
Title Number of Participants Who Lost Ambulation (LOA) by Week 96
Hide Description 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.
Time Frame Up to Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 67 20
Measure Type: Count of Participants
Unit of Measure: Participants
12
  17.9%
1
   5.0%
5.Secondary Outcome
Title Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96
Hide Description 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%.
Time Frame Baseline, Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 66 9
Mean (Standard Deviation)
Unit of Measure: Percentage of predicted FVC
-3.413  (12.4011) -2.461  (9.6000)
6.Secondary Outcome
Title Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96
Hide Description 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.
Time Frame Baseline, Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 61 9
Mean (Standard Deviation)
Unit of Measure: Unit on scale
-7.23  (5.173) -8.44  (9.812)
7.Secondary Outcome
Title Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96
Hide Description Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry.
Time Frame Baseline, Week 96
Outcome Measure Data Not Reported
Time Frame From start of study drug administration to Week 144
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Hide Arm/Group Description 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. 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.
All-Cause Mortality
Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Affected / at Risk (%) Affected / at Risk (%)
Total   0/79 (0.00%)   0/30 (0.00%) 
Hide Serious Adverse Events
Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Affected / at Risk (%) Affected / at Risk (%)
Total   11/79 (13.92%)   2/30 (6.67%) 
Cardiac disorders     
Arrhythmia * 1  1/79 (1.27%)  0/30 (0.00%) 
Myocarditis * 1  1/79 (1.27%)  0/30 (0.00%) 
General disorders     
Gait disturbance * 1  1/79 (1.27%)  0/30 (0.00%) 
Abasia * 1  0/79 (0.00%)  1/30 (3.33%) 
Infections and infestations     
Catheter site infection * 1  1/79 (1.27%)  0/30 (0.00%) 
Lymphadenitis viral * 1  0/79 (0.00%)  1/30 (3.33%) 
Injury, poisoning and procedural complications     
Femoral neck fracture * 1  2/79 (2.53%)  0/30 (0.00%) 
Femur fracture * 1  1/79 (1.27%)  0/30 (0.00%) 
Hip fracture * 1  1/79 (1.27%)  0/30 (0.00%) 
Pneumonitis chemical * 1  1/79 (1.27%)  0/30 (0.00%) 
Nervous system disorders     
Generalised tonic-clonic seizure * 1  1/79 (1.27%)  0/30 (0.00%) 
Loss of consciousness * 1  1/79 (1.27%)  0/30 (0.00%) 
Psychiatric disorders     
Aggression * 1  1/79 (1.27%)  0/30 (0.00%) 
Major depression * 1  1/79 (1.27%)  0/30 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory failure * 1  1/79 (1.27%)  0/30 (0.00%) 
Aspiration * 1  1/79 (1.27%)  0/30 (0.00%) 
Skin and subcutaneous tissue disorders     
Dermatitis contact * 1  1/79 (1.27%)  0/30 (0.00%) 
Urticaria * 1  1/79 (1.27%)  0/30 (0.00%) 
1
Term from vocabulary, MedDRA (17.1)
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eteplirsen 30 mg/kg Untreated Control Group (Non-exon 51 Amenable Participants)
Affected / at Risk (%) Affected / at Risk (%)
Total   78/79 (98.73%)   24/30 (80.00%) 
Ear and labyrinth disorders     
Ear pain * 1  4/79 (5.06%)  0/30 (0.00%) 
Gastrointestinal disorders     
Vomiting * 1  39/79 (49.37%)  0/30 (0.00%) 
Diarrhoea * 1  20/79 (25.32%)  1/30 (3.33%) 
Nausea * 1  17/79 (21.52%)  0/30 (0.00%) 
Abdominal pain upper * 1  14/79 (17.72%)  3/30 (10.00%) 
Abdominal pain * 1  8/79 (10.13%)  0/30 (0.00%) 
Constipation * 1  8/79 (10.13%)  0/30 (0.00%) 
Gastrooesophageal reflux disease * 1  5/79 (6.33%)  1/30 (3.33%) 
Abdominal discomfort * 1  4/79 (5.06%)  1/30 (3.33%) 
General disorders     
Pyrexia * 1  20/79 (25.32%)  1/30 (3.33%) 
Non-cardiac chest pain * 1  9/79 (11.39%)  0/30 (0.00%) 
Fatigue * 1  8/79 (10.13%)  0/30 (0.00%) 
Catheter site pain * 1  7/79 (8.86%)  0/30 (0.00%) 
Peripheral swelling * 1  5/79 (6.33%)  0/30 (0.00%) 
Infusion site bruising * 1  4/79 (5.06%)  0/30 (0.00%) 
Infusion site pain * 1  4/79 (5.06%)  0/30 (0.00%) 
Immune system disorders     
Seasonal allergy * 1  14/79 (17.72%)  0/30 (0.00%) 
Infections and infestations     
Nasopharyngitis * 1  29/79 (36.71%)  3/30 (10.00%) 
Upper respiratory tract infection * 1  25/79 (31.65%)  1/30 (3.33%) 
Ear infection * 1  11/79 (13.92%)  0/30 (0.00%) 
Influenza * 1  8/79 (10.13%)  1/30 (3.33%) 
Gastroenteritis * 1  7/79 (8.86%)  1/30 (3.33%) 
Gastroenteritis viral * 1  7/79 (8.86%)  0/30 (0.00%) 
Pharyngitis streptococcal * 1  6/79 (7.59%)  1/30 (3.33%) 
Sinusitis * 1  6/79 (7.59%)  1/30 (3.33%) 
Injury, poisoning and procedural complications     
Procedural pain * 1  25/79 (31.65%)  0/30 (0.00%) 
Contusion * 1  24/79 (30.38%)  1/30 (3.33%) 
Fall * 1  22/79 (27.85%)  6/30 (20.00%) 
Skin abrasion * 1  21/79 (26.58%)  0/30 (0.00%) 
Ligament sprain * 1  12/79 (15.19%)  3/30 (10.00%) 
Arthropod bite * 1  10/79 (12.66%)  1/30 (3.33%) 
Muscle strain * 1  7/79 (8.86%)  0/30 (0.00%) 
Laceration * 1  4/79 (5.06%)  1/30 (3.33%) 
Limb injury * 1  4/79 (5.06%)  0/30 (0.00%) 
Spinal compression fracture * 1  4/79 (5.06%)  0/30 (0.00%) 
Torus fracture * 1  0/79 (0.00%)  2/30 (6.67%) 
Musculoskeletal and connective tissue disorders     
Back pain * 1  29/79 (36.71%)  1/30 (3.33%) 
Pain in extremity * 1  26/79 (32.91%)  2/30 (6.67%) 
Arthralgia * 1  16/79 (20.25%)  2/30 (6.67%) 
Myalgia * 1  9/79 (11.39%)  0/30 (0.00%) 
Muscle spasms * 1  6/79 (7.59%)  1/30 (3.33%) 
Musculoskeletal pain * 1  6/79 (7.59%)  0/30 (0.00%) 
Muscular weakness * 1  4/79 (5.06%)  2/30 (6.67%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Skin papilloma * 1  5/79 (6.33%)  1/30 (3.33%) 
Nervous system disorders     
Headache * 1  40/79 (50.63%)  1/30 (3.33%) 
Dizziness * 1  9/79 (11.39%)  0/30 (0.00%) 
Migraine * 1  4/79 (5.06%)  0/30 (0.00%) 
Psychiatric disorders     
Anxiety * 1  6/79 (7.59%)  1/30 (3.33%) 
Insomnia * 1  4/79 (5.06%)  0/30 (0.00%) 
Renal and urinary disorders     
Proteinuria * 1  8/79 (10.13%)  0/30 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough * 1  36/79 (45.57%)  1/30 (3.33%) 
Nasal congestion * 1  25/79 (31.65%)  1/30 (3.33%) 
Rhinorrhoea * 1  22/79 (27.85%)  0/30 (0.00%) 
Oropharyngeal pain * 1  20/79 (25.32%)  0/30 (0.00%) 
Epistaxis * 1  11/79 (13.92%)  1/30 (3.33%) 
Respiratory tract congestion * 1  7/79 (8.86%)  0/30 (0.00%) 
Dyspnoea * 1  4/79 (5.06%)  0/30 (0.00%) 
Upper respiratory tract congestion * 1  4/79 (5.06%)  1/30 (3.33%) 
Skin and subcutaneous tissue disorders     
Rash * 1  17/79 (21.52%)  2/30 (6.67%) 
Erythema * 1  6/79 (7.59%)  0/30 (0.00%) 
Vascular disorders     
Flushing * 1  4/79 (5.06%)  0/30 (0.00%) 
1
Term from vocabulary, MedDRA (17.1)
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Director
Organization: Sarepta Therapeutics, Inc.
Phone: +1-800-690-2003
EMail: clinicaltrials@sarepta.com
Layout table for additonal information
Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02255552    
Other Study ID Numbers: 4658-301
First Submitted: September 25, 2014
First Posted: October 2, 2014
Results First Submitted: June 12, 2020
Results First Posted: July 1, 2020
Last Update Posted: July 1, 2020