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A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03769116
Recruitment Status : Completed
First Posted : December 7, 2018
Results First Posted : September 15, 2023
Last Update Posted : October 25, 2023
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Muscular Dystrophy, Duchenne
Interventions Genetic: delandistrogene moxeparvovec
Genetic: placebo
Enrollment 41
Recruitment Details The following interim results presented are based on data up to Part 1 (Week 48) only of the study. Results for Part 2 and Part 3 will be presented after study completion.
Pre-assignment Details  
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Hide Arm/Group Description Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3. Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Period Title: Overall Study
Started 20 21
Received At Least 1 Dose Of Study Drug [1] 20 21
Completed [2] 20 21
Not Completed 0 0
[1]
Intent-to-Treat (ITT) Population
[2]
Participants completing Part 1; study ongoing.
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2 Total
Hide Arm/Group Description Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3. Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. Total of all reporting groups
Overall Number of Baseline Participants 20 21 41
Hide Baseline Analysis Population Description
ITT Population: all randomized participants who received study treatment during Part 1.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants 21 participants 41 participants
6.29  (1.19) 6.24  (1.13) 6.27  (1.15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 21 participants 41 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
20
 100.0%
21
 100.0%
41
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 21 participants 41 participants
Hispanic or Latino
1
   5.0%
4
  19.0%
5
  12.2%
Not Hispanic or Latino
19
  95.0%
16
  76.2%
35
  85.4%
Unknown or Not Reported
0
   0.0%
1
   4.8%
1
   2.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 21 participants 41 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
4
  20.0%
1
   4.8%
5
  12.2%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
13
  65.0%
17
  81.0%
30
  73.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
3
  15.0%
3
  14.3%
6
  14.6%
NSAA Group   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
 Number Analyzed 20 participants 21 participants 41 participants
NSAA baseline total score ≥ median score
8
  40.0%
15
  71.4%
23
  56.1%
NSAA baseline total score < median score
12
  60.0%
6
  28.6%
18
  43.9%
[1]
Measure Description: Median score = 21
1.Primary Outcome
Title Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
Hide Description Change from baseline in delandistrogene moxeparvovec dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 12. For this endpoint, 2 blocks of tissues were analyzed by Western blot, each with 2 technical replicates to determine the delandistrogene moxeparvovec dystrophin protein level as compared to a healthy individual (Percent Normal). The block average value from 2 technical replicates was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for the analysis. Dystrophin protein measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein.
Time Frame Baseline, Week 12 (Part 1)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population: all randomized participants who received study treatment during Part 1.
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Hide Arm/Group Description:
Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.
Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Overall Number of Participants Analyzed 20 21
Mean (Standard Deviation)
Unit of Measure: Percent Normal
Baseline 4.23  (6.83) 1.91  (1.28)
Change from Baseline 23.82  (39.76) 0.14  (1.24)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2, Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Comments Analysis conducted on changes from baseline.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
Method of Estimation Estimation Parameter Hodges-Lehmann treatment diff
Estimated Value 6.11
Confidence Interval (2-Sided) 95%
2.08 to 12.58
Estimation Comments [Not Specified]
2.Primary Outcome
Title Change From Baseline at Week 48 in North Star Ambulatory Assessment (NSAA) Total Score
Hide Description The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
Time Frame Baseline, Week 48 (Part 1)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population: all randomized participants who received study treatment during Part 1. Here, 'Overall Number of Participants Analyzed' signifies those who were evaluable for this outcome measure.
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Hide Arm/Group Description:
Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.
Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Overall Number of Participants Analyzed 19 21
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
1.7  (0.6) 0.9  (0.6)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2, Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3730
Comments [Not Specified]
Method Mixed-model for Repeated Measures
Comments [Not Specified]
Method of Estimation Estimation Parameter LSM Change Difference
Estimated Value 0.8
Confidence Interval (2-Sided) 95%
-1.0 to 2.7
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.9
Estimation Comments [Not Specified]
3.Other Pre-specified Outcome
Title Change From Baseline at Week 48 in NSAA Total Score by Age Group
Hide Description The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
Time Frame Baseline, Week 48 (Part 1)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population: all randomized participants who received study treatment during Part 1. Here, 'Overall Number of Participants Analyzed' signifies those who were evaluable for this outcome measure and 'Number Analyzed' signifies those who were evaluable for the specific category.
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Hide Arm/Group Description:
Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.
Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Overall Number of Participants Analyzed 19 21
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Age Group: 4-5 years old Number Analyzed 8 participants 8 participants
4.3  (0.6) 1.9  (0.6)
Age Group: 6-7 years old Number Analyzed 11 participants 13 participants
-0.2  (0.7) 0.5  (0.7)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2, Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Comments Change from baseline - Age Group: 4-5 years old
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0172
Comments [Not Specified]
Method Mixed-model for Repeated Measures
Comments [Not Specified]
Method of Estimation Estimation Parameter LSM Change Difference
Estimated Value 2.5
Confidence Interval (2-Sided) 95%
0.5 to 4.4
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2, Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Comments Change from baseline - Age Group: 6-7 years old
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5384
Comments [Not Specified]
Method Mixed-model for Repeated Measures
Comments [Not Specified]
Method of Estimation Estimation Parameter LSM Change Difference
Estimated Value -0.7
Confidence Interval (2-Sided) 95%
-3.0 to 1.6
Parameter Dispersion
Type: Standard Error of the Mean
Value: 1.1
Estimation Comments [Not Specified]
4.Other Pre-specified Outcome
Title Baseline NSAA Total Score by Age Group
Hide Description The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). This outcome measure presents only the baseline NSAA total score of the ITT population by the following age groups: 4-5 years old; 6-7 years old.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population: all randomized participants who received study treatment during Part 1.
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Hide Arm/Group Description:
Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.
Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Overall Number of Participants Analyzed 20 21
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Age Group: 4-5 years old Number Analyzed 8 participants 8 participants
20.1  (1.9) 20.4  (2.7)
Age Group: 6-7 years old Number Analyzed 12 participants 13 participants
19.6  (4.1) 24.0  (2.9)
Time Frame From Day 1 up to Week 48
Adverse Event Reporting Description The following interim safety results presented are based on safety data up to Part 1 (Week 48) only of the study. Safety results for Part 2 and Part 3 will be presented after study completion. The Safety Population is defined as all participants who received study treatment during Part 1.
 
Arm/Group Title Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Hide Arm/Group Description Participant received delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3. Participant received matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
All-Cause Mortality
Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Affected / at Risk (%) Affected / at Risk (%)
Total   0/20 (0.00%)   0/21 (0.00%) 
Hide Serious Adverse Events
Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Affected / at Risk (%) Affected / at Risk (%)
Total   3/20 (15.00%)   2/21 (9.52%) 
Hepatobiliary disorders     
Liver injury † 1  1/20 (5.00%)  0/21 (0.00%) 
Injury, poisoning and procedural complications     
Humerus fracture † 1  0/20 (0.00%)  1/21 (4.76%) 
Investigations     
Transaminases increased † 1  1/20 (5.00%)  0/21 (0.00%) 
Musculoskeletal and connective tissue disorders     
Rhabdomyolysis † 1  2/20 (10.00%)  1/21 (4.76%) 
1
Term from vocabulary, MedDRA 20.1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Affected / at Risk (%) Affected / at Risk (%)
Total   20/20 (100.00%)   21/21 (100.00%) 
Ear and labyrinth disorders     
Ear pain † 1  1/20 (5.00%)  3/21 (14.29%) 
Gastrointestinal disorders     
Vomiting † 1  13/20 (65.00%)  7/21 (33.33%) 
Nausea † 1  7/20 (35.00%)  2/21 (9.52%) 
Abdominal pain † 1  5/20 (25.00%)  2/21 (9.52%) 
Abdominal pain upper † 1  5/20 (25.00%)  4/21 (19.05%) 
Constipation † 1  3/20 (15.00%)  0/21 (0.00%) 
Diarrhoea † 1  3/20 (15.00%)  2/21 (9.52%) 
General disorders     
Pyrexia † 1  4/20 (20.00%)  1/21 (4.76%) 
Vessel puncture site haemorrhage † 1  2/20 (10.00%)  2/21 (9.52%) 
Infections and infestations     
Upper respiratory tract infection † 1  13/20 (65.00%)  13/21 (61.90%) 
Viral infection † 1  8/20 (40.00%)  9/21 (42.86%) 
Gastroenteritis † 1  3/20 (15.00%)  2/21 (9.52%) 
Sinusitis † 1  2/20 (10.00%)  2/21 (9.52%) 
Otitis media † 1  1/20 (5.00%)  3/21 (14.29%) 
Nasopharyngitis † 1  0/20 (0.00%)  2/21 (9.52%) 
Rhinovirus infection † 1  2/20 (10.00%)  0/21 (0.00%) 
Injury, poisoning and procedural complications     
Procedural pain † 1  5/20 (25.00%)  7/21 (33.33%) 
Skin abrasion † 1  4/20 (20.00%)  2/21 (9.52%) 
Limb injury † 1  3/20 (15.00%)  1/21 (4.76%) 
Incision site haemorrhage † 1  2/20 (10.00%)  4/21 (19.05%) 
Arthropod bite † 1  1/20 (5.00%)  3/21 (14.29%) 
Post procedural contusion † 1  1/20 (5.00%)  2/21 (9.52%) 
Contusion † 1  2/20 (10.00%)  1/21 (4.76%) 
Craniocerebral injury † 1  2/20 (10.00%)  0/21 (0.00%) 
Lip injury † 1  2/20 (10.00%)  0/21 (0.00%) 
Investigations     
Gamma-glutamyltransferase increased † 1  5/20 (25.00%)  0/21 (0.00%) 
Blood bilirubin increased † 1  2/20 (10.00%)  0/21 (0.00%) 
Weight increased † 1  2/20 (10.00%)  0/21 (0.00%) 
Metabolism and nutrition disorders     
Decreased appetite † 1  8/20 (40.00%)  0/21 (0.00%) 
Increased appetite † 1  0/20 (0.00%)  2/21 (9.52%) 
Musculoskeletal and connective tissue disorders     
Arthralgia † 1  5/20 (25.00%)  1/21 (4.76%) 
Pain in extremity † 1  5/20 (25.00%)  5/21 (23.81%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Skin papilloma † 1  2/20 (10.00%)  0/21 (0.00%) 
Nervous system disorders     
Headache † 1  4/20 (20.00%)  6/21 (28.57%) 
Psychiatric disorders     
Irritability † 1  3/20 (15.00%)  1/21 (4.76%) 
Sleep disorder † 1  3/20 (15.00%)  1/21 (4.76%) 
Abnormal behaviour † 1  2/20 (10.00%)  0/21 (0.00%) 
Renal and urinary disorders     
Dysuria † 1  2/20 (10.00%)  0/21 (0.00%) 
Pollakiuria † 1  2/20 (10.00%)  0/21 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough † 1  9/20 (45.00%)  6/21 (28.57%) 
Rhinorrhoea † 1  4/20 (20.00%)  3/21 (14.29%) 
Nasal congestion † 1  3/20 (15.00%)  2/21 (9.52%) 
Epistaxis † 1  2/20 (10.00%)  3/21 (14.29%) 
Skin and subcutaneous tissue disorders     
Ecchymosis † 1  9/20 (45.00%)  4/21 (19.05%) 
Dermatitis † 1  1/20 (5.00%)  2/21 (9.52%) 
Dermatitis contact † 1  0/20 (0.00%)  3/21 (14.29%) 
Rash † 1  0/20 (0.00%)  3/21 (14.29%) 
Urticaria † 1  0/20 (0.00%)  3/21 (14.29%) 
1
Term from vocabulary, MedDRA 20.1
†
Indicates events were collected by systematic assessment
Caution should be taken in interpreting the treatment effect on the 6-7 years-old age group due to differences in baseline prognostic functional characteristics for certain assessments in treated versus placebo in Part 1.
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: 1-888-SAREPTA (1-888-727-3782)
EMail: SareptAlly@sarepta.com
Layout table for additonal information
Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03769116    
Other Study ID Numbers: SRP-9001-102
2021-000078-27 ( EudraCT Number )
First Submitted: December 6, 2018
First Posted: December 7, 2018
Results First Submitted: July 5, 2023
Results First Posted: September 15, 2023
Last Update Posted: October 25, 2023