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A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03375164
Recruitment Status : Completed
First Posted : December 15, 2017
Results First Posted : May 23, 2024
Last Update Posted : May 23, 2024
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
Intervention Genetic: delandistrogene moxeparvovec
Enrollment 4
Recruitment Details  
Pre-assignment Details In total, 4 participants were screened for the study. There were no screen failures.
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Period Title: Overall Study
Started 4
Received at Least 1 Dose of Study Drug 4 [1]
Completed 4
Not Completed 0
[1]
Full Analysis Set
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
Full Analysis Set: all participants who received study treatment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants
5.14  (0.91)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants
In utero 0
Preterm newborn infants (gestational age < 37 wks) 0
Newborns (0-27 days) 0
Infants and toddlers (28 days-23 months) 0
Children (2-11 years) 4
Adolescents (12-17 years) 0
Adults (18-64 years) 0
From 65-84 years 0
85 years and over 0
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
0
   0.0%
Male
4
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
4
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
3
  75.0%
More than one race
0
   0.0%
Unknown or Not Reported
1
  25.0%
1.Primary Outcome
Title Number of Participants With Adverse Events (AEs)
Hide Description An AE is any untoward medical occurrence in a clinical study participant that does not necessarily have a causal relationship with the study drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of a study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants who received study treatment.
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description:
Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Number of Participants Analyzed 4
Measure Type: Count of Participants
Unit of Measure: Participants
4
 100.0%
2.Secondary Outcome
Title Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot
Hide Description Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Day 90) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin protein levels in these muscle biopsy samples was determined by Western blot. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein.
Time Frame Baseline, Day 90
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants who received study treatment.
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description:
Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent control
70.52  (76.10)
3.Secondary Outcome
Title Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity
Hide Description Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Day 90) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined using IF. Automated software was used to quantify the intensity of dystrophin expression post-treatment compared to pre-treatment (Percent Normal). The number of muscle fibers expressing micro-dystrophin was quantified by independent trained evaluators. An increase in IF fiber intensity indicates increased delandistrogene moxeparvovec dystrophin expression.
Time Frame Baseline, Day 90
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants who received study treatment.
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description:
Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent fluorescent expression
93.59  (43.86)
4.Secondary Outcome
Title Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Hide Description Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Day 90) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined by IF PDPF. Automated software was used to quantify the intensity of dystrophin expression post-treatment compared to pre-treatment (Percent Normal). The number of muscle fibers expressing micro-dystrophin was quantified by independent trained evaluators. An increase in IF PDPF indicates increased delandistrogene moxeparvovec dystrophin expression.
Time Frame Baseline, Day 90
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants who received study treatment.
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description:
Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent dystrophin positive fibers
81.18  (10.19)
5.Secondary Outcome
Title Change From Baseline at Year 5 in the 100 Meter Timed Test
Hide Description This assessment measures the time needed to move 100 meters and served as the primary motor outcome measure for this study. A decrease in the time needed to move 100 meters indicates increased motor function.
Time Frame Baseline, Year 5
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants who received study treatment.
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description:
Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: second
-4.02  (4.64)
Time Frame Up to 5 years
Adverse Event Reporting Description All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
 
Arm/Group Title Delandistrogene Moxeparvovec
Hide Arm/Group Description Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
All-Cause Mortality
Delandistrogene Moxeparvovec
Affected / at Risk (%)
Total   0/4 (0.00%)    
Hide Serious Adverse Events
Delandistrogene Moxeparvovec
Affected / at Risk (%) # Events
Total   0/4 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Delandistrogene Moxeparvovec
Affected / at Risk (%) # Events
Total   4/4 (100.00%)    
Cardiac disorders   
Cardiomyopathy  1  1/4 (25.00%)  1
Eye disorders   
Eye irritation  1  1/4 (25.00%)  1
Gastrointestinal disorders   
Gastrooesophageal reflux disease  1  2/4 (50.00%)  2
Abdominal discomfort  1  1/4 (25.00%)  1
Abdominal distension  1  1/4 (25.00%)  1
Abdominal pain upper  1  1/4 (25.00%)  1
Anal incontinence  1  1/4 (25.00%)  1
Diarrhoea  1  1/4 (25.00%)  1
Nausea  1  1/4 (25.00%)  1
Vomiting  1  4/4 (100.00%)  15
General disorders   
Fatigue  1  2/4 (50.00%)  2
Asthenia  1  1/4 (25.00%)  1
Pyrexia  1  1/4 (25.00%)  1
Infections and infestations   
Upper respiratory tract infection  1  4/4 (100.00%)  11
COVID-19  1  2/4 (50.00%)  2
Gastroenteritis  1  1/4 (25.00%)  1
Gastroenteritis viral  1  1/4 (25.00%)  4
Subcutaneous abscess  1  1/4 (25.00%)  1
Viral infection  1  1/4 (25.00%)  1
Injury, poisoning and procedural complications   
Procedural pain  1  2/4 (50.00%)  2
Clavicle fracture  1  1/4 (25.00%)  1
Investigations   
Hepatic enzyme increased  1  3/4 (75.00%)  4
Influenza A virus test positive  1  1/4 (25.00%)  1
Metabolism and nutrition disorders   
Decreased appetite  1  2/4 (50.00%)  3
Musculoskeletal and connective tissue disorders   
Back pain  1  1/4 (25.00%)  1
Bone pain  1  1/4 (25.00%)  1
Pain in extremity  1  1/4 (25.00%)  3
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Skin papilloma  1  1/4 (25.00%)  1
Nervous system disorders   
Headache  1  1/4 (25.00%)  2
Psychiatric disorders   
Irritability  1  1/4 (25.00%)  1
Renal and urinary disorders   
Proteinuria  1  1/4 (25.00%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  2/4 (50.00%)  2
Asthma  1  1/4 (25.00%)  1
Skin and subcutaneous tissue disorders   
Dermatitis contact  1  1/4 (25.00%)  1
1
Term from vocabulary, MedDRA 24.1
Indicates events were collected by 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: 888-727-3782
EMail: SareptAlly@sarepta.com
Layout table for additonal information
Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03375164    
Other Study ID Numbers: SRP-9001-101
2021-000077-83 ( EudraCT Number )
First Submitted: December 4, 2017
First Posted: December 15, 2017
Results First Submitted: April 24, 2024
Results First Posted: May 23, 2024
Last Update Posted: May 23, 2024