Trial Outcomes & Findings for A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (NCT NCT03375164)

NCT ID: NCT03375164

Last Updated: 2024-11-14

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: Up to 5 years
• Results posted on: 2024-11-14

Participant Flow

In total, 4 participants were screened for the study. There were no screen failures.

Participant milestones

Measure	Delandistrogene Moxeparvovec Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Overall Study STARTED	4
Overall Study Received at Least 1 Dose of Study Drug	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Delandistrogene Moxeparvovec n=4 Participants Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Age, Continuous	5.14 years STANDARD_DEVIATION 0.91 • n=5 Participants
Age, Customized In utero	0 Participants n=5 Participants
Age, Customized Preterm newborn infants (gestational age < 37 wks)	0 Participants n=5 Participants
Age, Customized Newborns (0-27 days)	0 Participants n=5 Participants
Age, Customized Infants and toddlers (28 days-23 months)	0 Participants n=5 Participants
Age, Customized Children (2-11 years)	4 Participants n=5 Participants
Age, Customized Adolescents (12-17 years)	0 Participants n=5 Participants
Age, Customized Adults (18-64 years)	0 Participants n=5 Participants
Age, Customized From 65-84 years	0 Participants n=5 Participants
Age, Customized 85 years and over	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 5 years

Population: Full Analysis Set: all participants who received study treatment.

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.

Outcome measures

Measure	Delandistrogene Moxeparvovec n=4 Participants Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Number of Participants With Adverse Events (AEs)	4 Participants

SECONDARY outcome

Timeframe: Baseline, Day 90

Population: Full Analysis Set: all participants who received study treatment.

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.

Outcome measures

Measure	Delandistrogene Moxeparvovec n=4 Participants Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot	70.52 percent control Standard Deviation 76.10 • Interval 76.1 to

SECONDARY outcome

Timeframe: Baseline, Day 90

Population: Full Analysis Set: all participants who received study treatment.

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.

Outcome measures

Measure	Delandistrogene Moxeparvovec n=4 Participants Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity	93.59 percent fluorescent expression Standard Deviation 43.86 • Interval 43.86 to

SECONDARY outcome

Timeframe: Baseline, Day 90

Population: Full Analysis Set: all participants who received study treatment.

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.

Outcome measures

Measure	Delandistrogene Moxeparvovec n=4 Participants Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF)	81.18 percent dystrophin positive fibers Standard Deviation 10.19 • Interval 10.19 to

SECONDARY outcome

Timeframe: Baseline, Year 5

Population: Full Analysis Set: all participants who received study treatment.

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.

Outcome measures

Measure	Delandistrogene Moxeparvovec n=4 Participants Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Change From Baseline at Year 5 in the 100 Meter Timed Test	-4.02 second Standard Deviation 4.64 • Interval 4.64 to

Adverse Events

Delandistrogene Moxeparvovec

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Delandistrogene Moxeparvovec n=4 participants at risk Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1.
Gastrointestinal disorders Gastrooesophageal reflux disease	50.0% 2/4 • Number of events 2 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Abdominal discomfort	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Abdominal distension	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Abdominal pain upper	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Anal incontinence	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Diarrhoea	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Nausea	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
General disorders Fatigue	50.0% 2/4 • Number of events 2 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
General disorders Asthenia	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
General disorders Pyrexia	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Infections and infestations Upper respiratory tract infection	100.0% 4/4 • Number of events 11 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Infections and infestations COVID-19	50.0% 2/4 • Number of events 2 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Infections and infestations Gastroenteritis	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Infections and infestations Gastroenteritis viral	25.0% 1/4 • Number of events 4 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Infections and infestations Subcutaneous abscess	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Infections and infestations Viral infection	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Injury, poisoning and procedural complications Procedural pain	50.0% 2/4 • Number of events 2 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Injury, poisoning and procedural complications Clavicle fracture	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Investigations Hepatic enzyme increased	75.0% 3/4 • Number of events 4 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Investigations Influenza A virus test positive	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Metabolism and nutrition disorders Decreased appetite	50.0% 2/4 • Number of events 3 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Musculoskeletal and connective tissue disorders Back pain	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Musculoskeletal and connective tissue disorders Bone pain	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Musculoskeletal and connective tissue disorders Pain in extremity	25.0% 1/4 • Number of events 3 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Cardiac disorders Cardiomyopathy	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Eye disorders Eye irritation	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Gastrointestinal disorders Vomiting	100.0% 4/4 • Number of events 15 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin papilloma	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Nervous system disorders Headache	25.0% 1/4 • Number of events 2 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Psychiatric disorders Irritability	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Renal and urinary disorders Proteinuria	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Respiratory, thoracic and mediastinal disorders Cough	50.0% 2/4 • Number of events 2 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Respiratory, thoracic and mediastinal disorders Asthma	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.
Skin and subcutaneous tissue disorders Dermatitis contact	25.0% 1/4 • Number of events 1 • Up to 5 years All reported adverse events are based upon the Full Analysis Set: all participants who received study treatment.

Additional Information

Medical Director

Sarepta Therapeutics, Inc.

Phone: 888-727-3782

Email: SareptAlly@sarepta.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: OTHER