Trial Outcomes & Findings for A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D) (NCT NCT01976091)
NCT ID: NCT01976091
Last Updated: 2023-06-15
Results Overview
An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered study drug related. An AE was considered serious if, in the view of the investigator or sponsor, it resulted in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Treatment-related Treatment Emergent Adverse Event (TEAE) is defined as an TEAE that was classified by the investigator as related to treatment.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Up to 2 Years
Results posted on
2023-06-15
Participant Flow
Participants with limb-girdle muscular dystrophy, type 2D (LGMD2D) were enrolled into 3 cohorts: Cohort 1A (non-ambulant adult participants), Cohort 1B (pediatric participants), and Cohort 2 (pediatric participants).
Participant milestones
| Measure |
Cohort 1A: SRP-9004
SRP-9004 was administered once by Isolated Limb Infusion (ILI) to a single limb on Day 0. The administered dose was 1\*10\^12 vector genomes per kilogram body weight (vg/kg). Dose determined via supercoiled titer method.
|
Cohort 1B: SRP-9004
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 2\*10\^12 vg/kg split between the two extremities (1\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Cohort 2: SRP-9004
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 6\*10\^12 vg/kg split between the two extremities (3\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
|---|---|---|---|
|
Overall Study
STARTED
|
1
|
3
|
2
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
1
|
3
|
2
|
|
Overall Study
COMPLETED
|
1
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
Baseline characteristics by cohort
| Measure |
Cohort 1A: SRP-9004
n=1 Participants
SRP-9004 was administered once by ILI to a single limb on Day 0. The administered dose was 1\*10\^12 vector genomes per kilogram body weight (vg/kg). Dose determined via supercoiled titer method.
|
Cohort 1B: SRP-9004
n=3 Participants
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 2\*10\^12 vg/kg split between the two extremities (1\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Cohort 2: SRP-9004
n=2 Participants
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 6\*10\^12 vg/kg split between the two extremities (3\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Age, Continuous
|
49 Years
STANDARD_DEVIATION NA • n=5 Participants
|
10 Years
STANDARD_DEVIATION 2.65 • n=7 Participants
|
10 Years
STANDARD_DEVIATION 2.83 • n=5 Participants
|
16.5 Years
STANDARD_DEVIATION 16.06 • n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Up to 2 YearsPopulation: The FAS included all participants who received at least 1 administration of SRP-9004.
An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered study drug related. An AE was considered serious if, in the view of the investigator or sponsor, it resulted in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Treatment-related Treatment Emergent Adverse Event (TEAE) is defined as an TEAE that was classified by the investigator as related to treatment.
Outcome measures
| Measure |
Cohort 1A: SRP-9004
n=1 Participants
SRP-9004 was administered once by ILI to a single limb on Day 0. The administered dose was 1\*10\^12 vector genomes per kilogram body weight (vg/kg). Dose determined via supercoiled titer method.
|
Cohort 1B: SRP-9004
n=3 Participants
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 2\*10\^12 vg/kg split between the two extremities (1\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Cohort 2: SRP-9004
n=2 Participants
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 6\*10\^12 vg/kg split between the two extremities (3\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
|---|---|---|---|
|
Number of Participants With Adverse Events (AEs).
Adverse Events
|
1 Participants
|
3 Participants
|
2 Participants
|
|
Number of Participants With Adverse Events (AEs).
Serious Adverse Events
|
0 Participants
|
2 Participants
|
1 Participants
|
|
Number of Participants With Adverse Events (AEs).
Treatment Related TEAEs
|
1 Participants
|
3 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, Up to 2 YearsPopulation: The FAS included all participants who received at least 1 administration of SRP-9004. Here, Overall "Number of Participants Analyzed" signifies those who were evaluable for this outcome measure.
The 6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course in 6 minutes (timed), and the distance walked (in meters) was recorded.
Outcome measures
| Measure |
Cohort 1A: SRP-9004
n=1 Participants
SRP-9004 was administered once by ILI to a single limb on Day 0. The administered dose was 1\*10\^12 vector genomes per kilogram body weight (vg/kg). Dose determined via supercoiled titer method.
|
Cohort 1B: SRP-9004
n=2 Participants
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 2\*10\^12 vg/kg split between the two extremities (1\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Cohort 2: SRP-9004
n=1 Participants
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 6\*10\^12 vg/kg split between the two extremities (3\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
|---|---|---|---|
|
Change From Baseline of the Distance Walked in 6 Minutes (6MWT)
|
NA Meters
Standard Deviation NA
Only 1 participant was enrolled into Cohort 1A. The participant was non-ambulatory at baseline and was not able to complete the 6MWT. Therefore, the Mean and SD are not applicable to this arm.
|
-66.0 Meters
Standard Deviation 77.78
|
29.0 Meters
Standard Deviation NA
Only 1 participant had evaluable data in this arm. Therefore, the SD is not applicable to this arm.
|
Adverse Events
Cohort 1A: SRP-9004
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 1B: SRP-9004
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2: SRP-9004
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1A: SRP-9004
n=1 participants at risk
SRP-9004 was administered once by ILI to a single limb on Day 0. The administered dose was 1\*10\^12 vector genomes per kilogram body weight (vg/kg). Dose determined via supercoiled titer method.
|
Cohort 1B: SRP-9004
n=3 participants at risk
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 2\*10\^12 vg/kg split between the two extremities (1\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Cohort 2: SRP-9004
n=2 participants at risk
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 6\*10\^12 vg/kg split between the two extremities (3\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Renal and urinary disorders
Myoglobinuria
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
Other adverse events
| Measure |
Cohort 1A: SRP-9004
n=1 participants at risk
SRP-9004 was administered once by ILI to a single limb on Day 0. The administered dose was 1\*10\^12 vector genomes per kilogram body weight (vg/kg). Dose determined via supercoiled titer method.
|
Cohort 1B: SRP-9004
n=3 participants at risk
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 2\*10\^12 vg/kg split between the two extremities (1\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
Cohort 2: SRP-9004
n=2 participants at risk
SRP-9004 was administered once by ILI to both limbs on Day 0. The participants received a total dose of 6\*10\^12 vg/kg split between the two extremities (3\*10\^12 vg/kg per limb). Dose determined via supercoiled titer method.
|
|---|---|---|---|
|
Psychiatric disorders
Insomnia
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Skin and subcutaneous tissue disorders
Rash
|
100.0%
1/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
100.0%
1/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
General disorders
Catheter site haematoma
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
66.7%
2/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
100.0%
3/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
100.0%
2/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Nervous system disorders
Hypoaesthesia
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
General disorders
Fatigue
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
100.0%
2/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Infections and infestations
Viral infection
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Axillary mass
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
General disorders
Injection site haematoma
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Psychiatric disorders
Depression
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Psychiatric disorders
Attention deficit/hyperactivity disorder
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Blood and lymphatic system disorders
Increased tendency to bruise
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Nervous system disorders
Tremor
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Post procedural contusion
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Endocrine disorders
Adrenal suppression
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Nervous system disorders
Presyncope
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Psychiatric disorders
Depressed mood
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Metabolism and nutrition disorders
Insulin resistance
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
100.0%
2/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Investigations
Urine analysis abnormal
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Nervous system disorders
Headache
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Gastrointestinal disorders
Gastrointestinal hypomotility
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Infections and infestations
Post procedural cellulitis
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Gastrointestinal disorders
Faeces pale
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Vascular disorders
Flushing
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
General disorders
Axillary pain
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
General disorders
Malaise
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
33.3%
1/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
|
Renal and urinary disorders
Myoglobinuria
|
0.00%
0/1 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
0.00%
0/3 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
50.0%
1/2 • Baseline up to 2 Years
The FAS included all participants who were enrolled and received at least 1 dose of SRP-9004 during the study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER