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| ID | Type | Description | Link |
|---|---|---|---|
| 5U01AR060911 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| Nationwide Children's Hospital | OTHER |
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This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1A: SRP-9004 Low Dose (Single Limb Perfusion) | Experimental | Non-ambulant participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to a single limb on Day 0. |
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| Cohort 1B Low Dose (Bilateral Limb Perfusion) | Experimental | Participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to both limbs on Day 0. |
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| Cohort 2 High Dose (Bilateral Limb Perfusion) | Experimental | Participants with LGMD2D will receive 1 high dose of SRP-9004 via ILI to both limbs on Day 0. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-9004 | Genetic | Isolated Limb Infusion (ILI) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs). | 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. | Up to 2 Years |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline of the Distance Walked in 6 Minutes (6MWT) | 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. | Baseline, Up to 2 Years |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other inclusion/exclusion criteria apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30838895 | Background | Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Jul;30(7):794-801. doi: 10.1089/hum.2019.006. Epub 2019 Apr 19. | |
| 21031578 | Background | Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251. |
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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).
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | Cohort 1B: SRP-9004 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 10, 2017 | Mar 7, 2022 |
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| 19798725 | Background | Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732. |
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. |
| FG002 | 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. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
|
The full analysis set (FAS) included all participants who received at least 1 administration of SRP-9004 during the study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1A: SRP-9004 | 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. |
| BG001 | 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. |
| BG002 | 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. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs). | 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. | The FAS included all participants who received at least 1 administration of SRP-9004. | Posted | Count of Participants | Participants | Up to 2 Years |
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| Secondary | Change From Baseline of the Distance Walked in 6 Minutes (6MWT) | 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. | 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. | Posted | Mean | Standard Deviation | Meters | Baseline, Up to 2 Years |
|
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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1A: SRP-9004 | 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. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG001 | 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. | 0 | 3 | 2 | 3 | 3 | 3 |
| EG002 | 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. | 0 | 2 | 1 | 2 | 2 | 2 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Myoglobinuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Insomnia | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Catheter site haematoma | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 22.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Viral infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Axillary mass | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Injection site haematoma | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Haemoglobin decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
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| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Attention deficit/hyperactivity disorder | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Increased tendency to bruise | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
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| Tremor | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
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| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Adrenal suppression | Endocrine disorders | MedDRA 22.0 | Systematic Assessment |
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| Presyncope | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
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| Depressed mood | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Insulin resistance | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Proteinuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
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| Urine analysis abnormal | Investigations | MedDRA 22.0 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
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| Gastrointestinal hypomotility | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Post procedural cellulitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Faeces pale | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
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| Flushing | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
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| Femur fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Axillary pain | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Malaise | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Myoglobinuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Sarepta Therapeutics, Inc | 1-800-690-2003 | SareptAlly@sarepta.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 25, 2022 | Aug 16, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D058088 | Sarcoglycanopathies |
| D049288 | Muscular Dystrophies, Limb-Girdle |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D012120 | Respiration Disorders |
| D012140 | Respiratory Tract Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Title | Measurements |
|---|---|
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| Treatment Related TEAEs |
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