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The sponsor has decided to integrate 5051-102 into 5051-201. Participants from 5051-102 will be eligible to enroll in 5051-201 Part B.
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The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRP-5051 | Experimental | Participants will receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 studies. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-5051 | Drug | SRP-5051 administered as an IV infusion. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | A TEAE was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module. | Up to approximately 135 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Concentration of SRP-5051 | For pharmacokinetic (PK) analysis, plasma samples were collected pre-dose (approximately 30 minutes prior to the start of infusion), immediately prior to the end of infusion (prior to flush), and approximately 4 to 6 hours after the end of dosing. Results are reported in micrograms/liter (ug/L) and are presented as an average across the days specified in the time frame. |
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Inclusion Criteria:
• Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.
Exclusion Criteria:
Other inclusion/exclusion criteria apply.
* The dose of steroids must remain constant except for modifications to accommodate changes in weight.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | United States | ||
| NW FL Clinical Research Group, LLC |
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| ID | Title | Description |
|---|---|---|
| FG000 | SRP-5051 | Participants received SRP-5051 via intravenous (IV) infusion every 4 weeks (Q4W). |
| 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 | Apr 21, 2020 | Aug 21, 2024 |
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| Day 1, Day 84, every 84 days after Day 84 (up to a maximum of approximately 135 weeks) (pre-dose, immediately prior to end of infusion, up to 4-6 hours post-dose) |
| Gulf Breeze |
| Florida |
| 32561 |
| United States |
| Center for Integrative Rare Disease Research (CIRDR) | Atlanta | Georgia | 30318 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Children's Medical Center Dallas | Dallas | Texas | 75207 | United States |
| London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
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Safety Set: all participants who started the study drug infusion.
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| ID | Title | Description |
|---|---|---|
| BG000 | SRP-5051 | Participants received SRP-5051 via IV infusion Q4W. |
| Units | Counts |
|---|---|
| Participants |
|
| 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 |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 Experiencing Treatment-emergent Adverse Events (TEAEs) | A TEAE was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module. | Safety Set: all participants who started the study drug infusion. | Posted | Count of Participants | Participants | Up to approximately 135 weeks |
|
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| ||||||||||||||||||||||||||
| Secondary | Plasma Concentration of SRP-5051 | For pharmacokinetic (PK) analysis, plasma samples were collected pre-dose (approximately 30 minutes prior to the start of infusion), immediately prior to the end of infusion (prior to flush), and approximately 4 to 6 hours after the end of dosing. Results are reported in micrograms/liter (ug/L) and are presented as an average across the days specified in the time frame. | Pharmacokinetic Set: all participants who started the study drug infusion and had at least 1 PK concentration data collection. | Posted | Mean | Standard Deviation | ug/L | Day 1, Day 84, every 84 days after Day 84 (up to a maximum of approximately 135 weeks) (pre-dose, immediately prior to end of infusion, up to 4-6 hours post-dose) |
|
|
Up to approximately 135 weeks
Reported safety data based upon the Safety Set: all participants who started the study drug infusion.
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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 | SRP-5051 | Participants received SRP-5051 via IV infusion Q4W. | 2 | 15 | 6 | 15 | 14 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.1 | Systematic Assessment |
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| Cardiac arrest | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
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| Cardiogenic shock | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Colitis ischaemic | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Psychogenic seizure | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Atrioventricular block second degree | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal pain lower | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Haemorrhoids | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Impaired gastric emptying | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Melaena | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Mucous stools | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Catheter site pain | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Chest pain | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Injection site reaction | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Malaise | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Vessel puncture site bruise | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Tinea pedis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Tinea versicolour | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Back injury | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Joint injury | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Limb injury | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| C-reactive protein increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Blood glucose increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Cystatin C increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Glucose urine present | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Total complement activity increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Troponin I increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Glucose tolerance impaired | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Malnutrition | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Coccydynia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Joint stiffness | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Dizziness postural | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypoaesthesia | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Peripheral sensory neuropathy | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Presyncope | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Crystalluria | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Haematuria | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Ketonuria | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Proteinuria | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Laryngospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Restrictive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Livedo reticularis | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Flushing | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
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| Poor venous access | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | 1-888-727-3782 | SareptAlly@sarepta.com |
| Prot_000.pdf |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
|
| More than one race |
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| Unknown or Not Reported |
|
|