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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-000337-39 | EudraCT Number |
Not provided
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Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There are no safety concerns with Eteplirsen.
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The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eteplirsen | Experimental | Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eteplirsen | Drug | Eteplirsen IV infusion once weekly. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Experiencing Adverse Events (AEs) | A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section. | Up to 162 weeks |
| Number of Participants Experiencing Death Due to Adverse Events | A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section. | Up to 162 weeks |
| Number of Participants Experiencing Adverse Events of Special Interest (AESIs) | AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section. | Up to 162 weeks |
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Inclusion Criteria:
Exclusion Criteria:
Other inclusion/exclusion criteria apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UZ-Gent | Ghent | 9000 | Belgium | |||
| Hopital Trousseau, Bâtiment lemariey |
Not provided
In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Eteplirsen | Participants received eteplirsen via intravenous (IV) infusions, once weekly, for up to 162 weeks. |
| 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_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 7, 2020 |
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| Paris |
| 75021 |
| France |
| Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile | Rome | 00168 | Italy |
| UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre | London | England | WC1N 1EH | United Kingdom |
| Received At Least 1 Dose Of Study Drug | Safety Set |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety Set: all participants who received eteplirsen.
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| ID | Title | Description |
|---|---|---|
| BG000 | Eteplirsen | Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks. |
| 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 Adverse Events (AEs) | A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section. | Safety Set: all participants who received eteplirsen. | Posted | Number | Participants | Up to 162 weeks |
|
|
| ||||||||||||||||||||||||||
| Primary | Number of Participants Experiencing Death Due to Adverse Events | A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section. | Safety Set: all participants who received eteplirsen. | Posted | Number | Participants | Up to 162 weeks |
|
| |||||||||||||||||||||||||||
| Primary | Number of Participants Experiencing Adverse Events of Special Interest (AESIs) | AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section. | Safety Set: all participants who received eteplirsen. | Posted | Number | Participants | Up to 162 weeks |
|
|
Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eteplirsen | Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks. | 0 | 15 | 1 | 15 | 14 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Influenza | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Enterobiasis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Foot and mouth disease | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Genital infection fungal | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Helminthic infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Molluscum contagiosum | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Onychomycosis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Catheter site swelling | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Device dislocation | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hyperpyrexia | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Infusion site extravasation | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Face injury | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Eye contusion | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Human bite | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Subcutaneous haematoma | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Keloid scar | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Motor dysfunction | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Psychomotor hyperactivity | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Tympanic membrane hyperaemia | Ear and labyrinth disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Chalazion | Eye disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Weight gain poor | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Dilatation ventricular | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Initial insomnia | Psychiatric disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Chromaturia | Renal and urinary disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Myoglobinuria | Renal and urinary disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Urine albumin/creatinine ratio abnormal | Investigations | MedDRA 17.1 | Systematic Assessment |
|
In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
There are agreements between the Principal Investigators and the Sponsor (or its agents) that restrict the PIs rights to discuss or publish trial results after the trial is completed.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | 1-888-SAREPTA (1-888-727-3782) | SareptAlly@sarepta.com |
| Jul 25, 2023 |
| Prot_SAP_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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C000611335 | eteplirsen |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
|
|