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This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eteplirsen | Experimental | Eteplirsen will be administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose will be 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants will continue to receive eteplirsen at 30 mg/kg for the duration of the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eteplirsen | Drug | Infusion for intravenous use. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study drug. Abnormalities presented at Baseline were considered AEs if they reoccurred after resolution or worsen during the AE collection period. An SAE was defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | Baseline up to Week 100 |
| Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality | Clinical laboratory parameters that were evaluated included
| Baseline up to Week 100 |
| Number of Participants With at Least 1 Markedly Abnormal Vital Sign | The vital sign parameters that were evaluated included blood pressure, heart rate, respiration, and temperature. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | Baseline up to Week 100 |
| Abnormal Changes From Baseline or Worsening of Physical Examination Findings |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Plasma Concentration (Cmax) of Eteplirsen | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) | |
| Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Universitair ziekenhuis Gent | Ghent | 9000 | Belgium | |||
| Armand-Trousseau Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37207382 | Derived | Mercuri E, Seferian AM, Servais L, Deconinck N, Stevenson H, Ni X, Zhang W, East L, Yonren S, Muntoni F; 4658-102 Study Group. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping. Neuromuscul Disord. 2023 Jun;33(6):476-483. doi: 10.1016/j.nmd.2023.03.008. Epub 2023 Mar 24. |
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Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) featuring a deletion mutation amenable to exon 51 skipping were enrolled into 2 cohorts based on their age.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eteplirsen | Eteplirsen was administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Overall Study | Eteplirsen was administered once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study drug. Abnormalities presented at Baseline were considered AEs if they reoccurred after resolution or worsen during the AE collection period. An SAE was defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. | Posted | Count of Participants | Participants | Baseline up to Week 100 |
Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
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 | Cohort 1: Age 24 to 48 Months | Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchiolitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | 800-690-2003 | clinicaltrials@sarepta.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 7, 2020 | Nov 10, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 14, 2021 | Nov 10, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| C000611335 | eteplirsen |
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Data not collected during the study for this Outcome Measure. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. |
| Baseline up to Week 100 |
| Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO) | The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the centrally read ECG report were clinically significant. Clinical significance was defined as any variation in ECG findings that had medical relevance resulting in an alteration in medical care. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the ECHO report were clinically significant. Clinical significance was defined as any variation in ECHO findings that had medical relevance resulting in an alteration in medical care. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96 |
| Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
| Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
| Amount of Drug Eliminated in Urine | Amount of unchanged drug excreted in urine from time 0 to 4 hours after completion of dosing is reported. | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
| Paris |
| 75012 |
| France |
| Site Fondazione Policlinico Universitario Agostino Gemelli | Roma | Italy |
| UCL Great Ormond Street Institute of Child Health | London | WC1N 1EH | United Kingdom |
| months |
|
| Age, Customized | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Ethnicity data are reported as "Missing" for participants from France because French sites and the French Regulations prohibit the entry of race and ethnicity. | Count of Participants | Participants |
|
| Race (NIH/OMB) | Race data are reported as "Missing" for participants from France because French sites and the French Regulations prohibit the entry of race and ethnicity. | Count of Participants | Participants |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | Cohort 1: Age 24 to 48 Months | Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study. |
| OG001 | Cohort 2: Age 6 to <24 Months | Participants aged 6 to <24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study. |
|
|
| Primary | Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality | Clinical laboratory parameters that were evaluated included
| The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. | Posted | Count of Participants | Participants | Baseline up to Week 100 |
|
|
|
| Primary | Number of Participants With at Least 1 Markedly Abnormal Vital Sign | The vital sign parameters that were evaluated included blood pressure, heart rate, respiration, and temperature. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. | Posted | Count of Participants | Participants | Baseline up to Week 100 |
|
|
|
| Primary | Abnormal Changes From Baseline or Worsening of Physical Examination Findings | Data not collected during the study for this Outcome Measure. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. Data were not collected for this Outcome Measure. | Posted | Baseline up to Week 100 |
|
|
| Primary | Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO) | The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the centrally read ECG report were clinically significant. Clinical significance was defined as any variation in ECG findings that had medical relevance resulting in an alteration in medical care. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the ECHO report were clinically significant. Clinical significance was defined as any variation in ECHO findings that had medical relevance resulting in an alteration in medical care. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section. | The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. | Posted | Count of Participants | Participants | Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96 |
|
|
|
| Secondary | Maximum Plasma Concentration (Cmax) of Eteplirsen | The Safety Set was used for pharmacokinetic(s) (PK) analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number Analyzed' signifies number of participants evaluable at the specified timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | microgram/milliliter (μg/mL) | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
|
|
|
| Secondary | Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen | The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number Analyzed' signifies number of participants evaluable at the specified timepoint. | Posted | Median | Full Range | hours (hr) | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
|
|
|
| Secondary | Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma | The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number Analyzed' signifies number of participants evaluable at the specified timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | μg*hr/mL | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
|
|
|
| Secondary | Amount of Drug Eliminated in Urine | Amount of unchanged drug excreted in urine from time 0 to 4 hours after completion of dosing is reported. | The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable at the specified timepoint. | Posted | Mean | Standard Deviation | μg | Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) |
|
|
|
| 0 |
| 9 |
| 0 |
| 9 |
| 9 |
| 9 |
| EG001 | Cohort 2: Age 6 to <24 Months | Participants aged 6 to <24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study. | 0 | 6 | 1 | 6 | 6 | 6 |
| Rhinitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Eye infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Gastrointestinal viral infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Molluscum contagiosum | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Roseola | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Varicella | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Catheter site eczema | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Catheter site swelling | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Discomfort | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Hyperpyrexia | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Infusion site extravasation | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Localised oedema | General disorders | MedDRA 22.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Teething | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Dental discomfort | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Faeces discoloured | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Head injury | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Lip injury | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Penis injury | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Spinal fracture | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Tongue injury | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Tracheal obstruction | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Vaccination complication | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Eczema nummular | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Miliaria | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Pruritus generalised | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA 22.1 | Systematic Assessment |
|
| Cerumen impaction | Ear and labyrinth disorders | MedDRA 22.1 | Systematic Assessment |
|
| External ear inflammation | Ear and labyrinth disorders | MedDRA 22.1 | Systematic Assessment |
|
| Inner ear inflammation | Ear and labyrinth disorders | MedDRA 22.1 | Systematic Assessment |
|
| Tympanic membrane hyperaemia | Ear and labyrinth disorders | MedDRA 22.1 | Systematic Assessment |
|
| Lethargy | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Psychomotor hyperactivity | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
|
| Hyposideraemia | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
|
| Increased appetite | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
|
| Chalazion | Eye disorders | MedDRA 22.1 | Systematic Assessment |
|
| Eye irritation | Eye disorders | MedDRA 22.1 | Systematic Assessment |
|
| Eye pruritus | Eye disorders | MedDRA 22.1 | Systematic Assessment |
|
| Visual acuity reduced | Eye disorders | MedDRA 22.1 | Systematic Assessment |
|
| Body temperature | Investigations | MedDRA 22.1 | Systematic Assessment |
|
| Body temperature increased | Investigations | MedDRA 22.1 | Systematic Assessment |
|
| Amylase increased | Investigations | MedDRA 22.1 | Systematic Assessment |
|
| Blood iron decreased | Investigations | MedDRA 22.1 | Systematic Assessment |
|
| Haemophilus test positive | Investigations | MedDRA 22.1 | Systematic Assessment |
|
| Streptococcus test positive | Investigations | MedDRA 22.1 | Systematic Assessment |
|
| Abnormal behaviour | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
|
| Anger | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
|
| Initial insomnia | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
|
| Irritability | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
|
| Personality change | Psychiatric disorders | MedDRA 22.1 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 22.1 | Systematic Assessment |
|
| Haematoma | Vascular disorders | MedDRA 22.1 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 22.1 | Systematic Assessment |
|
| Autoimmune neutropenia | Blood and lymphatic system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Hypochromic anaemia | Blood and lymphatic system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Haemoglobinopathy | Congenital, familial and genetic disorders | MedDRA 22.1 | Systematic Assessment |
|
| Allergy to chemicals | Immune system disorders | MedDRA 22.1 | Systematic Assessment |
|
| Dysuria | Renal and urinary disorders | MedDRA 22.1 | Systematic Assessment |
|
| Genital cyst | Reproductive system and breast disorders | MedDRA 22.1 | Systematic Assessment |
|
Not provided
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Week 6 (10 mg/kg) |
|
|
| Week 8 (20 mg/kg) |
|
|
| Week 10 (30 mg/kg) |
|
|
| Week 24 (30 mg/kg) |
|
|
| 10 mg/kg (Week 6) |
|
|
| 20 mg/kg (Week 8) |
|
|
| 30 mg/kg (Week 10) |
|
|
| 30 mg/kg (Week 24) |
|
|
| 10 mg/kg (Week 6) |
|
|
| 20 mg/kg (Week 8) |
|
|
| 30 mg/kg (Week 10) |
|
|
| 30 mg/kg (Week 24) |
|
|
| 10 mg/kg (Week 6) |
|
|
| 20 mg/kg (Week 8) |
|
|
| 30 mg/kg (Week 10) |
|
|
| 30 mg/kg (Week 24) |
|
|