Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease).
This is an open-label, single-dose study of AT-GTX-501 administered by a single intrathecal injection. Safety and efficacy are evaluated over a 2 year period. The efficacy assessments in this study are to evaluate motor, language, visual, and cognitive function, as well as survival and other outcome measures. Participants are tested at baseline, receive AT-GTX-501 on Day 0, and return for visits on Days 7, 14, 21, and 30, and then every 3 months until Month 24. Following completion of this study, there is a long-term follow up study in which data will continue to be collected (Study AT-GTX-501-02 / NCT04273243).
For more information about this study, please contact Amicus Therapeutics Patient Advocacy at clinicaltrials@amicusrx.com or +1 609-662-2000.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open Label | Experimental | Participants with diagnosis of vLINCL6 Batten disease will receive a single intrathecal injection of AT-GTX-501 into the lumbar spinal cord region. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AT-GTX-501 | Genetic | CLN6 Gene delivered by Self-Complementary AAV9 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product, biologic, or medical device (medicinal products). An SAE is an AE occurring during any study phase (for example, baseline, treatment, or follow-up) that fulfils any of the following: results in death; is life-threatening; requires inpatient hospitalization or prolongs existing hospitalization; results in persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; and results in a congenital anomaly/birth defect. All AEs that occurred after receipt of AT-GTX-501 are classified as TEAEs. A summary of serious and all other non-SAEs, regardless of causality, is located in the Reported Adverse Events module. | Up to 38.7 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline In Hamburg Motor And Language Scores at Month 24 | The Hamburg scale is an established tool to capture the rate of decline or regression. This tool was developed to document by rating motor, language, and visual functions in participants with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) Batten disease, and to collect their incidence of grand mal seizures. To assess disease progression and evaluate efficacy, the combined score of the motor and language domains of the Hamburg scale was used. Each domain was scored from 0 (no function) to 3 (normal function) for a maximal possible score of 6 for the combined score, referred to as the Hamburg Motor + Language aggregate score. The rate of decline in Hamburg Motor + Language aggregate, Motor, and Language scores were summarized using descriptive statistics. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Emily de los Reyes, MD | Nationwide Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nationwide Children's Hosptial | Columbus | Ohio | 43205 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | AT-GTX-501 | Participants received a single intrathecal injection of AT-GTX-501 into the lumbar spinal cord region. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety population: all participants who were treated with AT-GTX-501.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | AT-GTX-501 | Participants received a single intrathecal injection of AT-GTX-501 into the lumbar spinal cord region. |
| 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 | Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product, biologic, or medical device (medicinal products). An SAE is an AE occurring during any study phase (for example, baseline, treatment, or follow-up) that fulfils any of the following: results in death; is life-threatening; requires inpatient hospitalization or prolongs existing hospitalization; results in persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; and results in a congenital anomaly/birth defect. All AEs that occurred after receipt of AT-GTX-501 are classified as TEAEs. A summary of serious and all other non-SAEs, regardless of causality, is located in the Reported Adverse Events module. | Safety population: all participants who were treated with AT-GTX-501. | Posted | Count of Participants | Participants | Up to 38.7 months |
|
Up to 38.7 months
Safety population: all participants who were treated with AT-GTX-501.
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 | AT-GTX-501 | Participants received a single intrathecal injection of AT-GTX-501 into the lumbar spinal cord region. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (23.0) | Systematic Assessment |
Due to the nature of the disease, coupled with the impact of a global pandemic, adjustments were made to the study design to prioritize collection of primary safety and efficacy data, including development assessments through remote telehealth visits. The results of this data are presented in this posting.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Amicus Therapeutics Patient Advocacy | Amicus Therapeutics | +1 609-662-2000 | clinicaltrials@amicusrx.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 | May 20, 2020 | Oct 26, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 2, 2022 | Oct 25, 2022 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| C566627 | Ceroid Lipofuscinosis, Neuronal, 6 |
| D009472 | Neuronal Ceroid-Lipofuscinoses |
| ID | Term |
|---|---|
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Screening (Day -30 up to -2) up to Month 24 |
| Change From Baseline In Unified Batten Disease Rating Scale (UBDRS) Scores At Month 24 | The Unified Batten Disease Rating Scale (UBDRS) was developed to monitor rate of progression. This scale includes assessment of extrapyramidal movement abnormalities and seizures, behavioral and capability assessments (Actual Vision), as well as history relevant to variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6) disease and scoring for global impression of symptom severity. A higher UBDRS subscale score indicated greater physical impairment, with zero indicating a better outcome. Subscales included Physical Assessment (range 0-84), Seizure Assessment (range 0-54), Behavioral Assessment (range 0-55) and Capability Assessment of Actual Vision and Normal Vision (range 0-14). A positive change from baseline score indicates increased impairment and a negative score indicates decreased impairment. | Screening (Day -30 up to -2) and Day 30 up to Month 24 |
| Change From Baseline In Mullen (Age-Equivalent) Scales Of Early Learning At Month 24 | The Mullen Scales of Early Learning were utilized to assess cognitive and motor ability in 4 areas (visual reception, fine motor, expressive language, and receptive language) in infants and children. Raw scores range from 0-50 for visual reception, 0-49 for fine motor, 0-50 for expressive language, and 0-48 for receptive language. Lower scores from baseline indicated increased developmental delay and higher scores from baseline indicated decreased developmental delay. A positive change from baseline score indicates decreased developmental delay and a negative score indicates increased developmental delay. | Screening (Day -30 up to -2) and Month 24 |
| Change From Baseline In Preschool Language Scales-5th Edition (PLS-5) (Age-Equivalent) At Month 24 | The Preschool Language Scales-5th Edition (PLS-5) (Age-Equivalent) was a comprehensive developmental language assessment. Standard scores from each domain (auditory comprehension, expressive communication, and total language) range from 50-150. Standard scores between 85 and 115 are considered to be within normal limits. Total scores are the sum of standard scores for auditory comprehension and expressive communication, which is then converted to a total standard score using PLS-5 Appendix B. Age-equivalent scores in each area were summarized by study visit with descriptive statistics. Higher scores represent better language development and lower scores reflect a possible language delay or disorder. A positive change from baseline score indicates better language development. | Screening (Day -30 up to -2) and Month 24 |
| Change From Baseline In Development Profile-3 At Month 24 | The Development Profile-3 was used to screen for developmental delays in 5 areas (physical [score range 0-35], adaptive behavior [0-37], social-emotional [0-36], cognitive [0-38], communication [0-34]), where lower scores indicated increased developmental delay. The General Development Score is obtained by adding the sum of standard scores for the five scales together (range 0-180). Standard Score ranges are <70 Delayed, 70-84 Below Average, 85-115 Average, 116-130 Above Average, and >130 Well Above Average. A positive change from baseline score indicates decreased developmental delay and a negative score indicates increased developmental delay. | Screening (Day -30 up to -2) and Month 24 |
| months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| AT-GTX-501 |
Participants received a single intrathecal injection of AT-GTX-501 into the lumbar spinal cord region. |
|
|
| Secondary | Change From Baseline In Hamburg Motor And Language Scores at Month 24 | The Hamburg scale is an established tool to capture the rate of decline or regression. This tool was developed to document by rating motor, language, and visual functions in participants with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) Batten disease, and to collect their incidence of grand mal seizures. To assess disease progression and evaluate efficacy, the combined score of the motor and language domains of the Hamburg scale was used. Each domain was scored from 0 (no function) to 3 (normal function) for a maximal possible score of 6 for the combined score, referred to as the Hamburg Motor + Language aggregate score. The rate of decline in Hamburg Motor + Language aggregate, Motor, and Language scores were summarized using descriptive statistics. | Full Analysis Set (FAS): participants who were treated with AT-GTX-501 and had an analyzable outcome (Hamburg Motor + Language aggregate score) at baseline. | Posted | Mean | Standard Deviation | score on a scale | Screening (Day -30 up to -2) up to Month 24 |
|
|
|
| Secondary | Change From Baseline In Unified Batten Disease Rating Scale (UBDRS) Scores At Month 24 | The Unified Batten Disease Rating Scale (UBDRS) was developed to monitor rate of progression. This scale includes assessment of extrapyramidal movement abnormalities and seizures, behavioral and capability assessments (Actual Vision), as well as history relevant to variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6) disease and scoring for global impression of symptom severity. A higher UBDRS subscale score indicated greater physical impairment, with zero indicating a better outcome. Subscales included Physical Assessment (range 0-84), Seizure Assessment (range 0-54), Behavioral Assessment (range 0-55) and Capability Assessment of Actual Vision and Normal Vision (range 0-14). A positive change from baseline score indicates increased impairment and a negative score indicates decreased impairment. | Full Analysis Set (FAS): participants who were treated with AT-GTX-501 and had an analyzable outcome (Unified Batten Disease Rating Scale [UBDRS]) scores at baseline. | Posted | Mean | Standard Deviation | score on a scale | Screening (Day -30 up to -2) and Day 30 up to Month 24 |
|
|
|
| Secondary | Change From Baseline In Mullen (Age-Equivalent) Scales Of Early Learning At Month 24 | The Mullen Scales of Early Learning were utilized to assess cognitive and motor ability in 4 areas (visual reception, fine motor, expressive language, and receptive language) in infants and children. Raw scores range from 0-50 for visual reception, 0-49 for fine motor, 0-50 for expressive language, and 0-48 for receptive language. Lower scores from baseline indicated increased developmental delay and higher scores from baseline indicated decreased developmental delay. A positive change from baseline score indicates decreased developmental delay and a negative score indicates increased developmental delay. | Full Analysis Set (FAS): participants who were treated with AT-GTX-501 and had an analyzable outcome (Mullen [Age-Equivalent] Scales Of Early Learning score) at baseline. | Posted | Mean | Standard Deviation | score on a scale | Screening (Day -30 up to -2) and Month 24 |
|
|
|
| Secondary | Change From Baseline In Preschool Language Scales-5th Edition (PLS-5) (Age-Equivalent) At Month 24 | The Preschool Language Scales-5th Edition (PLS-5) (Age-Equivalent) was a comprehensive developmental language assessment. Standard scores from each domain (auditory comprehension, expressive communication, and total language) range from 50-150. Standard scores between 85 and 115 are considered to be within normal limits. Total scores are the sum of standard scores for auditory comprehension and expressive communication, which is then converted to a total standard score using PLS-5 Appendix B. Age-equivalent scores in each area were summarized by study visit with descriptive statistics. Higher scores represent better language development and lower scores reflect a possible language delay or disorder. A positive change from baseline score indicates better language development. | Full Analysis Set (FAS): participants who were treated with AT-GTX-501 and had an analyzable outcome (Preschool Language Scales-5th Edition [PLS-5] [Age-Equivalent] scores) at baseline. | Posted | Mean | Standard Deviation | score on a scale | Screening (Day -30 up to -2) and Month 24 |
|
|
|
| Secondary | Change From Baseline In Development Profile-3 At Month 24 | The Development Profile-3 was used to screen for developmental delays in 5 areas (physical [score range 0-35], adaptive behavior [0-37], social-emotional [0-36], cognitive [0-38], communication [0-34]), where lower scores indicated increased developmental delay. The General Development Score is obtained by adding the sum of standard scores for the five scales together (range 0-180). Standard Score ranges are <70 Delayed, 70-84 Below Average, 85-115 Average, 116-130 Above Average, and >130 Well Above Average. A positive change from baseline score indicates decreased developmental delay and a negative score indicates increased developmental delay. | Full Analysis Set (FAS): participants who were treated with AT-GTX-501 and had an analyzable outcome (Development Profile-3 Score) at baseline and had scores available at Month 24. | Posted | Mean | Standard Deviation | score on a scale | Screening (Day -30 up to -2) and Month 24 |
|
|
|
| 0 |
| 13 |
| 7 |
| 13 |
| 13 |
| 13 |
| Abdominal pain | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Epstein-Barr virus infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA (23.0) | Systematic Assessment |
|
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Myoclonic epilepsy | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Myoclonus | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hallucination | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Cerumen impaction | Ear and labyrinth disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hypothyroidism | Endocrine disorders | MedDRA (23.0) | Systematic Assessment |
|
| Precocious puberty | Endocrine disorders | MedDRA (23.0) | Systematic Assessment |
|
| Astigmatism | Eye disorders | MedDRA (23.0) | Systematic Assessment |
|
| Gaze palsy | Eye disorders | MedDRA (23.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Impaired gastric emptying | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (23.0) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (23.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Candida infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Gastrointestinal viral infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Impetigo | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Molluscum contagiosum | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Pyuria | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Respiratory tract infection viral | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Vaginal infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Viral rash | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Skin laceration | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA (23.0) | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA (23.0) | Systematic Assessment |
|
| Amylase increased | Investigations | MedDRA (23.0) | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA (23.0) | Systematic Assessment |
|
| Blood creatine phosphokinase increased | Investigations | MedDRA (23.0) | Systematic Assessment |
|
| Oxygen saturation decreased | Investigations | MedDRA (23.0) | Systematic Assessment |
|
| Weight decreased | Investigations | MedDRA (23.0) | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (23.0) | Systematic Assessment |
|
| Failure to thrive | Metabolism and nutrition disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hypophagia | Metabolism and nutrition disorders | MedDRA (23.0) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Seizure | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Myoclonic epilepsy | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Atonic seizures | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Dyskinesia | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Myoclonus | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Drooling | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Dystonia | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Partial seizures | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Petit mal epilepsy | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Abnormal behaviour | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hallucination | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Irritability | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Restlessness | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Sleep disorder | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Staring | Psychiatric disorders | MedDRA (23.0) | Systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
|
| Glycosuria | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
|
| Proteinuria | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
|
| Sterile pyuria | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
|
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Umbilical erythema | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
|
| Pallor | Vascular disorders | MedDRA (23.0) | Systematic Assessment |
|
The investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Capability Assuming Normal Vision |
|
| Capability with Actual Vision |
|
| Title | Measurements |
|---|---|
|
| Expressive Language |
|
| Title | Measurements |
|---|---|
|
|
| Social-Emotional |
|
|
| Cognitive |
|
|
| Communication |
|
|
| General Development |
|
|