Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| UX111-CL201 | Other Identifier | Ultragenyx Pharmaceutical Inc | |
| 2018-000504-42 | EudraCT Number |
Not provided
Not provided
Not provided
Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
Not provided
Not provided
| Name | Class |
|---|---|
| Abeona Therapeutics, Inc | INDUSTRY |
Not provided
Not provided
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Not provided
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
This is an open-label, single dose clinical trial. All participants will receive 3 X 10^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ABO-102 | Experimental | Dose of 3x10^13 vg/kg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ABO-102 | Drug | Single dose of ABO-102 (scAAV9.U1a.hSGSH) administered by intravenous injection through a peripheral limb vein at a dose of 3 X 10^13 vg/kg |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely. | From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12 |
| Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame | An SAE is defined as any untoward medical occurrence that, at any dose:
Relationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely. | From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months) |
| Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment | As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight.
| Baseline, Day 30, 180, Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Plasma Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Change From Baseline in Urine Glycosaminoglycans After Treatment | Baseline, Day 30, Day 180, Month 12 |
Not provided
Inclusion Criteria:
Diagnosis of MPS IIIA confirmed by the following methods:
Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)
Must be ambulatory, though may receive assistance with ambulation
Age range of 2 years up to 18 years (excluded)
Exclusion Criteria:
Not provided
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States | ||
| Adelaide Women's and Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27331076 | Background | Fu H, Cataldi MP, Ware TA, Zaraspe K, Meadows AS, Murrey DA, McCarty DM. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery. Mol Ther Methods Clin Dev. 2016 Jun 8;3:16036. doi: 10.1038/mtm.2016.36. eCollection 2016. | |
| 29064732 | Background |
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| ID | Title | Description |
|---|---|---|
| FG000 | scAAV9.U1a.hSGSH, 3x1013 vg/kg | An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | scAAV9.U1a.hSGSH, 3x1013 vg/kg | An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg |
| 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, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely. | Posted | Number | participants | From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12 |
|
All-Cause Mortality: From enrollment to the end of study; mean of 18 months. Adverse Events: from first dose of study drug up to Day 454.
Not provided
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 | scAAV9.U1a.hSGSH, 3x1013 vg/kg | An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dysphagia | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8567 | medinfo@ultragenyx.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 | Sep 22, 2022 | Jun 9, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 15, 2022 | Jun 9, 2023 | SAP_001.pdf |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D009084 | Mucopolysaccharidosis III |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
Not provided
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| Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment |
Change from baseline in CSF heparan sulfate levels after treatment |
| Baseline, Day 30, Day 180, Month 12 |
| Change From Baseline in Urine Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 |
| Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment | Baseline, Day 30, Day 180, and Month 12 |
| Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment | Baseline, Day 30, Day 180, Month 12 |
| Change From Baseline in Plasma SGSH After Treatment | Baseline, Day 30, Day 180, Month 12 |
| Change From Baseline in Brain Volumes After Treatment | As measured by MRI | Baseline, 12 months |
| Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness | As measured by MRI | Baseline, 12 months |
| Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment | CSHQ is a caregiver-completed, 35-item questionnaire that assesses the frequency of behaviors associated with common pediatric sleep difficulties. Eight domains of sleep, including Bedtime Resistance, Sleep Onset Delay, Sleep Duration, Sleep Anxiety, Night Awakenings, Parasomnias, Sleep Disordered Breathing, and Daytime Sleepiness are assessed, producing eight individual subdomain scores and an overall CSHQ subscore total. CSHQ total score is calculated by adding all the 8 subscores, and ranges from 36 to 108. A higher score is indicative of more disturbed sleep. | Baseline, Day 180, Month 12 |
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score | PedsQL is a brief measure of health-related quality of life in children. The Peds QL Generic Core Scales was used in the study, consisting of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic conditions. The four scales include Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Item scores are added together and averaged to produce a Core total score, where higher scores on a scale of 0-100 indicate better Health-related Quality of Life (HRQOL). | Baseline, Day 180, Month 12 |
| Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score | The Parenting Stress Index, 4th Edition evaluates the magnitude and type of stress in a parent/child relationship. The short form version was used in the study, consisting of 36 items divided into three domains: Parental Distress (PD), Parent-Child Dysfunctional Interaction (P-CDI), and Difficult Child (DC), which combine together to form a Total Stress raw score. Total Stress raw scores can range from 36 - 180, with higher raw scores indicating higher levels of stress. | Baseline, Day 180, Month 12 |
| Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score | The PedsQL Gastrointestinal Symptoms Scale is a specific module of the PedsQL that measures gastrointestinal symptoms in patients with acute and chronic health conditions as well as healthy school and community populations. The Parent Report version was used on the study, consisting of 58 items across 10 dimensions, assessing parents' perceptions of their child's GI-specific symptoms. Item scores are added together and averaged to produce a GI symptoms scales score, where higher scores on a scale of 0-100 indicate better GI specific QOL. | Baseline, Day 180, Month 12 |
| Change From Baseline in Parent Global Impression (PGI) Total Score | The Parent Global Impression scale evaluates all aspects of a patients' health and assesses if there has been an improvement or decline in clinical status, as reported by the parent/caregiver. This study used a modified version with symptoms relevant to the patient population in the trial. Nine symptoms were scored at each visit, using a 7-point rating scale where 3 = much better, 2 = better, 1 = slightly better, 0 = same, -1 = slightly worse, -2 = worse, and -3 = much worse. The nine symptom scores are added together to produce a PGI total score, ranging from -27 to 27. | Baseline, Day 180, Month 12 |
| Clinical Global Impression Improvement Scale at Day 180 and Month 12 | The Clinical Global Impression of Improvement scale is a brief, stand-alone assessment of the clinician's view of the patient's global functioning prior to and after initiating a study medication, specifically looking at whether the patient has demonstrated improvement or not. Assessment of improvement was scored at each visit, using a 7-point rating scale where 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. | Day 180, Month 12 |
| Change From Baseline in Parent Symptoms Score Questionnaire | The Parent Symptoms Score Questionnaire contains 29 symptoms with an indicator of whether the symptom was present or absent. | Baseline, Day 180, Month 12 |
| Percent Change From Baseline in Body Mass Index After Treatment | Baseline, Day 30, Day 180, Month 12 |
| Number of Participants With Abnormalities in Standard Awake 45-Minutes-Electroencephalogram (EEG) Monitoring at Baseline and Day 180 | NCS=not clinically significant CS=clinically significant | Baseline, Day 180 |
| Change From Baseline in Vector Shedding Analysis in Plasma, Saliva, Stool and Urine | Detection of the adeno-associated Virus 9 (AAV9) viral deoxyribonucleic acid (DNA) in plasma, saliva, urine and feces was analyzed. Per protocol, data were not collected for urine at Month 12. | Baseline, Day 30, Day 180, Month 12 |
| North Adelaide |
| South Australia |
| 5006 |
| Australia |
| Hospital Clínico Universitario de Santiago | Santiago de Compostela | 15706 | Spain |
| Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24. |
| months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Primary | Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame | An SAE is defined as any untoward medical occurrence that, at any dose:
Relationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely. | Posted | Count of Participants | Participants | From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months) |
|
|
|
| Primary | Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment | As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight.
| Participants with an assessment at baseline and given time point | Posted | Mean | Standard Deviation | ratio | Baseline, Day 30, 180, Month 12 |
|
|
|
| Primary | Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment | Change from baseline in CSF heparan sulfate levels after treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | nmol/mL | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Plasma Heparan Sulfate After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | pmol/mL | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Urine Glycosaminoglycans After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | mg/mmol creatinine | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Urine Heparan Sulfate After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | μmol/mmol creatinine | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | nmol/17 h/mL | Baseline, Day 30, Day 180, and Month 12 |
|
|
|
| Secondary | Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | nmol/17 h/mL | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Plasma SGSH After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | nmol/17 h/mL | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Brain Volumes After Treatment | As measured by MRI | Participants with an assessment | Posted | Mean | Standard Deviation | mL | Baseline, 12 months |
|
|
|
| Secondary | Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness | As measured by MRI | Participants with an assessment | Posted | Mean | Standard Deviation | mm | Baseline, 12 months |
|
|
|
| Secondary | Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment | CSHQ is a caregiver-completed, 35-item questionnaire that assesses the frequency of behaviors associated with common pediatric sleep difficulties. Eight domains of sleep, including Bedtime Resistance, Sleep Onset Delay, Sleep Duration, Sleep Anxiety, Night Awakenings, Parasomnias, Sleep Disordered Breathing, and Daytime Sleepiness are assessed, producing eight individual subdomain scores and an overall CSHQ subscore total. CSHQ total score is calculated by adding all the 8 subscores, and ranges from 36 to 108. A higher score is indicative of more disturbed sleep. | participants with an assessment at given time point | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score | PedsQL is a brief measure of health-related quality of life in children. The Peds QL Generic Core Scales was used in the study, consisting of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic conditions. The four scales include Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Item scores are added together and averaged to produce a Core total score, where higher scores on a scale of 0-100 indicate better Health-related Quality of Life (HRQOL). | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score | The Parenting Stress Index, 4th Edition evaluates the magnitude and type of stress in a parent/child relationship. The short form version was used in the study, consisting of 36 items divided into three domains: Parental Distress (PD), Parent-Child Dysfunctional Interaction (P-CDI), and Difficult Child (DC), which combine together to form a Total Stress raw score. Total Stress raw scores can range from 36 - 180, with higher raw scores indicating higher levels of stress. | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score | The PedsQL Gastrointestinal Symptoms Scale is a specific module of the PedsQL that measures gastrointestinal symptoms in patients with acute and chronic health conditions as well as healthy school and community populations. The Parent Report version was used on the study, consisting of 58 items across 10 dimensions, assessing parents' perceptions of their child's GI-specific symptoms. Item scores are added together and averaged to produce a GI symptoms scales score, where higher scores on a scale of 0-100 indicate better GI specific QOL. | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Parent Global Impression (PGI) Total Score | The Parent Global Impression scale evaluates all aspects of a patients' health and assesses if there has been an improvement or decline in clinical status, as reported by the parent/caregiver. This study used a modified version with symptoms relevant to the patient population in the trial. Nine symptoms were scored at each visit, using a 7-point rating scale where 3 = much better, 2 = better, 1 = slightly better, 0 = same, -1 = slightly worse, -2 = worse, and -3 = much worse. The nine symptom scores are added together to produce a PGI total score, ranging from -27 to 27. | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 180, Month 12 |
|
|
|
| Secondary | Clinical Global Impression Improvement Scale at Day 180 and Month 12 | The Clinical Global Impression of Improvement scale is a brief, stand-alone assessment of the clinician's view of the patient's global functioning prior to and after initiating a study medication, specifically looking at whether the patient has demonstrated improvement or not. Assessment of improvement was scored at each visit, using a 7-point rating scale where 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. | Participants with an assessment at given time point | Posted | Count of Participants | Participants | Day 180, Month 12 |
|
|
|
| Secondary | Change From Baseline in Parent Symptoms Score Questionnaire | The Parent Symptoms Score Questionnaire contains 29 symptoms with an indicator of whether the symptom was present or absent. | Due to the inconsistent use of forms across visits and sites for this study, the data collected for this endpoint was incomplete, unreliable, and could not be evaluated. | Posted | Baseline, Day 180, Month 12 |
|
|
| Secondary | Percent Change From Baseline in Body Mass Index After Treatment | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | percent change | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| Secondary | Number of Participants With Abnormalities in Standard Awake 45-Minutes-Electroencephalogram (EEG) Monitoring at Baseline and Day 180 | NCS=not clinically significant CS=clinically significant | Participants with an assessment | Posted | Count of Participants | Participants | Baseline, Day 180 |
|
|
|
| Secondary | Change From Baseline in Vector Shedding Analysis in Plasma, Saliva, Stool and Urine | Detection of the adeno-associated Virus 9 (AAV9) viral deoxyribonucleic acid (DNA) in plasma, saliva, urine and feces was analyzed. Per protocol, data were not collected for urine at Month 12. | Participants with an assessment at given time point | Posted | Mean | Standard Deviation | Copies per 1 mL of Specimen (copies/mL) | Baseline, Day 30, Day 180, Month 12 |
|
|
|
| 0 |
| 5 |
| 3 |
| 5 |
| 5 |
| 5 |
| Gait disturbance | General disorders | MedDRA 21.1 | Systematic Assessment |
|
| Foreign body in gastrointestinal tract | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
|
| Cognitive disorder | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Mental status changes | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
|
| Gastrostomy | Surgical and medical procedures | MedDRA 21.1 | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Mixed deafness | Ear and labyrinth disorders | MedDRA 21.1 | Systematic Assessment |
|
| Cushingoid | Endocrine disorders | MedDRA 21.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
|
| Gait disturbance | General disorders | MedDRA 21.1 | Systematic Assessment |
|
| Cellulitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Oral candidiasis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Amylase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Blood creatine phosphokinase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Blood lactate dehydrogenase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Gamma-glutamyltransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Haematocrit increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Haemoglobin increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Platelet count decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Red blood cell count increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Weight decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Weight increased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
|
| Increased appetite | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
|
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
| Myositis | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
| Synovial cyst | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
| Ataxia | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Cognitive disorder | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Dyskinesia | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Epilepsy | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Fine motor skill dysfunction | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Seizure | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
|
| Abnormal behaviour | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
|
| Agitation | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
|
| Initial insomnia | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
|
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
|
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Title | Measurements |
|---|---|
|
| 60 - < 90 days : Unlikely; Mild |
|
| 60 - < 90 days : Unlikely; Moderate |
|
| 60 - < 90 days : Unlikely; Severe |
|
| 60 - < 90 days : Possible; Mild |
|
| 60 - < 90 days : Possible; Moderate |
|
| 60 - < 90 days : Possible; Severe |
|
| 60 - < 90 days : Probable; Mild |
|
| 60 - < 90 days : Probable; Moderate |
|
| 60 - < 90 days : Probable; Severe |
|
| 60 - < 90 days : Definite; Mild |
|
| 60 - < 90 days : Definite; Moderate |
|
| 60 - < 90 days : Definite; Severe |
|
| 90 - < 180 days : Unrelated; Mild |
|
| 90 - < 180 days : Unrelated; Moderate |
|
| 90 - < 180 days : Unrelated; Severe |
|
| 90 - < 180 days : Unlikely; Mild |
|
| 90 - < 180 days : Unlikely; Moderate |
|
| 90 - < 180 days : Unlikely; Severe |
|
| 90 - < 180 days : Possible; Mild |
|
| 90 - < 180 days : Possible; Moderate |
|
| 90 - < 180 days : Possible; Severe |
|
| 90 - < 180 days : Probable; Mild |
|
| 90 - < 180 days : Probable; Moderate |
|
| 90 - < 180 days : Probable; Severe |
|
| 90 - < 180 days : Definite; Mild |
|
| 90 - < 180 days : Definite; Moderate |
|
| 90 - < 180 days : Definite; Severe |
|
| 180 days - < 12 months : Unrelated; Mild |
|
| 180 days - < 12 months : Unrelated; Moderate |
|
| 180 days - < 12 months : Unrelated; Severe |
|
| 180 days - < 12 months : Unlikely; Mild |
|
| 180 days - < 12 months : Unlikely; Moderate |
|
| 180 days - < 12 months : Unlikely; Severe |
|
| 180 days - < 12 months : Possible; Mild |
|
| 180 days - < 12 months : Possible; Moderate |
|
| 180 days - < 12 months : Possible; Severe |
|
| 180 days - < 12 months : Probable; Mild |
|
| 180 days - < 12 months : Probable; Moderate |
|
| 180 days - < 12 months : Probable; Severe |
|
| 180 days - < 12 months : Definite; Mild |
|
| 180 days - < 12 months : Definite; Moderate |
|
| 180 days - < 12 months : Definite; Severe |
|
| >= 12 months : Unrelated; Mild |
|
| >= 12 months : Unrelated; Moderate |
|
| >= 12 months : Unrelated; Severe |
|
| >= 12 months : Unlikely; Mild |
|
| >= 12 months : Unlikely; Moderate |
|
| >= 12 months : Unlikely; Severe |
|
| >= 12 months : Possible; Mild |
|
| >= 12 months : Possible; Moderate |
|
| >= 12 months : Possible; Severe |
|
| >= 12 months : Probable; Mild |
|
| >= 12 months : Probable; Moderate |
|
| >= 12 months : Probable; Severe |
|
| >= 12 months : Definite; Mild |
|
| >= 12 months : Definite; Moderate |
|
| >= 12 months : Definite; Severe |
|
|
| Liver volume: change from BL to Month 12 |
|
|
| Spleen volume: change from BL to Day 30 |
|
|
| Spleen volume: change from BL to Day 180 |
|
|
| Spleen volume: change from BL to Month 12 |
|
|
|
| Change from BL to Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
|
| Change in total cerebellar gray matter volume |
|
| Change in total cerebellar white matter volume |
|
| Change in total brain volume |
|
| Change in total cerebral volume |
|
| Change in total cerebral gray matter volume |
|
| Change in total cortical volume |
|
| Change in total cerebellar volume |
|
| Change in total cerebral white matter volume |
|
| Change in total gray matter volume |
|
| Change in total intercranial volume |
|
| Change in total white matter volume |
|
| Change in ventricular volumes |
|
|
|
|
|
|
| 2 = much improved |
|
| 3 = minimally improved |
|
| 4 = no change |
|
| 5 = minimally worse |
|
| 6 = much worse |
|
| 7 = very much worse |
|
| Global Improvement, Month 12 |
|
|
|
| Change from Baseline to Month 12 |
|
|
| Overall EEG : Day 180 |
|
|
| Plasma Change from Baseline to Month 12 |
|
|
| Saliva Change from Baseline to Day 30 |
|
|
| Saliva Change from Baseline to Day 180 |
|
|
| Saliva Change from Baseline to Month 12 |
|
|
| Stool Change from Baseline to Day 30 |
|
|
| Stool Change from Baseline to Day 180 |
|
|
| Stool Change from Baseline to Month 12 |
|
|
| Urine Change from Baseline to Day 30 |
|
|
| Urine Change from Baseline to Day 180 |
|
|