Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2016-000067-16 | EudraCT Number |
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The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy. The pharmacokinetics of tideglusib and its primary metabolite will also be investigated.
Not provided
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 - Tideglusib | Experimental | 1000 mg tideglusib, orally, once daily |
|
| Cohort 2 - Tideglusib | Experimental | 400 mg tideglusib, orally, once daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tideglusib | Drug | Tideglusib for oral suspension, |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety (Adverse Events) | Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study. | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Concentration of Tideglusib | Pharmacokinetic samples were collected to determine Tideglusib plasma concentration | 12 weeks |
| Blood Pharmacokinetics of Tideglusib | Pharmacokinetic samples were collected to determine time of the maximum plasma concentration and terminal elimination half-life of tideglusib |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
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| Name | Affiliation | Role |
|---|---|---|
| Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP | Institute of Neuroscience, Newcastle University. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Newcastle-upon-Tyne Hospitals NHS Trust | Newcastle upon Tyne | Tyne and Wear | NE1 4LP | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32942085 | Derived | Horrigan J, Gomes TB, Snape M, Nikolenko N, McMorn A, Evans S, Yaroshinsky A, Della Pasqua O, Oosterholt S, Lochmuller H. A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1). Pediatr Neurol. 2020 Nov;112:84-93. doi: 10.1016/j.pediatrneurol.2020.08.001. Epub 2020 Aug 5. |
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Subjects were to be male or female aged 12 to 45 years with a diagnosis of genetically confirmed congenital or juvenile-onset type 1 myotonic dystrophy. Subjects were to have a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2) and were to be ambulatory and able to complete the 10-metre walk/run test.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 | Subjects were given tideglusib 1000 mg for 12 weeks (weeks 0 to 12) |
| FG001 | Cohort 2 | Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12) |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 | 1000 mg tideglusib |
| BG001 | Cohort 2 | 400 mg tideglusib |
| BG002 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | 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 | Safety (Adverse Events) | Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study. | Safety Analysis Set | Posted | Count of Participants | Participants | 12 weeks |
|
Consent to end of study, up to 14 weeks
SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
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 | 1000 mg tideglusib | 0 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Back injury | Injury, poisoning and procedural complications | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Mike Snape, Chief Scientific Officer | AMO Pharma Ltd. | +44 (0) 7775915639 | mike.snape@amo-pharma.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 | Jan 9, 2017 | Jun 5, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 1, 2017 | Jun 5, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C520571 | tideglusib |
Not provided
Not provided
Not provided
Not provided
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Not provided
Not provided
| 12 weeks |
| Area Under the Plasma Concentration vs. Time Curve of Tideglusib | Pharmacokinetic samples were collected to determine area under the plasma concentration vs. time curve from 0 to 12 h and area under the plasma concentration vs. time curve from 0 to 24 h of tideglusib | 12 weeks |
| 10 Metre Walk/Run Test | The 10-metre walk/run test is a performance measure used to assess walking speed in metres per second over a short distance and was used as an assessment of functional mobility. Time taken to complete the 10m walk/run test at fastest and preferred speed is measured. | 12 weeks |
| Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time | Handgrip myometry is used as a measure of myotonia and muscle strength for the dominant hand | 12 weeks |
| Respiratory Forced Vital Capacity (FVC) | FVC is a measure of lung function (the total amount of air exhaled during a Forced Expiratory Volume is measured using a spirometer) | 12 weeks |
| Dual-energy X-ray Absorptiometry (DXA) | DXA utilises two low energy X-ray beams, with different energy levels, which are aimed at the subject's bones. The DXA scan is more typically used to measure bone mineral density, however it can also be used to measure total lean muscle mass | 12 weeks |
| Clinical Global Impressions- Severity (CGI-S) | The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. Change in CGI-S was observed in only 1 subject. Consequently statistical analysis was not conducted. | 12 weeks |
| Clinical Global Impressions- Improvement (CGI-I) | The CGI-I requires the clinician to rate how much the subject's illness has improved or worsened relative to a baseline state. A seven point Likert type scale is used from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse. | 12 weeks |
| Actigraphy (3-minute Bouts of Activity) | Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual. | 12 weeks |
| Actigraphy (>10-minute Bouts of Activity) | Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual. | 12 weeks |
| Actigraphy (Steps) | Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual. | 12 weeks |
| Nine Hole Peg Test (NHPT) | Measure of fine manual dexterity. Time to taken to complete the NHPT (dominant hand) is recorded. | 12 weeks |
| Top 3 Concerns Visual Analogue Scale (VAS) Score | The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study. Subjects, where possible, and caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long visual analogue scale with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). A score for each concern was to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 3 concerns (minimum = 0 cm, maximum = 30 cm). A higher score represents a worse outcome. | 12 weeks |
| Ohio State University (OSU) Autism Rating Scale (OARS) | The OARS-4 contains the autism signs and symptoms in the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. These were to be rated with the degree of impairment the subject experiences for the given symptom. The symptoms were to be elicited in a semi-structured interview with the subject's primary caregiver. The assessor was to take both frequency/duration and degree of impairment into account and how much the item interferes with relationships, learning, and/or activities of daily living. The scores for this assessment were from 0 (Never or Rarely; Not a Problem) to 3 (Very Often; A Severe Problem): a score for each symptom of social impairment, communication impairment and restricted patterns were averaged to provide a total impairment score. A higher score represents a worse outcome (minimum = 0, maximum 3). | 12 weeks |
| Ohio State University (OSU) Autism Clinical Global Impression (CGI) | The OSU Autism CGI scale contains separate subscales for symptom severity and for global improvement. These are rated in a similar way to the National Institute of Mental Health (NIMH) CGI Severity scale, but it is focused on autism spectrum symptoms. OSU Autism CGI-severity and OSU Autism CGI-improvement scores use a seven point Likert rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse. | 12 weeks |
| Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy | The Clinician-completed Domain Specific Causes for Concern is a Visual Analogue Scale completed by the clinician that scores the severity of concerns of domains that are clinically relevant in myotonic dystrophy. The severity of the clinician's concern is scored by using a 10 cm visual analogue scale (VAS), with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). The clinician is asked to make a vertical line indicating his/her level of concern in each domain, using a time frame of the past week for reference. A score is to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 17 domains (minimum = 0, maximum = 170 cm). A higher score represents a worse outcome. | 12 weeks |
| Peabody Picture Vocabulary Test (PPVT) | The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary of children and adults. It contains training items and 228 test items, each consisting of four full-colour pictures as response options on a page. For each item, the examiner says a word, and the examinee responds by selecting the picture that best illustrates that word's meaning. Each administration of the test produces a raw score (number of test items answered correctly), which can be converted to a standard score (using age-based norms) with a mean of 100 and a standard deviation of 15. Higher scores mean a better performance/receptive vocabulary. | 12 weeks |
| Biomarker - Lymphocyte GSK3β Levels and Activity | Total levels of GSK3β protein was determined via x-MAP technology in a Luminex 200 platform using the AKT Pathway Total Multispecies 7-Plex Panel from ThermoFisher Scientific. | 12 weeks |
| Total |
Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Secondary | Plasma Concentration of Tideglusib | Pharmacokinetic samples were collected to determine Tideglusib plasma concentration | Full Analysis Set | Posted | Mean | Standard Deviation | ng/mL | 12 weeks |
|
|
|
| Secondary | Blood Pharmacokinetics of Tideglusib | Pharmacokinetic samples were collected to determine time of the maximum plasma concentration and terminal elimination half-life of tideglusib | Full Analysis Set | Posted | Mean | Standard Deviation | h | 12 weeks |
|
|
|
| Secondary | Area Under the Plasma Concentration vs. Time Curve of Tideglusib | Pharmacokinetic samples were collected to determine area under the plasma concentration vs. time curve from 0 to 12 h and area under the plasma concentration vs. time curve from 0 to 24 h of tideglusib | Full Analysis Set | Posted | Mean | Standard Deviation | ng/mL.h | 12 weeks |
|
|
|
| Secondary | 10 Metre Walk/Run Test | The 10-metre walk/run test is a performance measure used to assess walking speed in metres per second over a short distance and was used as an assessment of functional mobility. Time taken to complete the 10m walk/run test at fastest and preferred speed is measured. | Full Analysis Set | Posted | Mean | Standard Deviation | seconds | 12 weeks |
|
|
|
|
| Secondary | Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time | Handgrip myometry is used as a measure of myotonia and muscle strength for the dominant hand | Full Analysis Set | Posted | Mean | Standard Deviation | kg | 12 weeks |
|
|
|
|
| Secondary | Respiratory Forced Vital Capacity (FVC) | FVC is a measure of lung function (the total amount of air exhaled during a Forced Expiratory Volume is measured using a spirometer) | Full Analysis Set | Posted | Mean | Standard Deviation | litres | 12 weeks |
|
|
|
|
| Secondary | Dual-energy X-ray Absorptiometry (DXA) | DXA utilises two low energy X-ray beams, with different energy levels, which are aimed at the subject's bones. The DXA scan is more typically used to measure bone mineral density, however it can also be used to measure total lean muscle mass | Full Analysis Set | Posted | Mean | Standard Deviation | gram | 12 weeks |
|
|
|
|
| Secondary | Clinical Global Impressions- Severity (CGI-S) | The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. Change in CGI-S was observed in only 1 subject. Consequently statistical analysis was not conducted. | Full Analysis Set | Posted | Mean | Standard Deviation | Values on a scale | 12 weeks |
|
|
|
| Secondary | Clinical Global Impressions- Improvement (CGI-I) | The CGI-I requires the clinician to rate how much the subject's illness has improved or worsened relative to a baseline state. A seven point Likert type scale is used from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse. | Full Analysis Set | Posted | Mean | Standard Deviation | Scores on a scale | 12 weeks |
|
|
|
|
| Secondary | Actigraphy (3-minute Bouts of Activity) | Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual. | Full Analysis Set | Posted | Mean | Standard Deviation | 3 minute bouts of activity per hour | 12 weeks |
|
|
|
|
| Secondary | Actigraphy (>10-minute Bouts of Activity) | Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual. | Full Analysis Set | Posted | Mean | Standard Deviation | 10 minute bouts of activity per hour | 12 weeks |
|
|
|
| Secondary | Actigraphy (Steps) | Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual. | Full Analysis Set | Posted | Mean | Standard Deviation | steps per hour wear time | 12 weeks |
|
|
|
| Secondary | Nine Hole Peg Test (NHPT) | Measure of fine manual dexterity. Time to taken to complete the NHPT (dominant hand) is recorded. | Full Analysis Set | Posted | Mean | Standard Deviation | seconds | 12 weeks |
|
|
|
|
| Secondary | Top 3 Concerns Visual Analogue Scale (VAS) Score | The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study. Subjects, where possible, and caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long visual analogue scale with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). A score for each concern was to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 3 concerns (minimum = 0 cm, maximum = 30 cm). A higher score represents a worse outcome. | Full Analysis Set | Posted | Mean | Standard Deviation | Score on a scale | 12 weeks |
|
|
|
|
| Secondary | Ohio State University (OSU) Autism Rating Scale (OARS) | The OARS-4 contains the autism signs and symptoms in the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. These were to be rated with the degree of impairment the subject experiences for the given symptom. The symptoms were to be elicited in a semi-structured interview with the subject's primary caregiver. The assessor was to take both frequency/duration and degree of impairment into account and how much the item interferes with relationships, learning, and/or activities of daily living. The scores for this assessment were from 0 (Never or Rarely; Not a Problem) to 3 (Very Often; A Severe Problem): a score for each symptom of social impairment, communication impairment and restricted patterns were averaged to provide a total impairment score. A higher score represents a worse outcome (minimum = 0, maximum 3). | Full Analysis Set | Posted | Mean | Standard Deviation | Units on a scale | 12 weeks |
|
|
|
|
| Secondary | Ohio State University (OSU) Autism Clinical Global Impression (CGI) | The OSU Autism CGI scale contains separate subscales for symptom severity and for global improvement. These are rated in a similar way to the National Institute of Mental Health (NIMH) CGI Severity scale, but it is focused on autism spectrum symptoms. OSU Autism CGI-severity and OSU Autism CGI-improvement scores use a seven point Likert rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse. | Full Analysis Set | Posted | Mean | Standard Deviation | Units on a scale | 12 weeks |
|
|
|
|
| Secondary | Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy | The Clinician-completed Domain Specific Causes for Concern is a Visual Analogue Scale completed by the clinician that scores the severity of concerns of domains that are clinically relevant in myotonic dystrophy. The severity of the clinician's concern is scored by using a 10 cm visual analogue scale (VAS), with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). The clinician is asked to make a vertical line indicating his/her level of concern in each domain, using a time frame of the past week for reference. A score is to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 17 domains (minimum = 0, maximum = 170 cm). A higher score represents a worse outcome. | Full Analysis Set | Posted | Mean | Standard Deviation | Score on a scale | 12 weeks |
|
|
|
|
| Secondary | Peabody Picture Vocabulary Test (PPVT) | The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary of children and adults. It contains training items and 228 test items, each consisting of four full-colour pictures as response options on a page. For each item, the examiner says a word, and the examinee responds by selecting the picture that best illustrates that word's meaning. Each administration of the test produces a raw score (number of test items answered correctly), which can be converted to a standard score (using age-based norms) with a mean of 100 and a standard deviation of 15. Higher scores mean a better performance/receptive vocabulary. | Full Analysis Set | Posted | Mean | Standard Deviation | Units on a scale | 12 weeks |
|
|
|
|
| Secondary | Biomarker - Lymphocyte GSK3β Levels and Activity | Total levels of GSK3β protein was determined via x-MAP technology in a Luminex 200 platform using the AKT Pathway Total Multispecies 7-Plex Panel from ThermoFisher Scientific. | Full Analysis Set | Posted | Mean | Standard Deviation | microgram per microliter | 12 weeks |
|
|
|
| 8 |
| 0 |
| 8 |
| 8 |
| 8 |
| EG001 | Cohort 2 | 400 mg tideglusib | 0 | 8 | 0 | 8 | 6 | 8 |
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Joint dislocation | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Limb injury | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Nervous system cyst | Nervous system disorders | Systematic Assessment |
|
| Somnolence | Nervous system disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
|
| Post-tussive vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Dysmenorrhea | Reproductive system and breast disorders | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Rash papular | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Axillary mass | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Vitamin D deficiency | Metabolism and nutrition disorders | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | Systematic Assessment |
|
| Subcutaneous abscess | Infections and infestations | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | Systematic Assessment |
|
The investigator has the right to publish study results from his/her specific site. However, any publication that includes AMO Pharma confidential information cannot be submitted for publication without AMO Pharma's prior written approval.
| D020967 | Myotonic Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Steady-state concentration (CSS) |
|
| Fastest speed (observed value at week 12) |
|
| Fastest speed (change from baseline at week 12) |
|
| Change from baseline to week 12 in the time taken (seconds) to complete 10-metre walk/run test at the preferred speed | Mixed Models Analysis | 0.0565 | Mean Difference (Net) | -0.6449 | 2-Sided | 95 | -1.3094 | 0.0195 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in grip strength (kg) | Mixed Models Analysis | 0.1782 | Mean Difference (Net) | -1.3897 | 2-Sided | 95 | -3.4525 | 0.6731 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in FVC (litres) | Mixed Models Analysis | 0.9204 | Mean Difference (Net) | 0.0152 | 2-Sided | 95 | -0.3003 | 0.3307 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Legs (observed value at week 12) |
|
| Legs (change from baseline at week 12) |
|
| Total (observed value at week 12) |
|
| Total (change from baseline at week 12) |
|
|
Changes from baseline to week 12 in the DXA scan total lean muscle mass (g) |
| ANCOVA |
| 0.3125 |
| Median Difference (Net) |
| 426.75 |
| 2-Sided |
| 95 |
| -289.50 |
| 1198.50 |
Hodges-Lehmann estimates for the median and confidence intervals are presented |
| Other |
The exact Wilcoxon signed rank test was used to test for a change in the DXA scan total lean muscle mass (g) from baseline to end of treatment |
| Observed value at week 12 in Clinical Global Impression Global Improvement Scale (CGI-I) | Mixed Models Analysis | 0.0009 | The p-value is presented for the comparison of adjusted Least Square Means to a score of 4, representing 'No change' | Mean Difference (Net) | 3.0 | 2-Sided | 95 | 2.4 | 3.6 | Other | A mixed effect model repeated measure (MMRM) was fitted to the observed values at week12. The model included dose group and visit as fixed effects, and the treatment-by-visit interaction. F-tests from PROC MIXED were based on Kenward-Roger's adjusted degrees of freedom. |
| Change from baseline to week 12 in the weekly total number of 3 minute bouts of activity per hour wear time | Mixed Models Analysis | 0.0054 | Mean Difference (Net) | -0.325 | 2-Sided | 95 | -0.549 | -0.102 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in the time taken (seconds) to complete the nine hole peg test for the dominant arm | Mixed Models Analysis | 0.2088 | Mean Difference (Net) | -0.955 | 2-Sided | 95 | -2.525 | 0.614 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Caregiver Top 3 Concerns VAS total score (observed value at week 12) |
|
| Caregiver Top 3 Concerns VAS total score (change from baseline at week 12) |
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| Change from baseline to week 12 in the subject top three concerns VAS total score (cm) | Mixed Models Analysis | 0.0456 | Mean Difference (Net) | -1.99 | 2-Sided | 95 | -3.94 | -0.04 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in the caregiver top three concerns VAS total score (cm) | Mixed Models Analysis | 0.0058 | Mean Difference (Net) | -2.41 | 2-Sided | 95 | -4.03 | -0.80 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in the caregiver top three concerns VAS total score (cm) | Mixed Models Analysis | 0.0208 | Mean Difference (Net) | -1.95 | 2-Sided | 95 | -3.56 | -0.34 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in the OSU Autism Rating Scale - DSM-IV (OARS-4) total impairment mean | Mixed Models Analysis | 0.6090 | Mean Difference (Net) | -0.009 | 2-Sided | 95 | -0.047 | 0.028 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| OSU autism CGI-Improvement (observed value at week 12) |
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| Observed value at week 12 in OSU global improvement scale for autism (OSU CGI-I) | Mixed Models Analysis | 1.0000 | p-values are presented for the comparison of adjusted Least Square Means to a score of 4, representing 'No change' | Mean Difference (Net) | 4.0 | 2-Sided | 95 | 3.6 | 4.4 | Other | A mixed effect model repeated measure (MMRM) was fitted to the observed values at week12 in OSU CGI-I. The model included dose group and visit as fixed effects, and the treatment-by-visit interaction. F-tests from PROC MIXED were based on Kenward-Roger's adjusted degrees of freedom. A compound symmetry covariance matrix was used for the repeated visits within subject. |
| Change from baseline at week 12 in the clinician-completed domain specific causes for concern VAS total score (cm) | Mixed Models Analysis | 0.0116 | Mean Difference (Net) | -3.27 | 2-Sided | 95 | -5.71 | -0.83 | The mean difference is an adjusted Least Square Mean | Other | Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values. |
| Change from baseline to week 12 in the peabody picture vocabulary test age-based standard score | ANCOVA | 0.9546 | Mean Difference (Net) | -0.1 | 2-Sided | 95 | -5.5 | 5.2 | The mean difference is an adjusted Least Square Mean | Other | An ANCOVA was fitted to the change in the Peabody Picture Vocabulary Test age-based standard score from baseline to end of treatment. The model included the baseline value as a covariate and dose group as a fixed effect. |