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This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of EPI-589 therapy on known trajectory.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| EPI-589 | Experimental | Participants will receive EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EPI-589 | Drug | An immediate release film-coated table at a 250 mg dosage strength will be administered per dose and schedule specified in arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Drug-Related Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. | Baseline (Day 0) to Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12) | Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 | |
| Maximum Observed Plasma Concentration (Cmax) | Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Matthew B Klein, MD, FACS | PTC Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cedar's Sinai | Los Angeles | California | 90048 | United States | ||
| California Pacific Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | EPI-589 | Participants received EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Efficacy intent-to-treat (EITT) population or safety population included any participant who received at least 1 dose of EPI-589.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | EPI-589 | Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Drug-Related Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. | Safety population included any participant who received at least 1 dose of EPI-589. | Posted | Count of Participants | Participants | Baseline (Day 0) to Month 6 |
|
Baseline (Day 0) to Month 6
Safety population included any participant who received at least 1 dose of EPI-589.
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 | EPI-589 | Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Aortic valve incompetence | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Patient Advocacy | PTC Therapeutics, Inc. | 1-866-562-4620 | medinfo@ptcbio.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 17, 2017 | Aug 31, 2020 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 20, 2018 | Sep 23, 2020 | SAP_002.pdf |
Not provided
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Change From Baseline in ALSFRS-R Total Score at Month 6 | ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task). | Baseline, Month 6 |
| Change From Baseline in Vital Capacity at Month 6 | Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation. | Baseline, Month 6 |
| Change From Baseline in MIP at Month 6 | MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength. | Baseline, Month 6 |
| Change From Baseline in Respiratory Rate at Month 6 | Respiratory rate is the rate at which breathing occurs. | Baseline, Month 6 |
| Change From Baseline in Heart Rate at Month 6 | The heart rate measures the number of times the heart beats per minute. | Baseline, Month 6 |
| Change From Baseline in SpO2 at Month 6 | SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen. | Baseline, Month 6 |
| Change From Baseline in ETCO2 at Month 6 | ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation. | Baseline, Month 6 |
| Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6 | The failure to thrive as measured by body weight was defined as weight loss of more than 5% from baseline | Baseline, Month 6 |
| Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6 | Participants were observed and timed swallowing water as well as solid foods in accordance with a standardized protocol. | Baseline, Month 6 |
| Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion) | The strength of designated muscle groups was measured using handheld dynamometry. | Baseline, Month 6 |
| Change From Baseline in Number of Words Participant Read at Month 6 | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | Baseline, Month 6 |
| Change From Baseline in Time Spent in Reading at Month 6 | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | Baseline, Month 6 |
| Change From Baseline in Number of Words Per Minute Read at Month 6 | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | Baseline, Month 6 |
| Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | Month 6 |
| Level of Disease-Related Biomarker (Glutathione) in Plasma | Glutathione lowest limit of quantification (LLOQ) = 0.089 micromoles (uM) and upper limit of quantification (ULOQ) = 13.916 uM in plasma. | Baseline up to Month 6 |
| Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF) | Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF. | Baseline up to Month 3 |
| Level of Disease-Related Biomarker (Glutathione) in Urine | Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine. | Baseline up to Month 6 |
| San Francisco |
| California |
| 94115 |
| United States |
| Providence Brain and Spine Institute ALS Center | Portland | Oregon | 97213 | United States |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score | ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in amyotrophic lateral sclerosis (ALS). Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task). | Mean | Standard Deviation | units on a scale |
|
| Vital Capacity | Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation. | Mean | Standard Deviation | percentage of capacity |
|
| Maximum Inspiratory Pressure (MIP) | MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength. | Mean | Standard Deviation | centimetres of water at (cm H2O) |
|
| Respiratory Rate | Respiratory rate is the rate at which breathing occurs. | 'Number analyzed' signifies participants evaluable for specified categories. | Mean | Standard Deviation | breaths/minute |
|
| Heart Rate | The heart rate measures the number of times the heart beats per minute. | 'Number analyzed' signifies participants evaluable for specified categories. | Mean | Standard Deviation | beats/minute |
|
| Oxygen Saturation (SpO2) | SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen. | 'Number analyzed' signifies participants evaluable for specified categories. | Mean | Standard Deviation | percentage of hemoglobin |
|
| End-Tidal Carbon Dioxide (ETCO2) | ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation. | 'Number analyzed' signifies participants evaluable for specified categories. | Mean | Standard Deviation | percentage of CO2 |
|
| Average Solid Swallowing Time | Mean | Standard Deviation | seconds |
|
| Average Water Swallowing Time | Mean | Standard Deviation | seconds |
|
| Muscle Function, as Assessed by Handheld Dynamometry Parameters | Handheld dynamometry parameters included grip strength, shoulder flexion, knee extension, hip flexion, elbow flexion, elbow extension, and ankle dorsi flexion. | 'Number analyzed' signifies participants evaluable for specified categories. | Mean | Standard Deviation | kilograms |
|
| Number of Words Participant Read | Mean | Standard Deviation | Words |
|
| Time Spent in Reading | Mean | Standard Deviation | seconds |
|
| Number of Words Per Minute Read | Mean | Standard Deviation | words/minute |
|
|
|
| Secondary | Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12) | EITT population or safety population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Error | hours*nanograms/milliliter | Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 |
|
|
|
| Secondary | Maximum Observed Plasma Concentration (Cmax) | EITT population or safety population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Error | ng/mL | Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 |
|
|
|
| Secondary | Change From Baseline in ALSFRS-R Total Score at Month 6 | ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task). | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Vital Capacity at Month 6 | Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | percentage of capacity | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in MIP at Month 6 | MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | cm H2O | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Respiratory Rate at Month 6 | Respiratory rate is the rate at which breathing occurs. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | breaths/minute | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Heart Rate at Month 6 | The heart rate measures the number of times the heart beats per minute. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | beats/minute | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in SpO2 at Month 6 | SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | percentage of hemoglobin | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in ETCO2 at Month 6 | ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | percentage of CO2 | Baseline, Month 6 |
|
|
|
| Secondary | Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6 | The failure to thrive as measured by body weight was defined as weight loss of more than 5% from baseline | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Count of Participants | Participants | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6 | Participants were observed and timed swallowing water as well as solid foods in accordance with a standardized protocol. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | seconds | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion) | The strength of designated muscle groups was measured using handheld dynamometry. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | kilograms | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Number of Words Participant Read at Month 6 | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | words | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Time Spent in Reading at Month 6 | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | seconds | Baseline, Month 6 |
|
|
|
| Secondary | Change From Baseline in Number of Words Per Minute Read at Month 6 | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | words/minute | Baseline, Month 6 |
|
|
|
| Secondary | Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility | The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | Posted | Count of Participants | Participants | Month 6 |
|
|
|
| Secondary | Level of Disease-Related Biomarker (Glutathione) in Plasma | Glutathione lowest limit of quantification (LLOQ) = 0.089 micromoles (uM) and upper limit of quantification (ULOQ) = 13.916 uM in plasma. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 6 glutathione in plasma data. | Posted | Mean | Full Range | µM | Baseline up to Month 6 |
|
|
|
| Secondary | Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF) | Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 3 glutathione in CSF data. | Posted | Mean | Full Range | µM | Baseline up to Month 3 |
|
|
|
| Secondary | Level of Disease-Related Biomarker (Glutathione) in Urine | Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine. | EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 6 glutathione in urine data. | Posted | Mean | Full Range | µM | Baseline up to Month 6 |
|
|
|
| 0 |
| 19 |
| 0 |
| 19 |
| 15 |
| 19 |
| Vertigo | Ear and labyrinth disorders | MedDRA 15.0 | Systematic Assessment |
|
| Eye pain | Eye disorders | MedDRA 15.0 | Systematic Assessment |
|
| Vision blurred | Eye disorders | MedDRA 15.0 | Systematic Assessment |
|
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Seasonal allergy | Immune system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Clostridium difficile infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 15.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 15.0 | Systematic Assessment |
|
| Blood pressure increased | Investigations | MedDRA 15.0 | Systematic Assessment |
|
| Blood testosterone decreased | Investigations | MedDRA 15.0 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 15.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Benign hepatic neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
|
| Aura | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Balance disorder | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dysarthria | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Hypokinesia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Tension headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Depression | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
|
| Disorientation | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
|
|
|
| After 10 minutes supine |
|
|
|
| After 10 minutes supine |
|
|
|
| After 10 minutes supine |
|
|
|
| After 10 minutes supine |
|
|
|
|
| Shoulder Flexion - Left |
|
|
| Shoulder Flexion - Right |
|
|
| Knee Extension - Left |
|
|
| Knee Extension - Right |
|
|
| Knee Flexion - Left |
|
|
| Knee Flexion - Right |
|
|
| Hip Flexion - Left |
|
|
| Hip Flexion - Right |
|
|
| Elbow Flexion - Left |
|
|
| Elbow Flexion - Right |
|
|
| Elbow Extension - Left |
|
|
| Elbow Extension - Right |
|
|
| Ankle Dorsi Flexion - Left |
|
|
| Ankle Dorsi Flexion - Right |
|
|
| Title | Measurements |
|---|---|
|