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This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.
In nonsense mutation DMD (nmDMD), early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. This study included a 4-week screening period, a 52-week treatment period (the first 4 weeks of which included PK parameters), and a 4-week follow-up period for participants who completed the treatment period (60 weeks total duration). The objective of the extension period (treatment period after PK parameters have been completed) was to assess the long-term safety of chronic administration of ataluren in this participant population.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ataluren | Experimental | Participants will be administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose will be provided based upon the weight of each participant, which will be assessed every 12 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ataluren | Drug | White to off-white powder for oral suspension. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An SAE was an adverse event (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) or persistent or significant disability/incapacity not related to dystrophinopathy. An event was not reported as an SAE, if event was exclusively a relapse or expected change or progression of baseline dystrophinopathy. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all non-serious AEs, regardless of causality, is located in the Reported Adverse Events section. | Baseline up to Week 56 |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter | Clinical laboratory results that were considered clinically meaningful were to be determined by the Investigator and Sponsor. Biochemistry parameters included sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, bilirubin (total, direct, and indirect), aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, creatine kinase, lactate dehydrogenase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters included white blood cell count with differential, hemoglobin, hematocrit, other red cell parameters, and platelet count. Urinalysis parameters included pH, specific gravity, glucose, ketones, blood, protein, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. | Baseline up to Week 56 |
| Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr) | Ataluren concentrations in plasma were analyzed using a validated high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) method. | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Francesco Bibbiani, MD | PTC Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Child Neuro NWF | Gulf Breeze | Florida | 32561 | United States | ||
| Rush University Medical Center |
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Participants in the Evaluable Population included all participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for Timed Function Test (TFT), North Star Ambulatory Assessment (NSAA), or response to the palatability of ataluren questions.
Participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene were recruited for this study.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Ataluren | Participants were administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
All participants who received at least 1 dose of ataluren (Safety Population).
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Ataluren | Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An SAE was an adverse event (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) or persistent or significant disability/incapacity not related to dystrophinopathy. An event was not reported as an SAE, if event was exclusively a relapse or expected change or progression of baseline dystrophinopathy. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all non-serious AEs, regardless of causality, is located in the Reported Adverse Events section. | All participants who received at least 1 dose of ataluren (Safety Population). | Posted | Count of Participants | Participants | Baseline up to Week 56 |
Baseline up to Week 56
Adverse events were collected from all participants who received at least 1 dose of ataluren (Safety Population).
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 | Ataluren | Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 19.0 | 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 | Jan 28, 2016 | Aug 12, 2020 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 19, 2018 | Aug 12, 2020 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
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| ID | Term |
|---|---|
| C515878 | ataluren |
Not provided
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ECG results that were considered clinically meaningful were to be determined by the Investigator. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Baseline up to Week 56 |
| Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity | Dose-limiting toxicity was measured through clinical evaluations for potential hepatic and renal toxicities. The clinical evaluations included the following:
| Baseline up to Week 56 |
| Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr) |
Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. |
| 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 |
| Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr) | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. AUC0-10hr was measured using the linear trapezoidal rule during the ascending portion of the curve and the log-trapezoidal rule during the descending portion of the curve. | 0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28 |
| Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr) | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 |
| Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs | TFTs included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. | Baseline, Week 28 and Week 52 |
| Change From Baseline in Physical Function as Measured by the NSAA | NSAA consists of 17 activities, including items assessing abilities necessary to remain functionally ambulant (that is, ability to rise from floor, to get from lying to sitting/sitting to standing, and that are known to progressively deteriorate); items that can be partly present in DMD early stages (that is, assessing head raise and standing on heels); and a number of activities such as hopping, jumping, and running. Since the boys were <5 years old, revised 16 point, 8-point, and 3-point scales were used over the 17 point scale. Scores for evaluations=0 (Unable to achieve independently), 1 (Modified method but achieved goal independent of physical assistance), or 2 (Normal, no obvious modification of activity). Maximum total score for the 16-point scale=32, 8-point scale=16, and 3-point scale=6. If an activity couldn't be performed due to PD/loss of ambulation, a score of 0 was assigned. Change from Baseline calculated by subtracting Baseline value from value at Week 28 and Week 52. | Baseline, Week 28 and Week 52 |
| Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56 | Baseline, Weeks 4, 16, 28, 40, 52, and 56 |
| Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56 | Baseline, Weeks 4, 16, 28, 40, 52, and 56 |
| Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56 | Body mass index is an estimate of body fat based on body weight divided by height squared. | Baseline, Weeks 4, 16, 28, 40, 52, and 56 |
| Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire | To assess palatability characteristics, participants/parents or guardians were asked to provide a response of "Strongly disagree", "Disagree", "Neither agree or disagree", "Agree", or "Strongly Agree" to the following 3 questions: Question 1. "Is the medicine palatable?" Question 2. "On the basis of reaction / facial expression of your child, do you think that the medication is pleasant?" Question 3."You sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up?" | Baseline up to Week 28 |
| Chicago |
| Illinois |
| 60612 |
| United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Children's Medical Center Dallas | Dallas | Texas | 75390-8843 | United States |
| University of Utah | Salt Lake City | Utah | 84112 | United States |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | Ataluren | Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks. |
|
|
| Primary | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter | Clinical laboratory results that were considered clinically meaningful were to be determined by the Investigator and Sponsor. Biochemistry parameters included sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, bilirubin (total, direct, and indirect), aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, creatine kinase, lactate dehydrogenase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters included white blood cell count with differential, hemoglobin, hematocrit, other red cell parameters, and platelet count. Urinalysis parameters included pH, specific gravity, glucose, ketones, blood, protein, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. | All participants who received at least 1 dose of ataluren (Safety Population). | Posted | Count of Participants | Participants | Baseline up to Week 56 |
|
|
|
| Primary | Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results | ECG results that were considered clinically meaningful were to be determined by the Investigator. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. | All participants who received at least 1 dose of ataluren (Safety Population). | Posted | Count of Participants | Participants | Baseline up to Week 56 |
|
|
|
| Primary | Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity | Dose-limiting toxicity was measured through clinical evaluations for potential hepatic and renal toxicities. The clinical evaluations included the following:
| All participants who received at least 1 dose of ataluren (Safety Population). | Posted | Number | participants | Baseline up to Week 56 |
|
|
|
| Secondary | Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr) | Ataluren concentrations in plasma were analyzed using a validated high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) method. | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | Posted | Mean | Standard Deviation | microgram/milliliter (µg/mL) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 |
|
|
|
| Secondary | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr) | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | Posted | Mean | Standard Deviation | hours | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 |
|
|
|
| Secondary | Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr) | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. AUC0-10hr was measured using the linear trapezoidal rule during the ascending portion of the curve and the log-trapezoidal rule during the descending portion of the curve. | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | Posted | Mean | Standard Deviation | hour*μg/mL | 0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28 |
|
|
|
| Secondary | Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr) | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | Posted | Mean | Standard Deviation | microgram per milliliter (µg/ml) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 |
|
|
|
| Secondary | Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs | TFTs included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. | All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable data for the applicable timed function test. | Posted | Mean | Standard Deviation | seconds | Baseline, Week 28 and Week 52 |
|
|
|
| Secondary | Change From Baseline in Physical Function as Measured by the NSAA | NSAA consists of 17 activities, including items assessing abilities necessary to remain functionally ambulant (that is, ability to rise from floor, to get from lying to sitting/sitting to standing, and that are known to progressively deteriorate); items that can be partly present in DMD early stages (that is, assessing head raise and standing on heels); and a number of activities such as hopping, jumping, and running. Since the boys were <5 years old, revised 16 point, 8-point, and 3-point scales were used over the 17 point scale. Scores for evaluations=0 (Unable to achieve independently), 1 (Modified method but achieved goal independent of physical assistance), or 2 (Normal, no obvious modification of activity). Maximum total score for the 16-point scale=32, 8-point scale=16, and 3-point scale=6. If an activity couldn't be performed due to PD/loss of ambulation, a score of 0 was assigned. Change from Baseline calculated by subtracting Baseline value from value at Week 28 and Week 52. | All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable NSAA data. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 28 and Week 52 |
|
|
|
| Secondary | Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56 | All participants who received at least 1 dose of ataluren (Safety Population) and had evaluable height data. | Posted | Mean | Standard Deviation | centimeters | Baseline, Weeks 4, 16, 28, 40, 52, and 56 |
|
|
|
| Secondary | Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56 | All participants who received at least 1 dose of ataluren (Safety Population) and had evaluable weight data. | Posted | Mean | Standard Deviation | kg | Baseline, Weeks 4, 16, 28, 40, 52, and 56 |
|
|
|
| Secondary | Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56 | Body mass index is an estimate of body fat based on body weight divided by height squared. | All participants who received at least 1 dose of ataluren (Safety Population). | Posted | Mean | Standard Deviation | kilograms per square meter (kg/m^2) | Baseline, Weeks 4, 16, 28, 40, 52, and 56 |
|
|
|
| Secondary | Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire | To assess palatability characteristics, participants/parents or guardians were asked to provide a response of "Strongly disagree", "Disagree", "Neither agree or disagree", "Agree", or "Strongly Agree" to the following 3 questions: Question 1. "Is the medicine palatable?" Question 2. "On the basis of reaction / facial expression of your child, do you think that the medication is pleasant?" Question 3."You sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up?" | All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population). | Posted | Count of Participants | Participants | Baseline up to Week 28 |
|
|
|
| 0 |
| 14 |
| 0 |
| 14 |
| 14 |
| 14 |
| Constipation | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Stomatitis | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Gait Disturbance | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Bronchitis Viral | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Croup Infectious | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Ear Infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Gastroenteritis Norovirus | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Gingival Abscess | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Pharyngitis Streptococcal | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Respiratory Tract Infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Viral Rash | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Arthropod Bite | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Tibia Fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Hepatic Enzyme Increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Metabolic Acidosis | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abnormal Behaviour | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Enuresis | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Chromaturia | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tonsillar Hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hypertrichosis | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rash Generalised | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 19.0 | 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.
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
|
| Rise to Standing, Change from Baseline at Week 52 |
|
|
| Walk/Run 10 m, Baseline |
|
|
| Walk/Run 10 m, Change from Baseline at Week 28 |
|
|
| Walk/Run 10 m, Change from Baseline at Week 52 |
|
|
| Ascend 4 Stairs, Baseline |
|
|
| Ascend 4 Stairs, Change from Baseline at Week 28 |
|
|
| Ascend 4 Stairs, Change from Baseline at Week 52 |
|
|
| Descend 4 Stairs, Baseline |
|
|
| Descend 4 Stairs, Change from Baseline at Week 28 |
|
|
| Descend 4 Stairs, Change from Baseline at Week 52 |
|
|
|
| 16-Point Scale, Change from Baseline at Week 52 |
|
|
| 8-Point Scale, Baseline |
|
|
| 8-Point Scale, Change from Baseline at Week 28 |
|
|
| 8-Point Scale, Change from Baseline at Week 52 |
|
|
| 3-Point Scale, Baseline |
|
|
| 3-Point Scale, Change from Baseline at Week 28 |
|
|
| 3-Point Scale, Change from Baseline at Week 52 |
|
|
|
| Change at Week 16 |
|
|
| Change at Week 28 |
|
|
| Change at Week 40 |
|
|
| Change at Week 52 |
|
|
| Change at Week 56 |
|
|
|
| Change at Week 16 |
|
|
| Change at Week 28 |
|
|
| Change at Week 40 |
|
|
| Change at Week 52 |
|
|
| Change at Week 56 |
|
|
|
| Change at Week 16 |
|
|
| Change at Week 28 |
|
|
| Change at Week 40 |
|
|
| Change at Week 52 |
|
|
| Change at Week 56 |
|
|
| Title | Measurements |
|---|---|
|
| Question 1, Agree |
|
| Question 1, Strongly agree |
|
| Question 1, No Response |
|
| Question 2, Strongly disagree |
|
| Question 2, Disagree |
|
| Question 2, Neither Agree nor Disagree |
|
| Question 2, Agree |
|
| Question 2, Strongly Agree |
|
| Question 2, No response |
|
| Question 3, Strongly Disagree |
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| Question 3, Disagree |
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| Question 3, Neither agree or disagree |
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| Question 3, Agree |
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| Question 3, Strongly Agree |
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| Question 3, No response |
|