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The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.
This Phase 3b extension study will assess the long-term safety of Ace-ER in patients who participated in and completed study UX001-CL301 (NCT02377921), study UX001-CL202 (NCT01830972), and study UX001-CL203 (NCT02731690).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Drug: Aceneuramic Acid Extended-Release Tablets | Experimental | Participants will take 4 tablets (500 mg Ace-ER each for 2 g per dose) orally 3 times per day (TID). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Aceneuramic Acid Extended-Release Tablets | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. An SAE or serious suspected adverse reaction is an AE or suspected adverse reaction that at any dose, in the view of either the Investigator or Ultragenyx, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability (substantial disruption of the ability to conduct normal life functions); congenital anomaly/birth defect. TEAEs were defined as any AE that occurred after the first dose of study drug. The severity of all AEs were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.03: grade1=mild, grade 2=moderate, grade 3=severe, grade 4=life-threatening, grade 5=death. | From first dose of study drug through the end of treatment plus 30 days (+5 days). Mean (SD) duration of treatment was 256.3 (101.54) days. |
| Change From Baseline in HHD UEC Score Over Time | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. The UEC is derived from the sum of the average of the right and left total force (measured in kgf). Analyzed using a repeated measure generalized estimation equation (GEE) model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the GNEM-FAS Expanded Version Mobility Domain Score Over Time | GNEM-FAS Expanded Version Mobility subscale score has 13 items and ranges from 0 to 52 with higher scores representing greater mobility. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, 48 |
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Inclusion Criteria:
Exclusion Criteria:
Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA | Los Angeles | California | 90095 | United States | ||
| University of California, Irvine |
Of the143 participants who enrolled, 1 participant withdrew consent prior to receiving the first dose and is not included in the Safety Analysis Population presented below.
143 participants were screened and enrolled across 14 total sites in the United States, Israel, United Kingdom, Italy, France, Canada, and Bulgaria. 87 participants rolled over from study UX001-CL301 (NCT02377921), 49 participants rolled over from study UX001-CL202 (NCT01830972), and 7 participants rolled over from UX001-CL203 (NCT02731690).
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| ID | Title | Description |
|---|---|---|
| FG000 | Ace-ER 6 g/Day | 4 tablets (500 mg Ace-ER each for 2 g per dose) orally 3 times per day |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 17, 2016 | Dec 18, 2018 |
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| Change From Baseline on the GNEM-FAS Upper Extremity Domain Score Over Time | GNEM-FAS Expanded Version Upper Extremity subscale score has 9 items and ranges from 0 to 36 with higher scores representing more skilled, independent use of the arms during functional activity performance. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, 48 |
| Change From Baseline in HHD Lower Extremity Composite (LEC) Score Over Time | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. The LEC is derived from the sum of the average of the right and left total force (measured in kgf). Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Change From Baseline in the Number of Stands in the Sit-to-Stand Test Over Time | Lower extremity function was assessed using a sit-to-stand test. The number of times the participant can rise from a seated to a standing position in a 30-second period was recorded. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Change From Baseline in Number of Lifts in the 30-Second Weighted Arm Lift Test Over Time | Upper extremity function was assessed using a weighted arm lift test performed bilaterally. The number of times the participant can raise a 1 kg weight above the head in a 30-second period was recorded. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Change From Baseline in Meters Walked in 6MWT Over Time | The total distance walked (meters) in a 6-minute period was measured. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Change From Baseline in Percent Predicted Meters Walked in 6MWT Over Time | The total distance walked (meters) in a 6-minute period was measured, and the percent predicted distance based on normative data for age and gender was estimated. Predicted 6MWT distance (meters) = 868.8 - (2.99 x Age) - (74.7 x Sex), where age is baseline age in years, and sex = 0 for males, and 1 for females. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Change From Baseline in Total Force in Knee Extensors Over Time | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. Bilateral total force was defined as the average of the right and left force (measured in kgf). Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Change From Baseline in Percent Predicted Total Force in Knee Extensors Over Time | The percent predicted total force value of lower extremity muscle strength in the knee extensors was determined based on reference equations adjusting for age, gender, height, and weight. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Baseline, Weeks 8, 16, 24, and 48 |
| Orange |
| California |
| 92868 |
| United States |
| Washington University School of Medicine, St. Louis | St Louis | Missouri | 63110 | United States |
| New York University School of Medicine | New York | New York | 10016 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| UMHAT Alexandrovska, Bulgaria | Sofia | 1431 | Bulgaria |
| McMaster University | Hamilton | Ontario | L8N 3Z5 | Canada |
| CHU La Réunion - site GHSR | Saint-Pierre | Reunion | France |
| Institut de Myologie GH Pitié-Salpêtrière | Paris | France |
| Hadassah-Hebrew University Medical Center | Jerusalem | Israel |
| University of Messina | Messina | Italy |
| University of Milan | Milan | Italy |
| Università Cattolica | Rome | Italy |
| The Newcastle upon Tyne Hospitals | Newcastle upon Tyne | Tyne and Wear | NE1 4LP | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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Safety Analysis Set: all participants who received at least one dose of study drug in UX001-CL302.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ace-ER 6 g/Day | 4 tablets (500 mg Ace-ER each for 2 g per dose) orally 3 times per day |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Hand Held Dynamometry (HHD) Upper Extremity Composite Score (UEC) | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kilogram-force [kgf]) for each was recorded. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | kgf |
| |||||||||||||||
| Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy Functional Activities Scale(GNEM-FAS) Mobility Score | GNEM-FAS Expanded Version Mobility subscale score has 13 items and ranges from 0 to 52 with higher scores representing greater mobility. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | score on a scale |
| |||||||||||||||
| GNEM-FAS Expanded Version Upper Extremity Score | GNEM-FAS Expanded Version Upper Extremity subscale score has 9 items and ranges from 0 to 36 with higher scores representing more skilled, independent use of the arms during functional activity performance. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | score on a scale |
| |||||||||||||||
| HHD Lower Extremity Composite (LEC) Score | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | kgf |
| |||||||||||||||
| Sit-to-Stand Test | Lower extremity function was assessed using a sit-to-stand test. The number of times the participant can rise from a seated to a standing position in a 30-second period was recorded. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | stands |
| |||||||||||||||
| 30-second Weighted Arm Lift Test | Upper extremity function was assessed using a weighted arm lift test performed bilaterally. The number of times the participant can raise a 1 kg weight above the head in a 30-second period was recorded. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | lifts |
| |||||||||||||||
| Six-Minute Walk Test (6MWT) | The total distance walked (meters) in a 6-minute period was measured. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | meters |
| |||||||||||||||
| Percent Predicted Meters Walked in 6MWT | The total distance walked (meters) in a 6-minute period was measured, and the percent predicted distance based on normative data for age and gender was estimated. Predicted 6MWT distance (meters) = 868.8 - (2.99 x Age) - (74.7 x Sex), where age is baseline age in years, and sex = 0 for males, and 1 for females. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | percentage of predicted meters |
| |||||||||||||||
| Total Force in Knee Extensors | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | kgf |
| |||||||||||||||
| Percent of Predicted Total Force in Knee Extensors | The percent predicted total force value of lower extremity muscle strength in the knee extensors was determined based on reference equations adjusting for age, gender, height, and weight. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Mean | Standard Deviation | percent of predicted total force |
|
| 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), Serious AEs (SAEs), and Discontinuations Due to AEs | An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. An SAE or serious suspected adverse reaction is an AE or suspected adverse reaction that at any dose, in the view of either the Investigator or Ultragenyx, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability (substantial disruption of the ability to conduct normal life functions); congenital anomaly/birth defect. TEAEs were defined as any AE that occurred after the first dose of study drug. The severity of all AEs were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.03: grade1=mild, grade 2=moderate, grade 3=severe, grade 4=life-threatening, grade 5=death. | Safety Analysis Set: all participants who received at least one dose of study drug in UX001-CL302. | Posted | Count of Participants | Participants | From first dose of study drug through the end of treatment plus 30 days (+5 days). Mean (SD) duration of treatment was 256.3 (101.54) days. |
|
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| ||||||||||||||||||||||||||||||||||||
| Primary | Change From Baseline in HHD UEC Score Over Time | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. The UEC is derived from the sum of the average of the right and left total force (measured in kgf). Analyzed using a repeated measure generalized estimation equation (GEE) model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | kgf | Baseline, Weeks 8, 16, 24, 48 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the GNEM-FAS Expanded Version Mobility Domain Score Over Time | GNEM-FAS Expanded Version Mobility subscale score has 13 items and ranges from 0 to 52 with higher scores representing greater mobility. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Baseline, Weeks 8, 16, 24, 48 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline on the GNEM-FAS Upper Extremity Domain Score Over Time | GNEM-FAS Expanded Version Upper Extremity subscale score has 9 items and ranges from 0 to 36 with higher scores representing more skilled, independent use of the arms during functional activity performance. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Baseline, Weeks 8, 16, 24, 48 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in HHD Lower Extremity Composite (LEC) Score Over Time | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. The LEC is derived from the sum of the average of the right and left total force (measured in kgf). Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | kgf | Baseline, Weeks 8, 16, 24, and 48 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the Number of Stands in the Sit-to-Stand Test Over Time | Lower extremity function was assessed using a sit-to-stand test. The number of times the participant can rise from a seated to a standing position in a 30-second period was recorded. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | stands | Baseline, Weeks 8, 16, 24, and 48 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Number of Lifts in the 30-Second Weighted Arm Lift Test Over Time | Upper extremity function was assessed using a weighted arm lift test performed bilaterally. The number of times the participant can raise a 1 kg weight above the head in a 30-second period was recorded. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | lifts | Baseline, Weeks 8, 16, 24, and 48 |
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| Secondary | Change From Baseline in Meters Walked in 6MWT Over Time | The total distance walked (meters) in a 6-minute period was measured. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | meters | Baseline, Weeks 8, 16, 24, and 48 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Predicted Meters Walked in 6MWT Over Time | The total distance walked (meters) in a 6-minute period was measured, and the percent predicted distance based on normative data for age and gender was estimated. Predicted 6MWT distance (meters) = 868.8 - (2.99 x Age) - (74.7 x Sex), where age is baseline age in years, and sex = 0 for males, and 1 for females. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | percent of predicted distance | Baseline, Weeks 8, 16, 24, and 48 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Total Force in Knee Extensors Over Time | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. Bilateral total force was defined as the average of the right and left force (measured in kgf). Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | kgf | Baseline, Weeks 8, 16, 24, and 48 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Predicted Total Force in Knee Extensors Over Time | The percent predicted total force value of lower extremity muscle strength in the knee extensors was determined based on reference equations adjusting for age, gender, height, and weight. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | Posted | Least Squares Mean | 95% Confidence Interval | percent of predicted total force (kgf) | Baseline, Weeks 8, 16, 24, and 48 |
|
|
From first dose of study drug through the end of treatment plus 30 days (+5 days). Mean (SD) duration of treatment was 256.3 (101.54) days.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ace-ER 6 g/Day | 4 tablets (500 mg Ace-ER each for 2 g per dose) orally 3 times per day | 0 | 142 | 7 | 142 | 66 | 142 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Cholecystitis acute | Hepatobiliary disorders | MedDRA 17.1 | Systematic Assessment |
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| Atypical pneumonia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Biopsy kidney | Investigations | MedDRA 17.1 | Systematic Assessment |
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| Invasive ductal breast carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 17.1 | Systematic Assessment |
| |
| Malignant melanoma in situ | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 17.1 | Systematic Assessment |
| |
| Renal pain | Renal and urinary disorders | MedDRA 17.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Flatulence | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 17.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
|
No efficacy result summaries or analyses were performed for participants rolling over from UX001-CL202 or UX001-CL203 because of the limited data from those participants due to the early study closure.
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8657 | medinfo@ultragenyx.com |
| Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 15, 2017 | Dec 18, 2018 | SAP_000.pdf |
| ID | Term |
|---|---|
| C536816 | Distal myopathy, Nonaka type |
Not provided
Not provided
Not provided
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| Unknown or Not Reported |
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| Other, Not Specified |
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| Title | Measurements |
|---|---|
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| Grade 3 or 4 TEAEs |
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| TEAEs Leading to Study Drug Discontinuation |
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