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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-000104-26 | EudraCT Number |
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The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| UX003 | Experimental | UX003 4 mg/kg every other week (QOW). Initial treatment period 48 weeks. Continuation period up to 240 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| UX003 | Drug | solution for intravenous infusion |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in uGAG Excretion (LC-MS/MS-DS) at Week 48 | Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate (LS-MS/MS-DS) method. For the participant previously treated with UX003 under an eIND, percent change from initial baseline was used. | Baseline (Week 0), Week 48 |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Adverse event (AE): any untoward medical occurrence in a participant, whether or not considered drug related. Serious AE (SAE): an AE or suspected adverse reaction that at any dose results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. Other important medical events may also, in the opinion of the Investigator, be considered SAEs. An AE was considered a TEAE if it occurred on or after the first dose, and was not present prior to the first dose, or it was present at the first dose but increased in severity during the study. Events recorded as either possibly, probably, or definitely related to treatment were categorized as related. AE severity was graded using the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.03. | From first dose of study drug until 30 days after the last dose of study drug. Mean (SD) treatment duration was 98.11 (29.02) weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline Over Time in Standing Height | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Health System | Washington D.C. | District of Columbia | 20010 | United States | ||
| New York University Langone Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37415957 | Derived | Montano AM, Rozdzynska-Swiatkowska A, Jurecka A, Ramirez AN, Zhang L, Marsden D, Wang RY, Harmatz P. Growth patterns in patients with mucopolysaccharidosis VII. Mol Genet Metab Rep. 2023 Jun 26;36:100987. doi: 10.1016/j.ymgmr.2023.100987. eCollection 2023 Sep. | |
| 35331634 | Derived | Lau HA, Viskochil D, Tanpaiboon P, Lopez AG, Martins E, Taylor J, Malkus B, Zhang L, Jurecka A, Marsden D. Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII. Mol Genet Metab. 2022 May;136(1):28-37. doi: 10.1016/j.ymgme.2022.03.002. Epub 2022 Mar 9. |
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| ID | Title | Description |
|---|---|---|
| FG000 | UX003 | UX003 4 mg/kg every other week (QOW). Initial treatment period 48 weeks. Continuation period up to 240 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| 48-Week Treatment Period |
|
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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 | Apr 7, 2016 | Sep 4, 2019 |
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| Change From Baseline Over Time in Standing Height Z-Score |
The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. |
| Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
| Change From Baseline Over Time in Head Circumference | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
| Change From Baseline Over Time in Head Circumference Z-Score | The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Baseline, Weeks 12, 24, 36, 48 |
| Change From Baseline Over Time in Weight | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
| Post-UX003 Growth Velocity (cm/yr) for Participants With Both Historical Pre-UX003 (Within 2 Years) and Post-UX003 Data | The growth velocity for pre-treatment is based on standing height within 2 years prior to treatment. The growth velocity for post-treatment is based on all standing height data during the study period. For the participant previously treated with UX003 under an eIND, the growth velocity was calculated for pre initial UX003 treatment and post initial UX003 treatment. | Pre-treatment (based on standing height within 2 years prior to treatment), Post-treatment (based on all standing height data during the study period up to 240 weeks) |
| Change From Pre-Treatment (Within 2 Years) to Post-Treatment Growth Velocity Z-Score | The Z-score indicates the number of standard deviations away from a reference population (based on Tanner's standard [Tanner et al. 1985]) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. The growth velocity for pre-treatment is based on standing height within 2 years prior to treatment. The growth velocity for post-treatment is based on all standing height data during the study period. For the participant previously treated with UX003 under an eIND, the growth velocity was calculated for pre initial UX003 treatment and post initial UX003 treatment. | Pre-treatment (based on standing height within 2 years prior to treatment), Post-treatment (based on all standing height data during the study period up to Week 48) |
| Change From Baseline Over Time in Liver Measurement | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Baseline, Weeks 12, 24, 48, 96, 144 |
| Change From Baseline Over Time in Spleen Measurement | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Baseline, Weeks 12, 24, 48, 96, 144 |
| New York |
| New York |
| 10038 |
| United States |
| University of Utah Hospital | Salt Lake City | Utah | 84132 | United States |
| Centro Hospitalar do Porto | Porto | 4099-345 | Portugal |
| Hospital Universitario Virgen Del Rocio | Seville | 41013 | Spain |
| 30467742 | Derived | Qi Y, McKeever K, Taylor J, Haller C, Song W, Jones SA, Shi J. Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials. Clin Pharmacokinet. 2019 May;58(5):673-683. doi: 10.1007/s40262-018-0721-y. |
| Emergency IND (eIND) | Previously treated with UX003 under eIND application; did not complete the 48-Week Treatment Period. |
|
| COMPLETED |
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| NOT COMPLETED |
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| Continuation Period |
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|
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| ID | Title | Description |
|---|---|---|
| BG000 | UX003 | UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 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 | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Urinary Glycosaminoglycans (uGAG) Excretion | Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate (LS-MS/MS-DS) method. | Mean | Standard Deviation | g GAG/g creatinine |
| ||||||||||||||||
| Standing Height | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | eIND and non-eIND participants were analyzed separately for this measure. | Mean | Standard Deviation | cm |
| |||||||||||||||
| Standing Height Z-Score | The Z-score indicates the number of standard deviations away from a reference population (from the Centers for Disease Control [CDC] growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. | eIND and non-eIND participants were analyzed separately for this measure. | Mean | Standard Deviation | Z-score |
| |||||||||||||||
| Head Circumference | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | eIND and non-eIND participants were analyzed separately for this measure. | Mean | Standard Deviation | cm |
| |||||||||||||||
| Head Circumference Z-Score | The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. | eIND and non-eIND participants were analyzed separately for this measure. Participants with a non-missing baseline assessment are presented. | Mean | Standard Deviation | Z-score |
| |||||||||||||||
| Weight | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | eIND and non-eIND participants were analyzed separately for this measure. | Mean | Standard Deviation | kg |
| |||||||||||||||
| Historical Pre-Treatment (Within 2 Years) Growth Velocity | Pre-treatment data include baseline and pre-treatment data (within 2 years). For the eIND participant, the growth velocity was calculated for pre initial UX003 treatment. | All enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline. | Mean | Standard Deviation | cm/year |
| |||||||||||||||
| Pre-Treatment (Within 2 Years) Growth Velocity Z-Score | The Z-score indicates the number of standard deviations away from a reference population (based on Tanner's standard [Tanner et al. 1985]) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. | All enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline. Growth velocity Z-score was only calculated for participants ≥ 2.25 years. | Mean | Standard Deviation | Z-score |
| |||||||||||||||
| Liver Measurement | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | eIND and non-eIND participants were analyzed separately for this measure. One participant in the non-eIND group did not have a baseline measurement. | Mean | Standard Deviation | cm |
| |||||||||||||||
| Spleen Measurement | For all participants (including the participant previously treated with UX003 under an emergency IND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | e-IND and non-eIND participants were analyzed separately | Mean | Standard Deviation | cm |
|
| 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 | Percent Change From Baseline in uGAG Excretion (LC-MS/MS-DS) at Week 48 | Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate (LS-MS/MS-DS) method. For the participant previously treated with UX003 under an eIND, percent change from initial baseline was used. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study and had a non-missing baseline and Week 48 assessment. | Posted | Mean | Standard Deviation | percent change | Baseline (Week 0), Week 48 |
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| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Adverse event (AE): any untoward medical occurrence in a participant, whether or not considered drug related. Serious AE (SAE): an AE or suspected adverse reaction that at any dose results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. Other important medical events may also, in the opinion of the Investigator, be considered SAEs. An AE was considered a TEAE if it occurred on or after the first dose, and was not present prior to the first dose, or it was present at the first dose but increased in severity during the study. Events recorded as either possibly, probably, or definitely related to treatment were categorized as related. AE severity was graded using the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.03. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study. | Posted | Count of Participants | Participants | No | From first dose of study drug until 30 days after the last dose of study drug. Mean (SD) treatment duration was 98.11 (29.02) weeks |
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| Secondary | Change From Baseline Over Time in Standing Height | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | cm | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
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| Secondary | Change From Baseline Over Time in Standing Height Z-Score | The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | Z-score | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
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| Secondary | Change From Baseline Over Time in Head Circumference | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | cm | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
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| Secondary | Change From Baseline Over Time in Head Circumference Z-Score | The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | Z-score | Baseline, Weeks 12, 24, 36, 48 |
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| Secondary | Change From Baseline Over Time in Weight | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | kg | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132 |
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| Secondary | Post-UX003 Growth Velocity (cm/yr) for Participants With Both Historical Pre-UX003 (Within 2 Years) and Post-UX003 Data | The growth velocity for pre-treatment is based on standing height within 2 years prior to treatment. The growth velocity for post-treatment is based on all standing height data during the study period. For the participant previously treated with UX003 under an eIND, the growth velocity was calculated for pre initial UX003 treatment and post initial UX003 treatment. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with both historical pre-treatment (within 2 years) and post-treatment data. | Posted | Mean | Standard Deviation | cm/year | Pre-treatment (based on standing height within 2 years prior to treatment), Post-treatment (based on all standing height data during the study period up to 240 weeks) |
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| Secondary | Change From Pre-Treatment (Within 2 Years) to Post-Treatment Growth Velocity Z-Score | The Z-score indicates the number of standard deviations away from a reference population (based on Tanner's standard [Tanner et al. 1985]) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. The growth velocity for pre-treatment is based on standing height within 2 years prior to treatment. The growth velocity for post-treatment is based on all standing height data during the study period. For the participant previously treated with UX003 under an eIND, the growth velocity was calculated for pre initial UX003 treatment and post initial UX003 treatment. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with both historical pre-treatment (within 2 years) and post-treatment data. Growth velocity Z-score was only calculated for participants ≥ 2.25 years. | Posted | Mean | Standard Deviation | Z-score | Pre-treatment (based on standing height within 2 years prior to treatment), Post-treatment (based on all standing height data during the study period up to Week 48) |
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| Secondary | Change From Baseline Over Time in Liver Measurement | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | cm | Baseline, Weeks 12, 24, 48, 96, 144 |
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| Secondary | Change From Baseline Over Time in Spleen Measurement | For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline. | Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point. | Posted | Mean | Standard Deviation | cm | Baseline, Weeks 12, 24, 48, 96, 144 |
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From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | UX003 | UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. | 0 | 8 | 3 | 8 | 8 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bradycardia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
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| Device Fastener Issue | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Otitis Media | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Oxygen Saturation Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Cervical Spinal Stenosis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Spinal Column Stenosis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Spinal Instability | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cervical Cord Compression | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Febrile Convulsion | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Adenoidal Hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Microcytosis | Blood and lymphatic system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pulmonary Valve Stenosis | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Right Ventricular Hypertension | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Supraventricular Extrasystoles | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
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| Tachycardia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Mucopolysaccharidosis Vii | Congenital, familial and genetic disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pectus Carinatum | Congenital, familial and genetic disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cerumen Impaction | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
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| Ear Pain | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
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| Corneal Deposits | Eye disorders | MedDRA 18.1 | Systematic Assessment |
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| Myopia | Eye disorders | MedDRA 18.1 | Systematic Assessment |
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| Strabismus | Eye disorders | MedDRA 18.1 | Systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Gingival Bleeding | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Gingival Pain | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Lip Haemorrhage | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Oral Papule | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Device Defective | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Device Malfunction | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Discomfort | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Extravasation | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Inflammation | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Infusion Site Extravasation | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Secretion Discharge | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Tenderness | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Hepatomegaly | Hepatobiliary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Anaphylactoid Reaction | Immune system disorders | MedDRA 18.1 | Systematic Assessment |
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| Adenoiditis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Bacterial Disease Carrier | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
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| Device Related Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
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| Ear Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
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| Enterovirus Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Eye Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Fungal Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Fungal Skin Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gastrointestinal Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gingival Abscess | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Otitis Media | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Otitis Media Acute | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Otitis Media Chronic | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pharyngitis Streptococcal | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pharyngotonsillitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pneumonia Viral | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Respiratory Tract Infection Viral | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinovirus Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Gastrostomy Tube Site Complication | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Limb Injury | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Procedural Pain | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Scar | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Skin Abrasion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Alanine Aminotransferase Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood Calcium Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood Creatinine Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood Pressure Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Body Temperature Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Body Temperature Abnormal | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Gamma-Glutamyltransferase Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Hepatic Enzyme Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Oxygen Saturation Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Right Ventricular Systolic Pressure Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Vitamin D Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Bone Deformity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Muscular Weakness | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Musculoskeletal Chest Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Spinal Deformity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cerebellar Microhaemorrhage | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Enlarged Cerebral Perivascular Spaces | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Myelopathy | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Periodic Limb Movement Disorder | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Attention Deficit/Hyperactivity Disorder | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Sleep Disorder | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Bladder Disorder | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasal Mucosal Disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pneumonia Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinitis Allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Tonsillar Hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Blood Blister | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Intertrigo | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Keloid Scar | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rash Papular | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rash Pruritic | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Skin Irritation | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8567 | medinfo@ultragenyx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 22, 2015 | Sep 4, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D016538 | Mucopolysaccharidosis VII |
| D009083 | Mucopolysaccharidoses |
| D035583 | Rare Diseases |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| ID | Term |
|---|---|
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C000654126 | vestronidase alfa |
Not provided
Not provided
Not provided
|
| Unknown or Not Reported |
|
|
|
| White |
|
|
| Other, Not Specified |
|
|
| non-eIND |
|
|
| Non-eIND |
|
|
| Non-eIND |
|
|
| Non-eIND |
|
|
| Non-eIND |
|
|
| Non-eIND |
|
|
| Non-eIND |
|
|
| Units | Counts |
|---|
| Participants |
|
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
| Participants |
|
|
|
|
| Participants |
|
|
|
|
|
|
|