Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

ClinicalTrials.gov Identifier: NCT02418455

Recruitment Status : Completed

First Posted : April 16, 2015

Results First Posted : October 16, 2019

Last Update Posted : October 30, 2019

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Ultragenyx Pharmaceutical Inc

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Conditions	Sly Syndrome MPS VII Mucopolysaccharidosis Mucopolysaccharidosis VII
Intervention	Drug: UX003
Enrollment	8

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	UX003
Arm/Group Description	UX003 4 mg/kg every other week (QOW...
Arm/Group Description	UX003 4 mg/kg every other week (QOW). Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Period Title: 48-Week Treatment Period
Started	8
Emergency IND (eIND) ^[1]	1
Completed	7
Not Completed	1
Reason Not Completed
Other- Not Specified	1
[1] Previously treated with UX003 under eIND application; did not complete the 48-Week Treatment Period.
Period Title: Continuation Period
Started	7
Completed	0
Not Completed	7
Reason Not Completed
Sponsor Decision	7

Baseline Characteristics

Arm/Group Title		UX003
Arm/Group Description		UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Baseline Participants		8
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	8 participants
		3.25 (1.197)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	8 participants
	Female	3 37.5%
	Male	5 62.5%
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	8 participants
	Hispanic or Latino	3 37.5%
	Not Hispanic or Latino	4 50.0%
	Unknown or Not Reported	1 12.5%
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants
Asian	Number Analyzed	8 participants
Asian		2 25.0%
Black or African American	Number Analyzed	8 participants
Black or African American		1 12.5%
White	Number Analyzed	8 participants
White		3 37.5%
Other, Not Specified	Number Analyzed	8 participants
Other, Not Specified		2 25.0%
Urinary Glycosaminoglycans (uGAG) Excretion ^[1] Mean (Standard Deviation) Unit of measure: G GAG/g creatinine
	Number Analyzed	8 participants
		2.092 (1.3307)
		[1] Measure Description: Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate (LS-MS/MS-DS) method.
Standing Height ^[1] [2] Mean (Standard Deviation) Unit of measure: Cm
eIND	Number Analyzed	1 participants
eIND		52.20 ^[3] (NA)
non-eIND	Number Analyzed	7 participants
non-eIND		89.34 (7.198)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: eIND and non-eIND participants were analyzed separately for this measure. [3] 1 participant analyzed
Standing Height Z-Score ^[1] [2] Mean (Standard Deviation) Unit of measure: Z-score
eIND	Number Analyzed	1 participants
eIND		-5.352 ^[3] (NA)
Non-eIND	Number Analyzed	7 participants
Non-eIND		-2.241 (0.4327)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: eIND and non-eIND participants were analyzed separately for this measure. [3] 1 participant analyzed
Head Circumference ^[1] [2] Mean (Standard Deviation) Unit of measure: Cm
eIND	Number Analyzed	1 participants
eIND		37.00 ^[3] (NA)
Non-eIND	Number Analyzed	7 participants
Non-eIND		51.57 (2.130)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: eIND and non-eIND participants were analyzed separately for this measure. [3] 1 participant analyzed
Head Circumference Z-Score ^[1] [2] Mean (Standard Deviation) Unit of measure: Z-score
eIND	Number Analyzed	1 participants
eIND		-3.329 ^[3] (NA)
Non-eIND	Number Analyzed	5 participants
Non-eIND		1.465 (1.3186)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: eIND and non-eIND participants were analyzed separately for this measure. Participants with a non-missing baseline assessment are presented. [3] 1 participant analyzed
Weight ^[1] [2] Mean (Standard Deviation) Unit of measure: Kg
eIND	Number Analyzed	1 participants
eIND		4.26 ^[3] (NA)
Non-eIND	Number Analyzed	7 participants
Non-eIND		13.99 (2.274)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: eIND and non-eIND participants were analyzed separately for this measure. [3] 1 participant analyzed
Historical Pre-Treatment (Within 2 Years) Growth Velocity ^[1] [2] Mean (Standard Deviation) Unit of measure: Cm/year
	Number Analyzed	5 participants
		5.06 (1.885)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: All enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline.
Pre-Treatment (Within 2 Years) Growth Velocity Z-Score ^[1] [2] Mean (Standard Deviation) Unit of measure: Z-score
	Number Analyzed	4 participants
		-2.587 (1.4860)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: 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.
Liver Measurement ^[1] [2] Mean (Standard Deviation) Unit of measure: Cm
eIND	Number Analyzed	1 participants
eIND		7.60 ^[3] (NA)
Non-eIND	Number Analyzed	6 participants
Non-eIND		10.70 (1.138)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: eIND and non-eIND participants were analyzed separately for this measure. One participant in the non-eIND group did not have a baseline measurement. [3] 1 participant analyzed
Spleen Measurement ^[1] [2] Mean (Standard Deviation) Unit of measure: Cm
eIND	Number Analyzed	1 participants
eIND		6.40 ^[3] (NA)
Non-eIND	Number Analyzed	7 participants
Non-eIND		8.17 (1.115)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: e-IND and non-eIND participants were analyzed separately [3] 1 participant analyzed

Outcome Measures

1.Primary Outcome


Title	Percent Change From Baseline in uGAG Excretion (LC-MS/MS-DS) at Week 48
Description	Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sul...
Description	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.
Time Frame	Baseline (Week 0), Week 48

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title	UX003
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed	7
Mean (Standard Deviation) Unit of Measure: percent change
	-58.17 (16.915)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	UX003
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	< 0.0001
	Comments	P-values are from generalized estimating equation (GEE) model including baseline value and visit as a categorical variable. The covariance structure within subjects is assumed to be exchangeable.
	Method	GEE model
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-61.00
	Confidence Interval	(2-Sided) 95% -73.56 to -48.44
	Parameter Dispersion	Type: Standard Error of the Mean Value: 6.409
	Estimation Comments	[Not Specified]

2.Primary Outcome


Title	Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Description	Adverse event (AE): any untoward medical occurrence in a participant, ...
Description	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.
Time Frame	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

Outcome Measure Data

Analysis Population Description

Full analysis set: all enrolled participants who received at least one dose of UX003 during the study.


Arm/Group Title	UX003
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed	8
Measure Type: Count of Participants Unit of Measure: Participants
TEAEs	8
Serious TEAEs	3
Treatment-Related TEAEs	5
Treatment-Related Serious TEAEs	1
Grade 3 or 4 TEAEs	2
TEAEs Leading to Treatment Discontinuation	0
TEAEs Leading to Study Discontinuation	0
TEAEs Leading to Death	0

3.Secondary Outcome


Title	Change From Baseline Over Time in Standing Height
Description	For all participants (including the participant previously treated wit...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	7
Mean (Standard Deviation) Unit of Measure: cm
Change at Week 12	Number Analyzed	0 participants	7 participants
Change at Week 12			1.94 (1.927)
Change at Week 24	Number Analyzed	1 participants	7 participants
Change at Week 24		15.00 ^[1] (NA)	3.71 (0.994)
Change at Week 36	Number Analyzed	1 participants	7 participants
Change at Week 36		20.00 ^[1] (NA)	5.64 (1.725)
Change at Week 48	Number Analyzed	0 participants	7 participants
Change at Week 48			6.57 (1.058)
Change at Week 60	Number Analyzed	0 participants	7 participants
Change at Week 60			8.59 (1.318)
Change at Week 72	Number Analyzed	0 participants	6 participants
Change at Week 72			9.63 (1.451)
Change at Week 84	Number Analyzed	0 participants	6 participants
Change at Week 84			10.93 (2.095)
Change at Week 96	Number Analyzed	0 participants	5 participants
Change at Week 96			11.86 (3.146)
Change at Week 108	Number Analyzed	0 participants	3 participants
Change at Week 108			12.17 (3.329)
Change at Week 120	Number Analyzed	0 participants	3 participants
Change at Week 120			12.67 (3.617)
Change at Week 132	Number Analyzed	0 participants	1 participants
Change at Week 132			14.80 ^[1] (NA)

[1]

1 participant analyzed

4.Secondary Outcome


Title	Change From Baseline Over Time in Standing Height Z-Score
Description	The Z-score indicates the number of standard deviations away from a re...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	7
Mean (Standard Deviation) Unit of Measure: Z-score
Change at Week 12	Number Analyzed	0 participants	7 participants
Change at Week 12			0.106 (0.4938)
Change at Week 24	Number Analyzed	1 participants	7 participants
Change at Week 24		-0.481 ^[1] (NA)	0.188 (0.3013)
Change at Week 36	Number Analyzed	1 participants	7 participants
Change at Week 36		0.314 ^[1] (NA)	0.314 (0.4103)
Change at Week 48	Number Analyzed	0 participants	7 participants
Change at Week 48			0.196 (0.3012)
Change at Week 60	Number Analyzed	0 participants	7 participants
Change at Week 60			0.333 (0.3883)
Change at Week 72	Number Analyzed	0 participants	6 participants
Change at Week 72			0.246 (0.3299)
Change at Week 84	Number Analyzed	0 participants	6 participants
Change at Week 84			0.203 (0.3973)
Change at Week 96	Number Analyzed	0 participants	5 participants
Change at Week 96			0.237 (0.5312)
Change at Week 108	Number Analyzed	0 participants	3 participants
Change at Week 108			-0.035 (0.4531)
Change at Week 120	Number Analyzed	0 participants	3 participants
Change at Week 120			-0.176 (0.4915)
Change at Week 132	Number Analyzed	0 participants	1 participants
Change at Week 132			-0.147 ^[1] (NA)

[1]

1 participant analyzed

5.Secondary Outcome


Title	Change From Baseline Over Time in Head Circumference
Description	For all participants (including the participant previously treated wit...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	7
Mean (Standard Deviation) Unit of Measure: cm
Change at Week 12	Number Analyzed	0 participants	7 participants
Change at Week 12			0.36 (0.378)
Change at Week 24	Number Analyzed	1 participants	7 participants
Change at Week 24		12.50 ^[1] (NA)	0.54 (0.360)
Change at Week 36	Number Analyzed	1 participants	7 participants
Change at Week 36		12.00 ^[1] (NA)	-0.27 (1.732)
Change at Week 48	Number Analyzed	0 participants	7 participants
Change at Week 48			0.79 (0.267)
Change at Week 60	Number Analyzed	0 participants	7 participants
Change at Week 60			0.86 (0.378)
Change at Week 72	Number Analyzed	0 participants	5 participants
Change at Week 72			1.10 (0.224)
Change at Week 84	Number Analyzed	0 participants	6 participants
Change at Week 84			0.70 (0.837)
Change at Week 96	Number Analyzed	0 participants	5 participants
Change at Week 96			0.84 (1.108)
Change at Week 108	Number Analyzed	0 participants	3 participants
Change at Week 108			1.07 (1.793)
Change at Week 120	Number Analyzed	0 participants	3 participants
Change at Week 120			1.33 (2.082)
Change at Week 132	Number Analyzed	0 participants	1 participants
Change at Week 132			2.00 ^[1] (NA)

[1]

1 participant analyzed

6.Secondary Outcome


Title	Change From Baseline Over Time in Head Circumference Z-Score
Description	The Z-score indicates the number of standard deviations away from a re...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 36, 48

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	1
Mean (Standard Deviation) Unit of Measure: Z-score
Change at Week 12	Number Analyzed	0 participants	1 participants
Change at Week 12			0.052 ^[1] (NA)
Change at Week 24	Number Analyzed	1 participants	1 participants
Change at Week 24		3.804 ^[1] (NA)	0.177 ^[1] (NA)
Change at Week 36	Number Analyzed	1 participants	1 participants
Change at Week 36		3.263 ^[1] (NA)	-0.733 ^[1] (NA)
Change at Week 48	Number Analyzed	0 participants	1 participants
Change at Week 48			-0.218 ^[1] (NA)

[1]

1 participant analyzed

7.Secondary Outcome


Title	Change From Baseline Over Time in Weight
Description	For all participants (including the participant previously treated wit...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	7
Mean (Standard Deviation) Unit of Measure: kg
Change at Week 12	Number Analyzed	1 participants	7 participants
Change at Week 12		5.54 ^[1] (NA)	0.69 (0.626)
Change at Week 24	Number Analyzed	1 participants	7 participants
Change at Week 24		5.94 ^[1] (NA)	1.21 (0.904)
Change at Week 36	Number Analyzed	1 participants	7 participants
Change at Week 36		5.94 ^[1] (NA)	1.46 (1.416)
Change at Week 48	Number Analyzed	1 participants	7 participants
Change at Week 48		6.34 ^[1] (NA)	2.11 (1.110)
Change at Week 60	Number Analyzed	0 participants	7 participants
Change at Week 60			2.91 (1.447)
Change at Week 72	Number Analyzed	0 participants	7 participants
Change at Week 72			3.49 (2.084)
Change at Week 84	Number Analyzed	0 participants	7 participants
Change at Week 84			3.63 (2.170)
Change at Week 96	Number Analyzed	0 participants	5 participants
Change at Week 96			5.06 (2.889)
Change at Week 108	Number Analyzed	0 participants	3 participants
Change at Week 108			4.53 (3.415)
Change at Week 120	Number Analyzed	0 participants	3 participants
Change at Week 120			5.17 (3.968)
Change at Week 132	Number Analyzed	0 participants	1 participants
Change at Week 132			5.70 ^[1] (NA)

[1]

1 participant analyzed

8.Secondary Outcome


Title	Post-UX003 Growth Velocity (cm/yr) for Participants With Both Historical Pre-UX003 (Within 2 Years) and Post-UX003 Data
Description	The growth velocity for pre-treatment is based on standing height with...
Description	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.
Time Frame	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)

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title	UX003
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed	5
Mean (Standard Deviation) Unit of Measure: cm/year
	6.20 (1.954)

9.Secondary Outcome


Title	Change From Pre-Treatment (Within 2 Years) to Post-Treatment Growth Velocity Z-Score
Description	The Z-score indicates the number of standard deviations away from a re...
Description	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.
Time Frame	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)

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title	UX003
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed	4
Mean (Standard Deviation) Unit of Measure: Z-score
	2.291 (3.3555)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	UX003
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.2655
	Comments	[Not Specified]
	Method	t-test
	Comments	[Not Specified]

10.Secondary Outcome


Title	Change From Baseline Over Time in Liver Measurement
Description	For all participants (including the participant previously treated wit...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 48, 96, 144

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	6
Mean (Standard Deviation) Unit of Measure: cm
Change at Week 12	Number Analyzed	1 participants	6 participants
Change at Week 12		-0.30 ^[1] (NA)	-0.87 (1.873)
Change at Week 24	Number Analyzed	1 participants	5 participants
Change at Week 24		0.70 ^[1] (NA)	-1.16 (2.701)
Change at Week 48	Number Analyzed	1 participants	6 participants
Change at Week 48		-0.40 ^[1] (NA)	-0.85 (2.160)
Change at Week 96	Number Analyzed	0 participants	6 participants
Change at Week 96			-0.45 (1.868)
Change at Week 144	Number Analyzed	0 participants	2 participants
Change at Week 144			-1.50 (3.394)

[1]

1 participant analyzed

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 12
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0122
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.99
	Confidence Interval	(2-Sided) 95% -1.77 to -0.22
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.396
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 24
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0086
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-1.21
	Confidence Interval	(2-Sided) 95% -2.12 to -0.31
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.461
	Estimation Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 48
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0016
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.98
	Confidence Interval	(2-Sided) 95% -1.58 to -0.37
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.310
	Estimation Comments	[Not Specified]

Statistical Analysis 4

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 96
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.1792
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.57
	Confidence Interval	(2-Sided) 95% -1.41 to 0.26
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.428
	Estimation Comments	[Not Specified]

Statistical Analysis 5

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 144
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.1731
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.35
	Confidence Interval	(2-Sided) 95% -0.85 to 0.15
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.255
	Estimation Comments	[Not Specified]

11.Secondary Outcome


Title	Change From Baseline Over Time in Spleen Measurement
Description	For all participants (including the participant previously treated wit...
Description	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.
Time Frame	Baseline, Weeks 12, 24, 48, 96, 144

Outcome Measure Data

Analysis Population Description

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.


Arm/Group Title		UX003: eIND	UX003: Non-eIND
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatmen...	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description:		UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks. Participant previously treated with UX003 under an emergency Investigational New Drug (eIND) application.	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Overall Number of Participants Analyzed		1	7
Mean (Standard Deviation) Unit of Measure: cm
Change at Week 12	Number Analyzed	1 participants	7 participants
Change at Week 12		0.00 ^[1] (NA)	-0.37 (0.624)
Change at Week 24	Number Analyzed	1 participants	7 participants
Change at Week 24		-0.10 ^[1] (NA)	-0.03 (1.493)
Change at Week 48	Number Analyzed	1 participants	7 participants
Change at Week 48		-1.60 ^[1] (NA)	-0.16 (1.128)
Change at Week 96	Number Analyzed	0 participants	7 participants
Change at Week 96			0.21 (1.056)
Change at Week 144	Number Analyzed	0 participants	2 participants
Change at Week 144			-1.12 (0.686)

[1]

1 participant analyzed

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 12
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0843
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.37
	Confidence Interval	(2-Sided) 95% -0.78 to 0.05
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.213
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 24
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.9618
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.02
	Confidence Interval	(2-Sided) 95% -1.03 to 0.98
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.513
	Estimation Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 48
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.6954
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	GEE model
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.15
	Confidence Interval	(2-Sided) 95% -0.92 to 0.61
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.391
	Estimation Comments	[Not Specified]

Statistical Analysis 4

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 96
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.5492
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	0.22
	Confidence Interval	(2-Sided) 95% -0.50 to 0.93
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.364
	Estimation Comments	[Not Specified]

Statistical Analysis 5

Statistical Analysis Overview	Comparison Group Selection	UX003: Non-eIND
	Comments	Change at Week 144
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.2618
	Comments	From generalized estimating equation model including baseline value and visit as a categorical variable. Change from baseline in Liver Ultrasound (cm) as a dependent variable. Covariance structure within subjects is assumed to be exchangeable.
	Method	generalized estimating equation
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-0.56
	Confidence Interval	(2-Sided) 95% -1.55 to 0.42
	Parameter Dispersion	Type: Standard Error of the Mean Value: 0.503
	Estimation Comments	[Not Specified]

Adverse Events

Time Frame	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.
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	UX003
Arm/Group Description	UX003 4 mg/kg QOW. Initial treatmen...
Arm/Group Description	UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
All-Cause Mortality
	UX003
	Affected / at Risk (%)
Total	0/8 (0.00%)
Serious Adverse Events Serious Adverse Events
	UX003
	Affected / at Risk (%)
Total	3/8 (37.50%)
Cardiac disorders
Bradycardia ^† 1	1/8 (12.50%)
General disorders
Device Fastener Issue ^† 1	1/8 (12.50%)
Infections and infestations
Otitis Media ^† 1	1/8 (12.50%)
Investigations
Oxygen Saturation Decreased ^† 1	1/8 (12.50%)
Musculoskeletal and connective tissue disorders
Cervical Spinal Stenosis ^† 1	1/8 (12.50%)
Spinal Column Stenosis ^† 1	1/8 (12.50%)
Spinal Instability ^† 1	1/8 (12.50%)
Nervous system disorders
Cervical Cord Compression ^† 1	1/8 (12.50%)
Febrile Convulsion ^† 1	1/8 (12.50%)
Respiratory, thoracic and mediastinal disorders
Adenoidal Hypertrophy ^† 1	2/8 (25.00%)
1 Term from vocabulary, MedDRA 18.1 † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	UX003
	Affected / at Risk (%)
Total	8/8 (100.00%)
Blood and lymphatic system disorders
Anaemia ^† 1	1/8 (12.50%)
Lymphadenitis ^† 1	1/8 (12.50%)
Microcytosis ^† 1	1/8 (12.50%)
Thrombocytopenia ^† 1	1/8 (12.50%)
Cardiac disorders
Bradycardia ^† 1	1/8 (12.50%)
Pulmonary Valve Stenosis ^† 1	1/8 (12.50%)
Right Ventricular Hypertension ^† 1	1/8 (12.50%)
Supraventricular Extrasystoles ^† 1	1/8 (12.50%)
Tachycardia ^† 1	1/8 (12.50%)
Congenital, familial and genetic disorders
Mucopolysaccharidosis Vii ^† 1	1/8 (12.50%)
Pectus Carinatum ^† 1	1/8 (12.50%)
Ear and labyrinth disorders
Cerumen Impaction ^† 1	1/8 (12.50%)
Ear Pain ^† 1	2/8 (25.00%)
Eye disorders
Corneal Deposits ^† 1	1/8 (12.50%)
Myopia ^† 1	1/8 (12.50%)
Strabismus ^† 1	1/8 (12.50%)
Gastrointestinal disorders
Abdominal Pain ^† 1	1/8 (12.50%)
Abdominal Pain Upper ^† 1	1/8 (12.50%)
Constipation ^† 1	1/8 (12.50%)
Diarrhoea ^† 1	4/8 (50.00%)
Gingival Bleeding ^† 1	1/8 (12.50%)
Gingival Pain ^† 1	1/8 (12.50%)
Lip Haemorrhage ^† 1	1/8 (12.50%)
Nausea ^† 1	2/8 (25.00%)
Oral Papule ^† 1	1/8 (12.50%)
Vomiting ^† 1	4/8 (50.00%)
General disorders
Device Defective ^† 1	1/8 (12.50%)
Device Malfunction ^† 1	1/8 (12.50%)
Discomfort ^† 1	1/8 (12.50%)
Extravasation ^† 1	1/8 (12.50%)
Inflammation ^† 1	1/8 (12.50%)
Infusion Site Extravasation ^† 1	1/8 (12.50%)
Pyrexia ^† 1	5/8 (62.50%)
Secretion Discharge ^† 1	1/8 (12.50%)
Tenderness ^† 1	1/8 (12.50%)
Hepatobiliary disorders
Hepatomegaly ^† 1	1/8 (12.50%)
Immune system disorders
Anaphylactoid Reaction ^† 1	1/8 (12.50%)
Infections and infestations
Adenoiditis ^† 1	1/8 (12.50%)
Bacterial Disease Carrier ^† 1	1/8 (12.50%)
Device Related Infection ^† 1	1/8 (12.50%)
Ear Infection ^† 1	2/8 (25.00%)
Enterovirus Infection ^† 1	1/8 (12.50%)
Eye Infection ^† 1	1/8 (12.50%)
Fungal Infection ^† 1	1/8 (12.50%)
Fungal Skin Infection ^† 1	1/8 (12.50%)
Gastroenteritis ^† 1	4/8 (50.00%)
Gastroenteritis Viral ^† 1	1/8 (12.50%)
Gastrointestinal Infection ^† 1	1/8 (12.50%)
Gingival Abscess ^† 1	1/8 (12.50%)
Nasopharyngitis ^† 1	1/8 (12.50%)
Otitis Media ^† 1	3/8 (37.50%)
Otitis Media Acute ^† 1	2/8 (25.00%)
Otitis Media Chronic ^† 1	1/8 (12.50%)
Pharyngitis ^† 1	2/8 (25.00%)
Pharyngitis Streptococcal ^† 1	1/8 (12.50%)
Pharyngotonsillitis ^† 1	1/8 (12.50%)
Pneumonia Viral ^† 1	1/8 (12.50%)
Respiratory Tract Infection Viral ^† 1	1/8 (12.50%)
Rhinitis ^† 1	1/8 (12.50%)
Rhinovirus Infection ^† 1	1/8 (12.50%)
Sinusitis ^† 1	2/8 (25.00%)
Tracheitis ^† 1	1/8 (12.50%)
Upper Respiratory Tract Infection ^† 1	3/8 (37.50%)
Injury, poisoning and procedural complications
Contusion ^† 1	1/8 (12.50%)
Fall ^† 1	2/8 (25.00%)
Gastrostomy Tube Site Complication ^† 1	1/8 (12.50%)
Limb Injury ^† 1	1/8 (12.50%)
Procedural Pain ^† 1	1/8 (12.50%)
Scar ^† 1	1/8 (12.50%)
Scratch ^† 1	1/8 (12.50%)
Skin Abrasion ^† 1	2/8 (25.00%)
Sunburn ^† 1	1/8 (12.50%)
Investigations
Alanine Aminotransferase Increased ^† 1	1/8 (12.50%)
Blood Calcium Decreased ^† 1	1/8 (12.50%)
Blood Creatinine Decreased ^† 1	1/8 (12.50%)
Blood Pressure Increased ^† 1	1/8 (12.50%)
Body Temperature Increased ^† 1	1/8 (12.50%)
Body Temperature Abnormal ^† 1	1/8 (12.50%)
Gamma-Glutamyltransferase Increased ^† 1	1/8 (12.50%)
Hepatic Enzyme Increased ^† 1	2/8 (25.00%)
Oxygen Saturation Decreased ^† 1	2/8 (25.00%)
Right Ventricular Systolic Pressure Increased ^† 1	1/8 (12.50%)
Vitamin D Decreased ^† 1	1/8 (12.50%)
Musculoskeletal and connective tissue disorders
Arthralgia ^† 1	2/8 (25.00%)
Bone Deformity ^† 1	1/8 (12.50%)
Muscle Spasms ^† 1	1/8 (12.50%)
Muscular Weakness ^† 1	1/8 (12.50%)
Musculoskeletal Chest Pain ^† 1	1/8 (12.50%)
Musculoskeletal Pain ^† 1	1/8 (12.50%)
Pain In Extremity ^† 1	2/8 (25.00%)
Spinal Deformity ^† 1	1/8 (12.50%)
Nervous system disorders
Cerebellar Microhaemorrhage ^† 1	1/8 (12.50%)
Enlarged Cerebral Perivascular Spaces ^† 1	1/8 (12.50%)
Headache ^† 1	1/8 (12.50%)
Lethargy ^† 1	1/8 (12.50%)
Myelopathy ^† 1	1/8 (12.50%)
Periodic Limb Movement Disorder ^† 1	1/8 (12.50%)
Psychiatric disorders
Attention Deficit/Hyperactivity Disorder ^† 1	1/8 (12.50%)
Irritability ^† 1	1/8 (12.50%)
Sleep Disorder ^† 1	1/8 (12.50%)
Renal and urinary disorders
Bladder Disorder ^† 1	1/8 (12.50%)
Haematuria ^† 1	1/8 (12.50%)
Respiratory, thoracic and mediastinal disorders
Asthma ^† 1	1/8 (12.50%)
Cough ^† 1	3/8 (37.50%)
Hypoxia ^† 1	1/8 (12.50%)
Nasal Congestion ^† 1	2/8 (25.00%)
Nasal Mucosal Disorder ^† 1	1/8 (12.50%)
Oropharyngeal Pain ^† 1	1/8 (12.50%)
Pneumonia Aspiration ^† 1	1/8 (12.50%)
Rhinitis Allergic ^† 1	1/8 (12.50%)
Rhinorrhoea ^† 1	3/8 (37.50%)
Tonsillar Hypertrophy ^† 1	1/8 (12.50%)
Upper Respiratory Tract Congestion ^† 1	1/8 (12.50%)
Wheezing ^† 1	1/8 (12.50%)
Skin and subcutaneous tissue disorders
Alopecia ^† 1	1/8 (12.50%)
Blood Blister ^† 1	1/8 (12.50%)
Dermatitis ^† 1	1/8 (12.50%)
Ecchymosis ^† 1	1/8 (12.50%)
Intertrigo ^† 1	1/8 (12.50%)
Keloid Scar ^† 1	1/8 (12.50%)
Papule ^† 1	1/8 (12.50%)
Rash ^† 1	1/8 (12.50%)
Rash Papular ^† 1	1/8 (12.50%)
Rash Pruritic ^† 1	1/8 (12.50%)
Skin Irritation ^† 1	1/8 (12.50%)
Urticaria ^† 1	1/8 (12.50%)
Vascular disorders
Hypotension ^† 1	1/8 (12.50%)
1 Term from vocabulary, MedDRA 18.1 † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

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.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Medical Information
Organization:	Ultragenyx Pharmaceutical Inc
Phone:	1-888-756-8567
EMail:	medinfo@ultragenyx.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

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. Erratum In: Clin Pharmacokinet. 2018 Dec 4;:

Layout table for additonal information

Responsible Party:	Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier:	NCT02418455
Other Study ID Numbers:	UX003-CL203 2015-000104-26 ( EudraCT Number )
First Submitted:	April 12, 2015
First Posted:	April 16, 2015
Results First Submitted:	September 22, 2019
Results First Posted:	October 16, 2019
Last Update Posted:	October 30, 2019

To Top