An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

• ClinicalTrials.gov Identifier: NCT01856218
• Recruitment Status: Completed
• First Posted: May 17, 2013
• Results First Posted: January 5, 2018
• Last Update Posted: January 4, 2019
• Sponsor: Ultragenyx Pharmaceutical Inc
• 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
• Condition: Mucopolysaccharidosis Type 7
• Intervention: Drug: UX003
• Enrollment: 3

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	UX003
Arm/Group Description	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 every other week (QOW) for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Period Title: First Phase
Started	3
Completed	3
Not Completed	0
Period Title: Long-Term Extension Phase
Started	3
Completed	3
Not Completed	0

Baseline Characteristics

Arm/Group Title		UX003
Arm/Group Description		During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Baseline Participants		3
Baseline Analysis Population Description		[Not Specified]
Age, Customized; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	3 participants
Children (2-11 years)		2 66.7%
Adults (18-64 years)		1 33.3%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants
	Female	1 33.3%
	Male	2 66.7%

Outcome Measures

1. Primary Outcome

Title	Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate
Description	Percentage change from baseline in the concentration of uGAGs normalized to the urinary creatinine concentration as measured by liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate.
Time Frame	Baseline, Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	3
Mean (Standard Deviation); Unit of Measure: percentage change
Initial treatment-Week 14	-50.41 (7.895)
Forced dose titration-Week 22	-38.94 (7.137)
Forced dose titration-Week 30	-63.49 (6.889)
Forced dose titration-Week 38	-51.82 (9.033)
Continuation-Week 72	-54.2 (8.442)
Long term extension-end of study	-34.44 (48.56)

2. Primary Outcome

Title	Percentage Change From Baseline in uGAG Chondroitin Sulfate
Description	Percentage change from baseline in the concentration of uGAGs normalized to the urinary creatinine concentration as measured by liquid chromatography-mass spectrometry/mass spectrometry-chondroitin sulfate.
Time Frame	Baseline, Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	3
Mean (Standard Deviation); Unit of Measure: percentage change
Initial treatment-Week 14	-52.5 (13.726)
Forced dose titration-Week 22	-47.58 (3.958)
Forced dose titration-Week 30	-60.78 (7.207)
Forced dose titration-Week 38	-52.08 (13.298)
Continuation-Week 72	-56.96 (13.379)
Long term extension-end of study	-30.83 (40.794)

3. Primary Outcome

Title	Number of Participants With Any ≥ 50% Decrease in uGAG
Description	Participants with a ≥ 50% decrease in the concentration of uGAGs normalized to the urinary creatinine concentration as measured by liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate or chondroitin sulfate.
Time Frame	up to Week 132

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	3
Measure Type: Count of Participants; Unit of Measure: Participants
	3 100.0%

4. Primary Outcome

Title	Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Study/Treatment Discontinuations
Description	Adverse Event (AE): any untoward medical occurrence in a subject, whether or not considered drug related. 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	Up to 242 weeks + 30 days. SAEs were recorded beginning at the time the subject signed the informed consent form through 30 days following the last study visit. Non-serious AEs were recorded from the time of informed consent through the last study visit.

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	3
Measure Type: Number; Unit of Measure: participants
Any TEAE	3
Treatment-related TEAE	2
SAE	2
Grade 3 or 4 TEAE	2
TEAE leading to treatment discontinuation	1
TEAE leading to study discontinuation	0
Fatal TEAE	0

5. Secondary Outcome

Title	Acceptable Dose as Determined by Total uGAG Excretion Using a Forced Dose Titration Regimen
Description	The choice of the dose of UX003 QOW for the Long-Term Extension Phase was based on a preliminary efficacy analysis at Week 36 prior to all 3 participants completing the Forced-dose Titration Period of the First Phase of the study.
Time Frame	Week 36

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	3
Measure Type: Number; Unit of Measure: mg/kg
	4

6. Secondary Outcome

Title	Change From Baseline at Week 36 in Six-Minute Walk Test (6MWT)
Description	The total distance walked (meters) in a 6-minute period was measured once each test day. The test was conducted using a pre-measured walking course according to administration guidelines established by the American Thoracic Society (ATS 2002). Participants who could not walk could omit this test.
Time Frame	Baseline, Week 36

Outcome Measure Data

Analysis Population Description

Summary analyses for all 3 participants are not available due to the very small number of participants; and while data were collected for 1 participant, they are not being reported due to confidentiality issues.

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

7. Secondary Outcome

Title	Percent of Predicted Normal Distance Walked
Description	The percent of predicted normal distance walked (based on published normative data) in the total distance walked in a six-minute period.
Time Frame	36 Weeks

Outcome Measure Data

Analysis Population Description

Summary analyses for all 3 participants are not available due to the very small number of participants; and while data were collected for 1 participant, they are not being reported due to confidentiality issues.

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

8. Secondary Outcome

Title	Change From Baseline at Week 36 in the 3-Minute Stair Climb Test (3MSCT)
Description	The number of stairs climbed within a 3-minute period was assessed once each test day using available hospital stairs. Participants who could not climb stairs could omit this test.
Time Frame	Baseline, Week 36

Outcome Measure Data

Analysis Population Description

Summary analyses for all 3 participants are not available due to the very small number of participants; and while data were collected for 1 participant, they are not being reported due to confidentiality issues.

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

9. Secondary Outcome

Title	Change From Baseline at Week 36 in Pulmonary Function Testing (Spirometry)
Description	The following spirometry tests were administered to participants who did not require invasive ventilatory support or have a tracheostomy in accordance with American Thoracic Society/European Respiratory society (ATS/ERS) guidelines: forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1), maximum voluntary ventilation in one minute (MVV1). Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.
Time Frame	Baseline, Week 36

Outcome Measure Data

Analysis Population Description

Summary analyses for all 3 participants are not available due to the very small number of participants; and while data were collected for 1 participant, they are not being reported due to confidentiality issues.

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

10. Secondary Outcome

Title	Change From Baseline at Week 36 in Growth Velocity for Height and Weight
Description	Growth velocity (for males ≤18 years and females ≤15) was calculated from anthropometric measurements and compared with pretreatment growth velocity when available. Z-scores and percentiles were calculated using Centers for Disease Control (CDC) growth chart.
Time Frame	Baseline, Week 36

Outcome Measure Data

Analysis Population Description

Summary analyses for all 3 participants combined are not available. Due to the very small number of participants, formal statistical analyses were not performed; and data were presented per participant, which has the potential for participant re-identification given the rarity of disease and the very small population size.

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

11. Secondary Outcome

Title	Change From Baseline at Week 36 in Shoulder Range of Motion (Goniometry)
Description	The maximum passive shoulder range of motion in both flexion and extension will be measured in degrees using a goniometer.
Time Frame	Baseline, Week 36

Outcome Measure Data

Analysis Population Description

Summary analyses for all 3 participants are not available due to the very small number of participants; and while data were collected for 1 participant, they are not being reported due to confidentiality issues.

Arm/Group Title	UX003
Arm/Group Description:	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

Adverse Events

Time Frame	Up to 242 weeks + 30 days. SAEs were recorded beginning at the time the subject signed the informed consent form through 30 days following the last study visit. Non-serious AEs were recorded from the time of informed consent through the last study visit.
Adverse Event Reporting Description	TEAEs are presented. 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.
Arm/Group Title	UX003
Arm/Group Description	During the initial 14-week treatment period of the study, participants received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, participants continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: 1 mg/kg UX003 for 8 weeks beginning on Week 14; then 4 mg/kg UX003 for 8 weeks beginning on Week 22; then 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, participants who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the first phase of the study, participants who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
All-Cause Mortality
	UX003
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	UX003
	Affected / at Risk (%)
Total	2/3 (66.67%)
Gastrointestinal disorders
Incarcerated inguinal hernia † 1	1/3 (33.33%)
General disorders
Oedema peripheral † 1	1/3 (33.33%)
Musculoskeletal and connective tissue disorders
Osteoarthritis † 1	1/3 (33.33%)
Nervous system disorders
Spinal cord compression † 1	1/3 (33.33%)
Cerebral ventricle dilatation † 1	1/3 (33.33%)
Surgical and medical procedures
Inguinal hernia repair † 1	1/3 (33.33%)
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 17.1
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	UX003
	Affected / at Risk (%)
Total	3/3 (100.00%)
Blood and lymphatic system disorders
Anaemia † 1	1/3 (33.33%)
Gastrointestinal disorders
Constipation † 1	1/3 (33.33%)
Diarrhoea † 1	2/3 (66.67%)
Oral mucosal erythema † 1	1/3 (33.33%)
Toothache † 1	1/3 (33.33%)
Vomiting † 1	2/3 (66.67%)
General disorders
Discomfort † 1	1/3 (33.33%)
Gait disturbance † 1	1/3 (33.33%)
Infusion site extravasation † 1	1/3 (33.33%)
Oedema peripheral † 1	1/3 (33.33%)
Pyrexia † 1	1/3 (33.33%)
Generalised Oedema † 1	1/3 (33.33%)
Infections and infestations
Acute sinusitis † 1	1/3 (33.33%)
Dermatitis infected † 1	1/3 (33.33%)
Ear infection † 1	1/3 (33.33%)
Molluscum contagiosum † 1	1/3 (33.33%)
Nasopharyngitis † 1	1/3 (33.33%)
Nosocomial infection † 1	1/3 (33.33%)
Otitis media † 1	2/3 (66.67%)
Pharyngitis † 1	2/3 (66.67%)
Respiratory tract infection † 1	1/3 (33.33%)
Tonsillitis † 1	1/3 (33.33%)
Tooth abscess † 1	1/3 (33.33%)
Upper respiratory tract infection † 1	2/3 (66.67%)
Urinary tract infection † 1	1/3 (33.33%)
Viral rash † 1	1/3 (33.33%)
Injury, poisoning and procedural complications
Ligament sprain † 1	1/3 (33.33%)
Skin abrasion † 1	1/3 (33.33%)
Investigations
Body temperature increased † 1	1/3 (33.33%)
Weight increased † 1	2/3 (66.67%)
Metabolism and nutrition disorders
Increased appetite † 1	1/3 (33.33%)
Musculoskeletal and connective tissue disorders
Arthralgia † 1	3/3 (100.00%)
Groin pain † 1	1/3 (33.33%)
Musculoskeletal pain † 1	1/3 (33.33%)
Musculoskeletal stiffness † 1	1/3 (33.33%)
Myalgia † 1	1/3 (33.33%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papilloma † 1	1/3 (33.33%)
Nervous system disorders
Dizziness † 1	1/3 (33.33%)
Nystagmus † 1	1/3 (33.33%)
Reflexes abnormal † 1	1/3 (33.33%)
Visual field defect † 1	1/3 (33.33%)
Respiratory, thoracic and mediastinal disorders
Bronchospasm † 1	1/3 (33.33%)
Cough † 1	2/3 (66.67%)
Increased viscosity of bronchial secretion † 1	1/3 (33.33%)
Nasal congestion † 1	1/3 (33.33%)
Oropharyngeal pain † 1	1/3 (33.33%)
Respiratory failure † 1	1/3 (33.33%)
Rhinitis allergic † 1	2/3 (66.67%)
Rhinorrhoea † 1	1/3 (33.33%)
Skin and subcutaneous tissue disorders
Decubitus ulcer † 1	1/3 (33.33%)
Erythema † 1	1/3 (33.33%)
Pruritus † 1	1/3 (33.33%)
Rash † 1	1/3 (33.33%)
Rash macular † 1	1/3 (33.33%)
Seborrhoeic dermatitis † 1	1/3 (33.33%)
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 17.1

Limitations and Caveats

This study was terminated due to business considerations unrelated to safety or efficacy concerns.

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.

Results Point of Contact

• Name/Title: Kim Mooney, Associate Director, Patient Advocacy Medical Services
• Organization: Ultragenyx Pharmaceutical Inc
• Phone: 408-981-3526
• EMail: kmooney@ultragenyx.com

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

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;:

• Responsible Party: Ultragenyx Pharmaceutical Inc
• ClinicalTrials.gov Identifier: NCT01856218
• Other Study ID Numbers: UX003-CL201
• First Submitted: May 8, 2013
• First Posted: May 17, 2013
• Results First Submitted: November 29, 2017
• Results First Posted: January 5, 2018
• Last Update Posted: January 4, 2019