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Trial record 6 of 29 for:    GUSB

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)

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: NCT01856218
Recruitment Status : Completed
First Posted : May 17, 2013
Results First Posted : January 5, 2018
Last Update Posted : January 4, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Mucopolysaccharidosis Type 7
Intervention Drug: UX003
Enrollment 3
Recruitment Details  
Pre-assignment Details  
Arm/Group Title UX003
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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
Arm/Group Title UX003
Hide 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
Hide 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%
1.Primary Outcome
Title Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title UX003
Hide 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
Hide 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)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title UX003
Hide 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
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title UX003
Hide 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
Hide 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.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title UX003
Hide 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
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title UX003
Hide 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)
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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.
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
Hide 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   --/-- 
Show Serious Adverse Events Hide 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
Show Other (Not Including Serious) Adverse Events Hide 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
This study was terminated due to business considerations unrelated to safety or efficacy concerns.
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
Layout table for Results Point of Contact information
Name/Title: Kim Mooney, Associate Director, Patient Advocacy Medical Services
Organization: Ultragenyx Pharmaceutical Inc
Phone: 408-981-3526
EMail: kmooney@ultragenyx.com
Layout table for additonal information
Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT01856218     History of Changes
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