An Open-Label Phase 1/2 Study to Assess the Safety, Effic... | NCT01856218

NCT01856218

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
UX003	Experimental	During the initial 14-week treatment period of the study, participants receive 2 mg/kg UX003 every other week (QOW) for 12 weeks. At Week 14, participants continue on UX003 therapy and begin 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 continue 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 elect to continue drug treatment are transitioned to the long-term extension phase, where they are treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.	Drug: UX003

Name	Type	Description	Arm Group Labels	Other Names
UX003	Drug		UX003

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate	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.	Baseline, Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)
Percentage Change From Baseline in uGAG Chondroitin Sulfate	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.	Baseline, Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)
Number of Participants With Any ≥ 50% Decrease in uGAG	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.	up to Week 132
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Study/Treatment Discontinuations	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.

Secondary Outcomes

Measure	Description	Time Frame
Acceptable Dose as Determined by Total uGAG Excretion Using a Forced Dose Titration Regimen	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.	Week 36

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
Elevated urinary glycosaminoglycan (uGAG) excretion at a minimum of 2-fold over normal.
Between 5 and 30 years of age, inclusive (approximately 2 subjects between the ages of 20-30 years).
Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.
Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have had tubal ligation at least one year prior to Screening, or who have had total hysterectomy.

Exclusion Criteria:

Has undergone a successful bone marrow or stem cell transplant or has any degree of detectable chimaerism with donor cells.
Any known hypersensitivity to rhGUS or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
Use of any investigational product (drug or device or combination) within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments at any time during the study.
Has a condition of such severity and acuity, in the opinion of the Investigator, that it warrants immediate surgical intervention or other treatment or may not allow safe study participation.
Has a concurrent disease or condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or affect safety.

Contacts/Locations Module

Name	Affiliation	Role
Simon Jones, MD	Univeristy of Manchester	Principal Investigator

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
30467742	Derived	Qi Y, McKeever K, Taylor J, Haller C, Song W, Jones SA, Shi J. Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials. Clin Pharmacokinet. 2019 May;58(5):673-683. doi: 10.1007/s40262-018-0721-y.

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	UX003	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.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

No data available

No data is available for this block.

Masking Info

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	UX003	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.

Units	Counts
Participants	BG0003

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Customized

Count of Participants

Participants

Title

Denominators

Categories

Initial treatment-Week 14

Title	Measurements
	OG000-50.41± 7.895

Forced dose titration-Week 22

Title	Measurements
	OG000-38.94± 7.137

Forced dose titration-Week 30

Primary

Percentage Change From Baseline in uGAG Chondroitin Sulfate

Posted

Mean

Standard Deviation

percentage change

Baseline, Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)

ID	Title	Description
OG000	UX003	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.

Units	Counts
Participants

Primary

Number of Participants With Any ≥ 50% Decrease in uGAG

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.

Posted

Count of Participants

Participants

up to Week 132

ID	Title	Description
OG000	UX003	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.

Units	Counts
Participants

Primary

Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Study/Treatment Discontinuations

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.

Posted

Number

participants

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.

ID	Title	Description
OG000	UX003	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.

Secondary

Acceptable Dose as Determined by Total uGAG Excretion Using a Forced Dose Titration Regimen

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.

Posted

Number

mg/kg

Week 36

ID	Title	Description
OG000	UX003	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.

Units	Counts
Participants

Secondary

Change From Baseline at Week 36 in Six-Minute Walk Test (6MWT)

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.

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.

Posted

Baseline, Week 36

ID	Title	Description
OG000	UX003	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.

Secondary

Percent of Predicted Normal Distance Walked

The percent of predicted normal distance walked (based on published normative data) in the total distance walked in a six-minute period.

Posted

36 Weeks

ID	Title	Description
OG000	UX003	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.

Units	Counts
Participants

Secondary

Change From Baseline at Week 36 in the 3-Minute Stair Climb Test (3MSCT)

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.

Posted

Baseline, Week 36

ID	Title	Description
OG000	UX003	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.

Units	Counts

Secondary

Change From Baseline at Week 36 in Pulmonary Function Testing (Spirometry)

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.

Posted

Baseline, Week 36

ID	Title	Description
OG000	UX003	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.

Secondary

Change From Baseline at Week 36 in Growth Velocity for Height and Weight

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.

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.

Posted

Baseline, Week 36

ID	Title	Description
OG000	UX003	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.

Secondary

Change From Baseline at Week 36 in Shoulder Range of Motion (Goniometry)

The maximum passive shoulder range of motion in both flexion and extension will be measured in degrees using a goniometer.

Posted

Baseline, Week 36

ID	Title	Description
OG000	UX003	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.

Units	Counts
Participants

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	UX003	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.			2	3	3	3

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Inguinal hernia repair	Surgical and medical procedures	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Spinal cord compression	Nervous system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Cerebral ventricle dilatation	Nervous system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Oedema peripheral	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Incarcerated inguinal hernia	Gastrointestinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Ligament sprain	Injury, poisoning and procedural complications	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Skin abrasion	Injury, poisoning and procedural complications	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Body temperature increased	Investigations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Weight increased	Investigations	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Papilloma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Bronchospasm	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Increased viscosity of bronchial secretion	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Rhinitis allergic	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Dizziness	Nervous system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Nystagmus	Nervous system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Reflexes abnormal	Nervous system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Visual field defect	Nervous system disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Discomfort	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Gait disturbance	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Infusion site extravasation	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Oedema peripheral	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Pyrexia	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Generalised Oedema	General disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Constipation	Gastrointestinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Oral mucosal erythema	Gastrointestinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Toothache	Gastrointestinal disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Vomiting	Gastrointestinal disorders	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Decubitus ulcer	Skin and subcutaneous tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Rash macular	Skin and subcutaneous tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Seborrhoeic dermatitis	Skin and subcutaneous tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 17.1	Systematic Assessment	EG0003 affected3 at risk
Groin pain	Musculoskeletal and connective tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Musculoskeletal stiffness	Musculoskeletal and connective tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Increased appetite	Metabolism and nutrition disorders	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Acute sinusitis	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Dermatitis infected	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Ear infection	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Molluscum contagiosum	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Nasopharyngitis	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Nosocomial infection	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Otitis media	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Pharyngitis	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Respiratory tract infection	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Tonsillitis	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Tooth abscess	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0002 affected3 at risk
Urinary tract infection	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk
Viral rash	Infections and infestations	MedDRA 17.1	Systematic Assessment	EG0001 affected3 at risk

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

Certain Agreements

ID	Term
D016538	Mucopolysaccharidosis VII
D035583	Rare Diseases
D016464	Lysosomal Storage Diseases
D008659	Metabolic Diseases

Ancestor Terms

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D017520	Mucinoses
D003240	Connective Tissue Diseases
D017437	Skin and Connective Tissue Diseases
D009750	Nutritional and Metabolic Diseases
D020969	Disease Attributes
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms

Title	Measurements
	OG000-63.49± 6.889

Units	Counts
Participants	OG0000