Trial Outcomes & Findings for A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) (NCT NCT02432144)

Last Updated: 2020-07-30

Results Overview

An adverse event (AE) is defined as any untoward medical occurrence, whether or not considered drug related. A serious AE is an AE 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; or is an important medical event. AEs were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.0: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life-threatening), Grade 5 (death). TEAEs were defined as reported AEs with onset during the treatment.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

12 participants

Primary outcome timeframe

From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks.

Results posted on

2020-07-30

Participant Flow

Participants with mucopolysaccharidosis VII (MPS 7) who were UX003 treatment-naïve or previously enrolled and treated in a prior clinical study of UX003 could enroll into this treatment and extension study provided all eligibility criteria had been met for a given participant.

Ten of 12 participants entered this extension study at study Week 0 with ongoing UX003 treatment for the prior 24 or 48 weeks in study UX003-CL301 \[NCT02230566\]; 2 participants had a large gap between studies (61 weeks between doses).

Participant milestones

Participant milestones
Measure	UX003 4 mg/kg UX003 every other week (QOW)
Overall Study STARTED	12
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	UX003 4 mg/kg UX003 every other week (QOW)
Overall Study Participant Non-Compliance	1

Baseline Characteristics

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	UX003 n=12 Participants 4 mg/kg UX003 QOW
Age, Continuous	16.56 years STANDARD_DEVIATION 5.466 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized White	9 Participants n=5 Participants
Race/Ethnicity, Customized Other, Not Specified	3 Participants n=5 Participants
Urinary Glycosaminoglycans (uGAG)	1.54848 g GAG/g creatinine STANDARD_DEVIATION 0.413237 • n=5 Participants

PRIMARY outcome

Timeframe: From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks.

Population: Full Analysis Set: all enrolled participants who received at least one dose of investigational product in this study.

Outcome measures

Outcome measures
Measure	UX003 n=12 Participants 4 mg/kg UX003 QOW
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation Treatment-Related TEAEs	9 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation TEAEs	12 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation Serious TEAEs	4 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation Treatment-Related Serious TEAEs	1 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation Grade 3 or 4 TEAEs	3 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation TEAEs Leading to Treatment Discontinuation	0 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation TEAEs Leading to Study Discontinuation	0 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation TEAEs Leading to Death	0 Participants

SECONDARY outcome

Timeframe: Baseline (prior to the first dose of study drug in UX003-CL301), Weeks 0, 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144

Population: Full Analysis Set: all enrolled participants who received at least one dose of investigational product in this study; participants with an assessment at given time point.

First morning void urine was evaluated for uGAG concentration and normalized to urinary creatinine concentration.

Outcome measures

Outcome measures
Measure	UX003 n=12 Participants 4 mg/kg UX003 QOW
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 72	-78.52 percentage change in uGAG excretion Standard Deviation 10.367
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 0	-62.19 percentage change in uGAG excretion Standard Deviation 16.133
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 12	-67.31 percentage change in uGAG excretion Standard Deviation 13.953
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 24	-64.03 percentage change in uGAG excretion Standard Deviation 14.669
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 36	-60.58 percentage change in uGAG excretion Standard Deviation 23.552
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 48	-57.04 percentage change in uGAG excretion Standard Deviation 23.611
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 60	-72.25 percentage change in uGAG excretion Standard Deviation 18.609
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 84	-80.89 percentage change in uGAG excretion Standard Deviation 10.023
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 96	-82.39 percentage change in uGAG excretion Standard Deviation 6.011
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 108	-82.19 percentage change in uGAG excretion Standard Deviation 6.551
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 120	-88.74 percentage change in uGAG excretion Standard Deviation 4.023
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 132	-89.22 percentage change in uGAG excretion Standard Deviation 3.662
Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate) Week 144	-91.62 percentage change in uGAG excretion Standard Deviation 1.827

Adverse Events

UX003

Serious events: 4 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	UX003 n=12 participants at risk 4 mg/kg UX003 QOW
Infections and infestations Gastroenteritis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Head Injury	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Headache	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Asthmatic Crisis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Bronchospasm	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Interstitial Lung Disease	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Urticaria	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.

Other adverse events

Other adverse events
Measure	UX003 n=12 participants at risk 4 mg/kg UX003 QOW
Blood and lymphatic system disorders Eosinophilia	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Cardiac disorders Pericardial Effusion	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Cardiac disorders Tachycardia	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Ear and labyrinth disorders Ear Pain	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Ear and labyrinth disorders Otorrhoea	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Eye disorders Conjunctivitis Allergic	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Eye disorders Eye Pruritus	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Eye disorders Lacrimation Increased	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Aphthous Ulcer	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Diarrhoea	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Dyspepsia	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Gastrooesophageal Reflux Disease	25.0% 3/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Gingival Bleeding	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Haematochezia	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Lip Ulceration	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Nausea	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Oesophagitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Tooth Discolouration	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Tooth Loss	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Toothache	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Gastrointestinal disorders Vomiting	33.3% 4/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Chest Pain	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Fatigue	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Gait Disturbance	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Infusion Site Extravasation	41.7% 5/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Infusion Site Swelling	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Oedema Peripheral	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
General disorders Pyrexia	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Immune system disorders Seasonal Allergy	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Abscess Neck	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Acarodermatitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Conjunctivitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Ear Infection	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Folliculitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Furuncle	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Impetigo	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Nasopharyngitis	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Otitis Externa	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Otitis Media	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Otitis Media Acute	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Pharyngitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Rhinitis	25.0% 3/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Root Canal Infection	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Sinusitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Soft Tissue Infection	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Tinea Pedis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Tooth Infection	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Upper Respiratory Tract Infection	58.3% 7/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Infections and infestations Viral Infection	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Arthropod Bite	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Fall	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Head Injury	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Laceration	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Ligament Sprain	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Injury, poisoning and procedural complications Skin Abrasion	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Arthralgia	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Back Pain	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Dactylitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Joint Range Of Motion Decreased	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Joint Stiffness	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Joint Swelling	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Muscle Twitching	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Pain In Extremity	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Scoliosis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Spinal Instability	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Musculoskeletal and connective tissue disorders Trigger Finger	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acrochordon	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin Papilloma	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Brain Compression	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Cervical Cord Compression	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Headache	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Hydrocephalus	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Lethargy	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Memory Impairment	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Paraesthesia	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Sensory Disturbance	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Nervous system disorders Sinus Headache	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Psychiatric disorders Insomnia	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Psychiatric disorders Somnambulism	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Renal and urinary disorders Pollakiuria	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Asthma	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Bronchospasm	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Cough	33.3% 4/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Epistaxis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	25.0% 3/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Rhinitis Allergic	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Sinus Congestion	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Sleep Apnoea Syndrome	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Tract Congestion	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Dermatitis	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Dermatitis Atopic	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Erythema	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Macule	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Papule	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Pruritus	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Rash	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Rash Maculo-Papular	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Rash Papular	16.7% 2/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Rash Pruritic	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Skin and subcutaneous tissue disorders Urticaria	33.3% 4/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Vascular disorders Hypertension	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.
Vascular disorders Hypotension	8.3% 1/12 • From first dose of study drug until 30 days after the last dose of study drug. Mean duration of UX003 treatment was 100.5 weeks. TEAEs, defined as reported AEs with onset during the treatment, are presented.

Additional Information

Medical Information

Ultragenyx Pharmaceutical Inc

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Principal investigator is a sponsor employee 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.
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