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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02230566 |
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Date of registration:
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22/08/2014 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
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Scientific title:
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A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7 |
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Date of first enrolment:
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December 2014 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02230566 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 3
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Countries of recruitment
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United States
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Contacts
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Name:
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Raymond Wang, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital of Orange County |
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Name:
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Mislen Bauer, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Nicklaus Children's Hospital f/k/a Miami Children's Hospital |
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Name:
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Paul Harmatz, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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UCSF Benioff Children's Hospital Oakland |
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Name:
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Chester Whitley, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Minnesota |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme
assay or genetic testing.
- Elevated urinary glycosaminoglycan (uGAG) excretion at a minimum of 3-fold over the
mean normal for age (at Screening).
- Apparent clinical signs of lysosomal storage disease as judged by the Investigator,
including at least one of the following: enlarged liver and spleen, joint limitations,
airway obstruction or pulmonary problems, limitation of mobility while still
ambulatory.
- Aged 5 - 35 years, inclusive.
- Willing and able to provide written 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 acceptable highly effective methods of
contraception while participating in the study and for 30 days following the last
dose.
- 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 not experienced menarche, or have had
tubal ligation at least one year prior to Screening, or who have had total
hysterectomy.
- Naïve to treatment with UX003.
Exclusion Criteria:
- Undergone a successful bone marrow or stem cell transplant or has any degree of
detectable chimaerism with donor cells.
- Major surgery within 3 months prior to study entry or planned major surgery during the
study that may not allow safe participation in the study.
- Presence or history of any 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.
- Presence of a condition of such severity and acuity that, in the opinion of the
Investigator, warrants immediate surgical intervention or other treatment or may not
allow safe participation in the study.
- Concurrent disease or condition, or laboratory abnormality 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 introduce
additional safety concerns.
Age minimum:
5 Years
Age maximum:
35 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Sly Syndrome
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Mucopolysaccharidosis
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MPS 7
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MPS VII
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Intervention(s)
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Other: Placebo
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Drug: UX003
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Primary Outcome(s)
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European Union (EU) and Rest of World: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate (DS) at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the average of all assessments prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Secondary Outcome(s)
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Change From Baseline in Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Scores at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in Impactful Clinical Problem Total Score at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Multi-Domain Responder Index (MDRI) Score at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in 6-Minute Walk Test (6MWT) at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in Uncorrected Visual Acuity at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in Shoulder Flexion and Extension Maximum Range of Motion at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Percentage of Individual Clinical Response (ICR) Responders at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in Pulmonary Function Testing: Percentage of Predicted Forced Vital Capacity (FVC%Pred) at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Change From Baseline in Pulmonary Function Testing: Maximum Ventilatory Ventilation (MVV) at UX003 Treatment Week 24
[Time Frame: Baseline (defined as the last assessment prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment]
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Secondary ID(s)
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UX003-CL301
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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