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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: |
NCT02432144 |
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Date of registration:
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22/04/2015 |
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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 Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
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Scientific title:
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A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7 |
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Date of first enrolment:
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November 10, 2015 |
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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/NCT02432144 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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Brazil
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Mexico
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Portugal
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United States
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Contacts
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Name:
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Medical Director |
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Address:
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Telephone:
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Email:
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Affiliation:
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Ultragenyx Pharmaceuticals, Inc. |
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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.
- 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.
- Willing and able to comply with all study 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 Baseline 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 completion of the primary study, or have had
total hysterectomy.
- For UX003 treatment-naïve subjects only, 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.
- For UX003 treatment-naïve subjects only, elevated urinary glycosaminoglycans (uGAG)
excretion at a minimum of 2-fold over normal.
- For UX003 treatment-naïve subjects only, aged 5 years and older.
Exclusion Criteria:
- If enrolled in a prior UX003 clinical study, the subject experienced safety-related
event(s) in the prior UX003 clinical study that, in the opinion of the Investigator
and sponsor, precludes resuming UX003 treatment.
- Undergone a successful bone marrow or stem cell transplant or has any degree of
detectable chimaerism with donor cells.
- 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 Baseline or planning to become pregnant (self or partner)
at any time during the study.
- Other than the use of UX003, use of any investigational product (drug or device or
combination) within 30 days prior to Baseline, 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 study participation.
- 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:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis VII
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Mucopolysaccharidosis
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MPS VII
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Sly Syndrome
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Intervention(s)
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Drug: UX003
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Primary Outcome(s)
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Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
[Time Frame: 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.]
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Secondary Outcome(s)
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Percent Change From Baseline Over Time in Urinary Glycosaminoglycan (uGAG) Excretion (Liquid Chromatography-Tandem Mass Spectrometry, Dermatan Sulfate)
[Time Frame: 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]
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Secondary ID(s)
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UX003-CL202
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2015-001875-32
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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