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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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19 October 2017 |
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Main ID: |
NCT00884949 |
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Date of registration:
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10/04/2009 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
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Scientific title:
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A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) |
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Date of first enrolment:
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April 2009 |
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Target sample size:
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20 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00884949 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United Kingdom
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Contacts
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Name:
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Celeste Decker, 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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BioMarin Pharmceutical Inc. |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Documented history of reduced GALNS activity relative to the normal range of the
laboratory performing the assay, or documented result of molecular genetic testing
confirming diagnosis of MPS IVA.
- Willing and able to provide written, signed informed consent, or in the case of
subjects under the age of 16 years, 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.
- Between 5 and 18 years of age, inclusive.
- 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.
- Willing to perform all study procedures as physically possible.
Exclusion Criteria:
- Previous hematopoietic stem cell transplant (HSCT).
- Has known hypersensitivity to BMN 110 or its excipients.
- Pregnant or breastfeeding at Screening or planning to become pregnant (self or
partner) at any time during the study.
- Use of any investigational product or investigational medical device within 30 days
prior to Screening, or requirement for any investigational agent prior to completion
of all scheduled study assessments.
- Concurrent disease or condition that would interfere with study participation or
safety, including, but not limited to, symptomatic cervical spine instability.
- Any condition that, in the view of the Principal Investigator (PI), places the subject
at high risk of poor treatment compliance or of not completing the study.
Age minimum:
5 Years
Age maximum:
18 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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MPS IV A
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Intervention(s)
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Drug: BMN 110
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Primary Outcome(s)
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Subject Incidence of Treatment Emergent AEs
[Time Frame: Entire Study, through week 84]
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Secondary Outcome(s)
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Change From Baseline in 6MWT
[Time Frame: Baseline to Weeks 12, 24, 36, 48, 72]
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Percent Change From Baseline in MVV
[Time Frame: Baseline to Weeks 12, 24, 36, 72]
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Change From Baseline in 3MSCT
[Time Frame: Baseline to Weeks 12, 24, 36, 48, 72]
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Percent Change From Baseline in FVC
[Time Frame: Baseline to Weeks 12, 24, 36, 72]
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Percent Change From Baseline in uKS
[Time Frame: Baseline to Weeks 12, 24, 36, 72]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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