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. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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1 February 2016 |
Main ID: |
NCT00852358 |
Date of registration:
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25/02/2009 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
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Scientific title:
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A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I |
Date of first enrolment:
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June 2009 |
Target sample size:
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9 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT00852358 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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N/A
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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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Agnes Chen, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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Los Angeles Biomedical Institute at Harbor-UCLA |
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Name:
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Patricia I Dickson, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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Los Angeles Biomedical Institute at Harbor-UCLA |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- The presence of MPS I disease as documented by low a-L-iduronidase activity
- Age six years or older.
- The presence of acquired cognitive deficits as demonstrated by:
1. A score of one standard deviation below mean on IQ testing or in one domain of
neuropsychological function (language, memory, or non-verbal ability), OR
2. Documented historical evidence of a decline of greater than one standard
deviation on sequential testing, OR
3. A score between 0.75 and 1 standard deviation below the mean, AND the cognitive
deficit affects daily performance.
- The decline in function is not explainable by other neurological or psychiatric
factors.
- Subject and/or guardian willing and able to provide written informed consent.
- Negative urine pregnancy test at screening (non-sterile females of child-bearing
potential only)
- Currently using two acceptable methods of birth control as determined by the
investigator and willing to continue to use acceptable birth control during their
participation in the study (non-sterile females of child-bearing potential who are
sexually active only)
- Willing and able to comply with study procedures. For example, the subjects must be
able to complete written and computer-based testing. The subjects must be able to lie
still in the MRI scanner for at least 40 minutes without sedation.
Exclusion Criteria:
- The subject has undergone hematopoietic stem cell transplantation
- Recent initiation of intravenous Aldurazyme® therapy with less than 6 months of
therapy. Subjects who have been receiving Aldurazyme® therapy for more than 6 months,
and those who have never received Aldurazyme® therapy, will be allowed to enroll
- Pregnant or lactating, or considering pregnancy
- Receipt of an investigational drug or procedure within 30 days of enrollment
- A condition, medical or other, that prevents participation in the study, including
severe auditory or visual impairment, significant lumbar pathology, lumbar catheter,
or recent major surgery within 6 weeks that would preclude their ability to
participate.
- Infusion reactions to intravenous Aldurazyme® therapy that require ongoing medical
intervention, special prophylaxis or altered rate or dose of enzyme administration
- The subject has a programmable VP shunt that is incompatible with the 3 Tesla MRI
magnet and is unable or unwilling to undergo shunt revision to a MRI compatible
device.
- The subject has another contraindication for MRI, such as nonremovable metal in the
body.
- The subject has severely impaired spinal CSF flow, demonstrated by failure of
appearance of 99mTechnetium-DTPA in the basal cisterns by 4 hours after intra-lumbar
administration.
Age minimum:
6 Years
Age maximum:
N/A
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Lysosomal Storage Disease
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Hurler-Scheie Syndrome
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Cognitive Decline
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Mucopolysaccharidosis I
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Scheie Syndrome
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Intervention(s)
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Drug: laronidase
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Primary Outcome(s)
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The objective of this study is to assess the ability of intrathecal a-L-iduronidase to be administered safely
[Time Frame: 24 months]
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Secondary Outcome(s)
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The objective of this study is to assess the ability of intrathecal a-L-iduronidase to stabilize or reverse cognitive decline.
[Time Frame: 24 months]
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Secondary ID(s)
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IND 104,354
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MIRC-002
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U54NS065768
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IRB Project No. 13270-01
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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