Status:
COMPLETED
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Lead Sponsor:
Patricia I. Dickson, M.D.
Collaborating Sponsors:
The Ryan Foundation
BioMarin Pharmaceutical
Conditions:
Cognitive Decline
Mucopolysaccharidosis I
Eligibility:
All Genders
6+ years
Phase:
NA
Brief Summary
This is a 24-month study of the use of laronidase administered into the spinal fluid to treat cognitive decline in mucopolysaccharidosis I (MPS I). MPS I is a rare genetic condition due to deficiency ...
Detailed Description
This study is a 24-month open label, prospective, randomized trial in 16 MPS I patients age six years or older who have documented evidence of cognitive decline. The study will test the safety and eff...
Eligibility Criteria
Inclusion
- The presence of MPS I disease as documented by low α-L-iduronidase activity
- Age six years or older.
- The presence of acquired cognitive deficits as demonstrated by:
- 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
- Documented historical evidence of a decline of greater than one standard deviation on sequential testing, OR
- 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
- 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.
Key Trial Info
Start Date :
June 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2015
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00852358
Start Date
June 1 2009
End Date
April 1 2015
Last Update
January 26 2016
Active Locations (3)
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1
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609-1809
2
Los Angeles Biomedical Institute at Harbor-UCLA
Torrance, California, United States, 90502
3
University of Minnesota
Minneapolis, Minnesota, United States, 55455