Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Information source: Los Angeles Biomedical Research Institute
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Mucopolysaccharidosis I; Cognitive Decline
Intervention: Intrathecal recombinant human alpha iduronidase (Drug)
Phase: N/A
Status: Recruiting
Sponsored by: agnes chen Overall contact: Agnes Chen, MD, Phone: 310-222-4160, Email: ahchen@labiomed.org
Summary
This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for
Cognitive Decline in MPS I". Participants must have completed the pilot study to
participate in this study.
Clinical Details
Official title: An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Hopkins Verbal Learning Test
Eligibility
Minimum age: 6 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
1. The subject has completed the MIRC-002 study of intrathecal enzyme replacement
therapy for cognitive decline in mucopolysaccharidosis I
2. Age six years or older.
3. Subject and/or guardian willing and able to provide written informed consent.
4. Negative urine pregnancy test at screening (non-sterile females of child-bearing
potential only)
5. 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)
6. 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:
1. The subject has undergone hematopoietic stem cell transplantation
2. 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
3. Pregnant or lactating, or considering pregnancy
4. Receipt of an investigational drug or procedure other than intrathecal Aldurazyme®
within 30 days of enrollment
5. 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.
6. Infusion reactions to intravenous or intrathecal Aldurazyme® therapy that are
life-threatening or require emergent intervention such as epinephrine,
cardiopulmonary resuscitation, or hospitalization
7. The subject has severely impaired spinal CSF flow, demonstrated by failure of
appearance of radionuclide in the basal cisterns by 4 hours after intra-lumbar
administration.
8. The subject has a coagulopathy, as identified by a platelet count of less than
50,000, an INR of 1. 5 or greater, or a PTT that is 1. 5 times the upper limit of
normal for the laboratory from which it was drawn.
Locations and Contacts
Agnes Chen, MD, Phone: 310-222-4160, Email: ahchen@labiomed.org
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, California 90509, United States; Recruiting Agnes Chen, MD, Phone: 310-222-4160, Email: ahchen@labiomed.org
Additional Information
Starting date: May 2014
Last updated: March 30, 2015
|