Clinical trial NCT02232477: Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Official title: An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Mucopolysaccharidosis I
Cognitive Decline
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Sponsor class: OTHER
Collaborators: name: University of Minnesota
name: UCSF Benioff Children's Hospital Oakland
name: University of California, Los Angeles
name: The Ryan Foundation
name: Rare Diseases Clinical Research Network
name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2014-09-05
Enrollment: 6
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility 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.

Primary outcomes:
measure: Hopkins Verbal Learning Test
timeFrame: zero to 48 months
Secondary outcomes:
Other outcomes:
Locations:
city: Torrance
country: United States
facility: Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
state: California
Study officials:
References: