Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Primary Purpose
Mucopolysaccharidosis I, Cognitive Decline
Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Intrathecal recombinant human alpha iduronidase
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I
Eligibility Criteria
Inclusion Criteria:
- The subject has completed the MIRC-002 study of intrathecal enzyme replacement therapy for cognitive decline in mucopolysaccharidosis I
- Age six years or older.
- 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 other than intrathecal Aldurazyme® 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 or intrathecal Aldurazyme® therapy that are life-threatening or require emergent intervention such as epinephrine, cardiopulmonary resuscitation, or hospitalization
- 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.
- 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.
Sites / Locations
- Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open-label treatment
Arm Description
laronidase 1.74 mg IT q 3 months for five years
Outcomes
Primary Outcome Measures
Hopkins Verbal Learning Test
Mean intra-subject change in memory score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit
Secondary Outcome Measures
Full Information
NCT ID
NCT02232477
First Posted
September 3, 2014
Last Updated
October 5, 2020
Sponsor
agnes chen
Collaborators
University of Minnesota, UCSF Benioff Children's Hospital Oakland, University of California, Los Angeles, The Ryan Foundation, Rare Diseases Clinical Research Network, National Institute of Neurological Disorders and Stroke (NINDS)
1. Study Identification
Unique Protocol Identification Number
NCT02232477
Brief Title
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
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Terminated
Why Stopped
Termination of study by investigator related to COVID pandemic.
Study Start Date
May 2014 (undefined)
Primary Completion Date
October 1, 2020 (Actual)
Study Completion Date
October 1, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
agnes chen
Collaborators
University of Minnesota, UCSF Benioff Children's Hospital Oakland, University of California, Los Angeles, The Ryan Foundation, Rare Diseases Clinical Research Network, National Institute of Neurological Disorders and Stroke (NINDS)
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I, Cognitive Decline
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Open-label treatment
Arm Type
Experimental
Arm Description
laronidase 1.74 mg IT q 3 months for five years
Intervention Type
Drug
Intervention Name(s)
Intrathecal recombinant human alpha iduronidase
Other Intervention Name(s)
Aldurazyme
Intervention Description
Intrathecal recombinant human alpha iduronidase every 3 months
Primary Outcome Measure Information:
Title
Hopkins Verbal Learning Test
Description
Mean intra-subject change in memory score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit
Time Frame
5 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
The subject has completed the MIRC-002 study of intrathecal enzyme replacement therapy for cognitive decline in mucopolysaccharidosis I
Age six years or older.
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 other than intrathecal Aldurazyme® 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 or intrathecal Aldurazyme® therapy that are life-threatening or require emergent intervention such as epinephrine, cardiopulmonary resuscitation, or hospitalization
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.
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.
Facility Information:
Facility Name
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
City
Torrance
State/Province
California
ZIP/Postal Code
90509
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
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