Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

NCT ID: NCT02232477

Last Updated: 2023-10-31

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: NA
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-08-31
• Study Completion Date: 2020-02-29

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.

Conditions

Mucopolysaccharidosis I; Cognitive Decline

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open-label treatment

laronidase 1.74 mg IT q 3 months for five years

Group Type: EXPERIMENTAL

Intrathecal recombinant human alpha iduronidase

Intervention Type: DRUG

Intrathecal recombinant human alpha iduronidase every 3 months

Interventions

Intrathecal recombinant human alpha iduronidase

Intrathecal recombinant human alpha iduronidase every 3 months

Intervention Type: DRUG

Other Intervention Names

Aldurazyme

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.

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Minnesota

OTHER

Sponsor Role: collaborator

UCSF Benioff Children's Hospital Oakland

OTHER

Sponsor Role: collaborator

University of California, Los Angeles

OTHER

Sponsor Role: collaborator

The Ryan Foundation

OTHER

Sponsor Role: collaborator

Rare Diseases Clinical Research Network

NETWORK

Sponsor Role: collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

agnes chen

OTHER

Sponsor Role: lead

Responsible Party

agnes chen

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Torrance, California, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

MIRC-002 (100)

Identifier Type: -

Identifier Source: org_study_id