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

NCT02232477 | Terminated Results DMC

• 1 other identifier: MIRC-002 (100)
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• Hopkins Verbal Learning Test

  Mean intra-subject change in total recall score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit. The Hopkins Verbal Learning Test measures verbal memory. The subject is read a list of words and asked to memorize as many words as possible. The scale ranges from 0 to 36, with higher scores indicating a better outcome. Two relevant time points: Baseline and the subject's final visit. Because this study ended prematurely for due to the pandemic, the subject's final visit was at the 12-month time point for one subject, 24-months for one subject, 36-months for two subjects, and 48-months for two subjects. Calculation: The total recall score at Baseline was subtracted from the total recall score at the subject's final visit and mean change and standard deviation were reported.

  zero to 48 months

Study Arms (1)

Open-label treatment

EXPERIMENTAL

laronidase 1.74 mg IT q 3 months for five years

Drug: Intrathecal recombinant human alpha iduronidase

Interventions

Intrathecal recombinant human alpha iduronidase DRUG

Intrathecal recombinant human alpha iduronidase every 3 months

Also known as: Aldurazyme

Open-label treatment

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• agnes chen: lead
• University of Minnesota: collaborator
• UCSF Benioff Children's Hospital Oakland: collaborator
• University of California, Los Angeles: collaborator
• The Ryan Foundation: collaborator
• Rare Diseases Clinical Research Network: collaborator
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Torrance, California, 90509, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I; Cognitive Dysfunction

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Results Point of Contact

• Title: Dr. Agnes Chen
• Organization: Lundquist Institute

ahchen@lundquist.org

424-306-7635

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 3, 2014
• First Posted: September 5, 2014
• Study Start: August 1, 2014
• Primary Completion: February 1, 2020
• Study Completion: February 1, 2020
• Last Updated: October 31, 2023
• Results First Posted: October 31, 2023

Record last verified: 2023-10

Locations

US (1)