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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02232477
Recruitment Status : Terminated (Termination of study by investigator related to COVID pandemic.)
First Posted : September 5, 2014
Last Update Posted : October 8, 2020
Sponsor:
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)
Information provided by (Responsible Party):
agnes chen, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis I Cognitive Decline Drug: Intrathecal recombinant human alpha iduronidase Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
Study Start Date : May 2014
Actual Primary Completion Date : October 1, 2020
Actual Study Completion Date : October 1, 2020


Arm Intervention/treatment
Experimental: Open-label treatment
laronidase 1.74 mg IT q 3 months for five years
Drug: Intrathecal recombinant human alpha iduronidase
Intrathecal recombinant human alpha iduronidase every 3 months
Other Name: Aldurazyme




Primary Outcome Measures :
  1. Hopkins Verbal Learning Test [ Time Frame: 5 years ]
    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



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02232477


Locations
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United States, California
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States, 90509
Sponsors and Collaborators
agnes chen
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)
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Responsible Party: agnes chen, Principal Investigator, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
ClinicalTrials.gov Identifier: NCT02232477    
Other Study ID Numbers: MIRC-002 (100)
First Posted: September 5, 2014    Key Record Dates
Last Update Posted: October 8, 2020
Last Verified: October 2020
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis I
Cognitive Dysfunction
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases