ID

45955

Description

Principal Investigator: Moin Vera, MD, PhD, Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center, Torrance, CA, USA MeSH: Mucopolysaccharidosis I,Lysosomal Storage Diseases https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000862 In this study we characterized the immune response to intrathecal (IT) recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis I (MPS I) subjects with spinal cord compression who had been previously treated with intravenous rhIDU. Concentrations of specific antibodies and cytokines were measured in serum and cerebrospinal fluid (CSF) collected before monthly IT rhIDU infusions. These serologic findings were compared with clinical adverse event (AE) reports to establish temporal correlations with clinical symptoms. We found that IT rhIDU was generally well tolerated in the subjects studied although one subject had moderate to severe clinical symptoms and serologic abnormalities consistent with an immune response.

Link

dbGaP study=phs000862

Keywords

Versions (1)
  1. 3/16/24 3/16/24 - Madita Rudolph
Copyright Holder

Moin Vera, MD, PhD, Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center, Torrance, CA, USA

Uploaded on

March 16, 2024

DOI

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License

Creative Commons BY 4.0
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dbGaP phs000862 Immune Response to IT ERT in Mucopolysaccharidosis I Patient

English

Eligibility Criteria

6 forms  
Inclusion and exclusion criteria
Description

Inclusion and exclusion criteria

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0680251
Samples were taken from participants in the following four studies:
Description

Elig.phs000862.v1.p1.1

Data type

boolean

Alias
UMLS CUI [1,1]
C0200345
MIRC-001 (NCT 00215527, P. Dickson, PI): This is a pilot, 4-month study of 1.75 mg IT laronidase (recombinant human alpha-L-iduronidase) given monthly via lumbar spinal tap to MPS I patients age 8 years and older with spinal cord compression.
Description

Elig.phs000862.v1.p1.2

Data type

boolean

Alias
UMLS CUI [1,1]
C0087111
UMLS CUI [1,2]
C1302054
UMLS CUI [1,3]
C0023786
UMLS CUI [1,4]
C0001779
UMLS CUI [1,5]
C0037926
MIRC-001 (100) (NCT 00786968, P. Dickson, PI): This is an extension study for MIRC-001. Subjects with good response to IT laronidase defined by improvement in any outcome measure and no safety concerns receive 12 months of additional therapy at 30 to 90 day intervals.
Description

Elig.phs000862.v1.p1.3

Data type

boolean

Alias
UMLS CUI [1,1]
C0205170
UMLS CUI [1,2]
C0521982
UMLS CUI [1,3]
C1302054
UMLS CUI [1,4]
C1706712
MIRC-002 (NCT 00852358, P. Dickson, PI): This is a 1-year randomized controlled study followed by 1-year open label phase of 1.75 mg IT laronidase given at 30 to 90 day intervals to MPS I patients age 6 years and older with cognitive impairment or decline.
Description

Elig.phs000862.v1.p1.4

Data type

boolean

Alias
UMLS CUI [1,1]
C0087111
UMLS CUI [1,2]
C1302054
UMLS CUI [1,3]
C0023786
UMLS CUI [1,4]
C0001779
UMLS CUI [1,5]
C0338656
MT2007 (NCT 00638547, P. Orchard, PI): This is a 1-year study of 0.05 mg/kg IT laronidase given at 90 day intervals to MPS I Hurler patients age 0.5-3 years in conjunction with weekly 0.58 mg/kg IV laronidase and hematopoietic stem cell transplantation.
Description

Elig.phs000862.v1.p1.5

Data type

boolean

Alias
UMLS CUI [1,1]
C0087111
UMLS CUI [1,2]
C1302054
UMLS CUI [1,3]
C0472699
UMLS CUI [1,4]
C0023786
UMLS CUI [1,5]
C0001779
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Similar models

Eligibility Criteria

6 forms
Name
Type
Description | Question | Decode (Coded Value)
Data type
Alias
Item Group
Inclusion and exclusion criteria
C1512693 (UMLS CUI [1,1])
C0680251 (UMLS CUI [1,2])
Elig.phs000862.v1.p1.1
Item
Samples were taken from participants in the following four studies:
boolean
C0200345 (UMLS CUI [1,1])
Elig.phs000862.v1.p1.2
Item
MIRC-001 (NCT 00215527, P. Dickson, PI): This is a pilot, 4-month study of 1.75 mg IT laronidase (recombinant human alpha-L-iduronidase) given monthly via lumbar spinal tap to MPS I patients age 8 years and older with spinal cord compression.
boolean
C0087111 (UMLS CUI [1,1])
C1302054 (UMLS CUI [1,2])
C0023786 (UMLS CUI [1,3])
C0001779 (UMLS CUI [1,4])
C0037926 (UMLS CUI [1,5])
Elig.phs000862.v1.p1.3
Item
MIRC-001 (100) (NCT 00786968, P. Dickson, PI): This is an extension study for MIRC-001. Subjects with good response to IT laronidase defined by improvement in any outcome measure and no safety concerns receive 12 months of additional therapy at 30 to 90 day intervals.
boolean
C0205170 (UMLS CUI [1,1])
C0521982 (UMLS CUI [1,2])
C1302054 (UMLS CUI [1,3])
C1706712 (UMLS CUI [1,4])
Elig.phs000862.v1.p1.4
Item
MIRC-002 (NCT 00852358, P. Dickson, PI): This is a 1-year randomized controlled study followed by 1-year open label phase of 1.75 mg IT laronidase given at 30 to 90 day intervals to MPS I patients age 6 years and older with cognitive impairment or decline.
boolean
C0087111 (UMLS CUI [1,1])
C1302054 (UMLS CUI [1,2])
C0023786 (UMLS CUI [1,3])
C0001779 (UMLS CUI [1,4])
C0338656 (UMLS CUI [1,5])
Elig.phs000862.v1.p1.5
Item
MT2007 (NCT 00638547, P. Orchard, PI): This is a 1-year study of 0.05 mg/kg IT laronidase given at 90 day intervals to MPS I Hurler patients age 0.5-3 years in conjunction with weekly 0.58 mg/kg IV laronidase and hematopoietic stem cell transplantation.
boolean
C0087111 (UMLS CUI [1,1])
C1302054 (UMLS CUI [1,2])
C0472699 (UMLS CUI [1,3])
C0023786 (UMLS CUI [1,4])
C0001779 (UMLS CUI [1,5])
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