NL-OMON56183
Withdrawn
Not Applicable
A multi-center, non-randomized study to characterize biomarkers in cerebrospinal fluid (CSF) of patients with infantile-onset (IOPD) or late-onset Pompe disease (LOPD) - ASY17795
Sanofi BV0 sites3 target enrollmentTBD
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- acid alpha glucosidase deficiency
- Sponsor
- Sanofi BV
- Enrollment
- 3
- Status
- Withdrawn
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •I 01\. Participants who have a confirmed diagnosis of LOPD and clinical signs or
- •symptoms OR
- •participants who have a confirmed diagnosis of IOPD.
- •I 02\. Capable of giving signed informed consent as described in Appendix 1
- •(Section 10\.1\) of
- •the protocol which includes compliance with the requirements and restrictions
- •listed in the
- •informed consent form (ICF) and in this protocol; OR If participant is \<18
- •parent(s) or legally authorized representative(s) (LAR) must be capable of
- •giving signed
Exclusion Criteria
- •E 01\. Chronic illness, except for LOPD, IOPD, or directly resulting from Pompe
- •disease, that, at
- •the discretion of the Investigator, could have an impact on Hex4 and
- •inflammatory markers in the CSF.
- •E 02\. Any acute illness that, at the discretion of the Investigator, could have
- •an impact on Hex4
- •and inflammatory markers in the CSF.
- •E 03\. High risk of complications from lumbar puncture or potentially associated
- •sedation, as judged by the Investigator.
- •E 04\. Concurrent treatment with any experimental drug or experimental vaccine.
Outcomes
Primary Outcomes
Not specified
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