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)

Withdrawn

• Conditions: acid alpha glucosidase deficiency; Pompe Disease; 10027424

• Registration Number: NL-OMON56183
• Lead Sponsor: Sanofi BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 3

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
years old,
parent(s) or legally authorized representative(s) (LAR) must be capable of
giving signed
informed consent as described in Appendix 1 (Section 10.1) which includes
compliance
with the requirements and restrictions listed in the ICF and in this protocol.

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
procedural
sedation, as judged by the Investigator.
E 04. Concurrent treatment with any experimental drug or experimental vaccine.
E 05. Participation in another clinical trial with any investigational drug
within 30 days or 5 half-lives, whichever is greater, prior to study start (Day
1).
E 06. Previous participation in any clinical trial involving gene therapy.

Study & Design

• Study Type: Interventional
• Study Design: Not specified