Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)

Recruiting Phase 1 Phase 2 Interventional Study
Charcot-Marie-Tooth Disease Type 4J
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 – 20
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
8 (estimated)
Sponsor
Elpida Therapeutics SPC · Industry
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About This Trial

First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with CMT4J.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Male and females between the ages of 3 and 20 years at the time of screening. * Molecularly-confirmed diagnosis of CMT4J (confirmed by a CLIA certified, CE-marked, or equivalent lab) due to bi-allelic pathogenic variants (by ACMG criteria) in the FIG4 gene where one of the mut…
Contacts

Rachel Thomas, RN

1-833-335-7432

studyinfo@elpidatx.com

CONTACT