Trial Profile

A first-in-human single-patient, investigator-initiated clinical trial of RGX-181 for the treatment of ceroid lipofuscinosis type 2 (CLN2) disease

Status: Active, no longer recruiting

Phase of Trial: Phase I

Latest Information Update: 31 Aug 2023

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At a glance

Drugs RGX-181 (Primary)
Indications Neuronal ceroid lipofuscinosis
Focus Adverse reactions; First in man

Most Recent Events

30 Aug 2023 Initial interim data from a first-in-human single-patient, investigator-initiated trial of RGX-181 presented in the REGENXBIO Media Release
30 Aug 2023 According to a REGENXBIO media release, initial interim data from a first-in-human single-patient, investigator-initiated trial of RGX-181 presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem.
23 Aug 2023 Status changed from planning to recruiting, according to a REGENXBIO media release.

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