Trial Profile
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type I
Status:
Active, no longer recruiting
Phase of Trial:
Phase I/II
Latest Information Update: 08 Dec 2023
Price :
$35
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At a glance
- Drugs RGX 111 (Primary)
- Indications Mucopolysaccharidosis I
- Focus Adverse reactions; First in man
- Sponsors REGENXBIO
- 23 Aug 2023 According to a REGENXBIO media release, interim data from this trial will be presented at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023.
- 28 Feb 2023 According to a REGENXBIO media release, the company expects to share additional updates on program plans in the second half of 2023.
- 24 Feb 2023 Interim results presented in the REGENXBIO Media Release.