A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC

Summary

This Phase I/II trial is to prove the efficacy and safety of AAV2-hAADC to treat patients with AADC deficiency.

Timeline

Start

2014-10-22

Primary completion

2020-12-18

Completion

2022-03-07

Outcome

Met primary endpoint

paper All patients met the primary efficacy endpoint: PMID 30169182 ↗

paper PDMS-2 scores were increased by a median of 62 points (IQR 39-93; p=0·005) PMID 30169182 ↗

paper HVA concentrations by a median of 25 nmol/L (IQR 11-48; p=0·012) PMID 30169182 ↗

Publications

• Tai CH, Lee NC, Chien YH, Byrne BJ, Muramatsu SI, Tseng SH, Hwu WL. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022 Feb 2;30(2):509-518. doi: 10.1016/j.ymthe.2021.11.005. Epub 2021 Nov 8.
• Chien YH, Lee NC, Tseng SH, Tai CH, Muramatsu SI, Byrne BJ, Hwu WL. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial. Lancet Child Adolesc Health. 2017 Dec;1(4):265-273. doi: 10.1016/S2352-4642(17)30125-6. Epub 2017 Oct 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eladocagene Exuparvovec	Gene therapy (AAV / viral vector)	1.81e+11 vg	Other

Indications