A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion

Phase 2 Completed 12 enrolled National Taiwan University Hospital PTC Therapeutics · collab

NaSingle-groupOpen-labelTreatment

Summary

This clinical trial expansion is to offer patients, who are not enrolled into the Phase I/II trial, a chance of treatment, to provide the experience in this gene therapy, and to increase the dose slightly.

Timeline

Start: 2016-11-09
Primary completion: 2022-01-24
Completion: 2022-05-16

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eladocagene Exuparvovec	Gene therapy (AAV / viral vector)	1.81e+11 vg	Other
Subject	Eladocagene Exuparvovec	Gene therapy (AAV / viral vector)	2.37e+11 vg	Other

A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion

Summary

Timeline

Publications

Drugs

Indications