Phase I, Multicentre, Open Label, Single Dose, Dose-ranging Clinical Trial to Investigate the Safety and Tolerability of a Gene Therapy rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
Phase of Trial: Phase I
Latest Information Update: 31 May 2016
At a glance
- Drugs AMT 021 (Primary)
- Indications Acute intermittent porphyria
- Focus Adverse reactions
- Sponsors Digna Biotech
- 10 Jun 2017 Biomarkers information updated
- 31 May 2016 According to an uniQure media release, results from this trial were published in the Journal of Hepatology.
- 06 Apr 2015 Top-line, 1-year follow-up data has been reported, according to a uniQure media release.