Phase 1/2 Study of Intravenous or Intrapleural Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Alpha-1 Antitrypsin cDNA to Individuals With Alpha-1 Antitrypsin Deficiency
Phase of Trial: Phase I/II
Latest Information Update: 01 Aug 2018
At a glance
- Drugs ADVM 043 (Primary) ; ADVM 043 (Primary)
- Indications Alpha 1-antitrypsin deficiency
- Focus Adverse reactions; First in man
- Acronyms ADVANCE
- Sponsors Adverum Biotechnologies
- 01 Aug 2018 According to an Adverum Biotechnologies media release, based on a review of the preliminary safety data from patients in Cohort 2, the independent data monitoring co mmittee (DMC) recommended dose escalation to Cohort 3, and the first patient was dosed with a single administration of ADVM-043 in late July 2018.
- 01 Aug 2018 According to an Adverum Biotechnologies media release, the company expects to report preliminary data from patients in cohorts 1 through 3 in this trial by the end of this year.
- 03 May 2018 According to an Adverum Biotechnologies media release, In late April 2018, first patient had been dosed in Cohort 2.