MedTrace Logo

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

NCT02168686 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-10-05

Study results available
· View outcomes & findings →

Summary

The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or intrapleural administration.

Conditions

Interventions

GENETIC

ADVM-043

Gene transfer vector administration

Sponsors & Collaborators

  • Adverum Biotechnologies, Inc.

    lead INDUSTRY

Principal Investigators

  • Charlton Strange, MD · Medical University of South Carolina, Charleston, SC, USA

  • Friedrich Kueppers, MD · Temple University Hospital, Philadelphia, PA, USA

  • Mark Brantly, MD · University of Florida, Gainesville, FL, USA

  • Kyle Hogarth, MD · University of Chicago Medical Center, Chicago, IL, USA

  • Igor Barjakatarevic, MD · Ronald Reagan UCLA Medical Center, Santa Monica, CA, USA

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-11-28
Primary Completion
2019-08-29
Completion
2019-08-29
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02168686 on ClinicalTrials.gov