Long-Term Follow-up Study of ADVM-043



Status:Enrolling by invitation
Healthy:No
Age Range:18 - Any
Updated:1/17/2019
Start Date:January 11, 2019
End Date:December 31, 2021

Use our guide to learn which trials are right for you!

Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency

ADVM-043-03 is a long-term follow-up study of subjects in the prior ADVM 043-01 gene therapy
clinical study (ADVANCE) for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.

ADVM-043-03 is a long-term follow-up safety study of subjects in the ADVM 043 01 gene therapy
clinical study (ADVANCE) delivering ADVM-043 for treatment of Alpha-1 Antitrypsin (A1AT)
deficiency. Subjects will roll over after 12 months in the ADVANCE study into this study for
continued safety monitoring. ADVM-043-03 is a multi-site observational study, without
administration of investigational product, evaluating the long-term safety of ADVM-043
administered in a prior study. Subjects who received ADVM-043 will be followed for up to 3
years post-treatment

Key Inclusion Criteria:

- The subject has A1AT deficiency and has previously received ADVM-043 gene therapy

Key Exclusion Criteria:

- The subject is unwilling or unable to participate in all required study evaluations in
the long-term follow-up protocol

- The subject is participating in another investigational treatment protocol

- The subject is unable to understand the purpose and risks of the study or cannot
provide a signed and dated informed consent form (ICF)
We found this trial at
2
sites
171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
?
mi
from
Charleston, SC
Click here to add this to my saved trials
Gainesville, Florida 32610
(352) 392-3261
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
?
mi
from
Gainesville, FL
Click here to add this to my saved trials