- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03804021
Long-Term Follow-up Study of ADVM-043
March 16, 2022 updated by: Adverum Biotechnologies, Inc.
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 (LTFU) study of subjects who participated in the ADVM-043-01 multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
ADVM-043-03 was a multi-center, observational study without administration of the ADVM-043 investigational product.
Subjects who participated in the ADVANCE study were enrolled into this LTFU study for continued safety monitoring for a 2-year period.
Study Type
Observational
Enrollment (Actual)
6
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Florida
-
Gainesville, Florida, United States, 32610
- University of Florida
-
-
South Carolina
-
Charleston, South Carolina, United States, 29425
- Medical University of South Carolina
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Patients who received ADVM-043 in a treatment protocol
Description
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)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Other
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
1
Patients who received ADVM-043 in a treatment protocol
|
Long term follow-up of subjects that received ADVM-043
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043
Time Frame: 2 years
|
Safety was assessed by collection of severity and relatedness of Adverse Events to ADVM-043, Adverse Events of Interest and Serious Adverse Events related to ADVM-043
|
2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Charlton Strange, MD, Medical University of South Carolina, Charleston, SC, USA
- Principal Investigator: Mark Brantly, MD, University of Florida, Gainesville, FL, USA
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 27, 2018
Primary Completion (Actual)
December 6, 2021
Study Completion (Actual)
December 6, 2021
Study Registration Dates
First Submitted
January 11, 2019
First Submitted That Met QC Criteria
January 11, 2019
First Posted (Actual)
January 15, 2019
Study Record Updates
Last Update Posted (Actual)
March 31, 2022
Last Update Submitted That Met QC Criteria
March 16, 2022
Last Verified
March 1, 2022
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ADVM-043-03
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Alpha-1 Antitrypsin Deficiency
-
Thomayer University HospitalMasaryk UniversityRecruiting
-
University of FloridaAlpha-1 FoundationEnrolling by invitation
-
Grifols Therapeutics LLCCompletedAlpha₁-Antitrypsin DeficiencyUnited States
-
Michael Campos, MDCSL BehringCompletedAlpha 1 Antitrypsin DeficiencyUnited States
-
Washington University School of MedicineNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); N... and other collaboratorsTerminatedLiver Cirrhosis | Alpha-1-antitrypsin DeficiencyUnited States
-
Alnylam PharmaceuticalsTerminatedZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseUnited Kingdom
-
University of PittsburghNational Heart, Lung, and Blood Institute (NHLBI)CompletedAlpha 1 Antitrypsin Deficiency | AATDUnited States
-
Hospices Civils de LyonCompletedChildren With a Deficiency of Alpha-1 AntitrypsinFrance
-
Heidelberg UniversityTerminatedHereditary Emphysema (Alpha 1-antitrypsin Deficiency)Germany
-
Grifols Therapeutics LLCCompletedEmphysema | Alpha 1-antitrypsin Deficiency (AATD)United States
Clinical Trials on ADVM-043
-
Adverum Biotechnologies, Inc.CompletedAlpha 1-Antitrypsin DeficiencyUnited States
-
Michael HoelscherLeibniz Institute for Natural Product Research and Infection Biology Hans...CompletedLung Diseases | Mycobacterium Infections | Tuberculosis | Tuberculosis, Pulmonary | Bacterial Infections RespiratoryNetherlands
-
Adverum Biotechnologies, Inc.ParexelActive, not recruitingNeovascular Age-related Macular DegenerationUnited States, France, United Kingdom
-
Michael HoelscherGerman Federal Ministry of Education and Research; German Center for Infection... and other collaboratorsCompletedLung Diseases | Bacterial Infections | Mycobacterium Infections | Tuberculosis | Tuberculosis, PulmonaryGermany
-
Adverum Biotechnologies, Inc.CompletedDiabetic Retinopathy | Diabetic Macular EdemaUnited States, Puerto Rico
-
GreenLight Biosciences, Inc.Withdrawn
-
GlaxoSmithKlineClick-TB ConsortiumRecruiting
-
Adverum Biotechnologies, Inc.CompletedNeovascular Age-related Macular Degeneration | Wet Age-related Macular DegenerationUnited States
-
Adverum Biotechnologies, Inc.Active, not recruitingNeovascular Age-related Macular Degeneration | Wet Age-related Macular DegenerationUnited States
-
Adverum Biotechnologies, Inc.Enrolling by invitationDiabetic Retinopathy | Diabetic Macular EdemaUnited States, Puerto Rico