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Long-Term Follow-up Study of ADVM-043

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03804021
Recruitment Status : Completed
First Posted : January 15, 2019
Last Update Posted : March 31, 2022
Sponsor:
Information provided by (Responsible Party):
Adverum Biotechnologies, Inc.

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment
Alpha-1 Antitrypsin Deficiency Genetic: ADVM-043

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 Design
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Study Type : Observational
Actual Enrollment : 6 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency
Actual Study Start Date : December 27, 2018
Actual Primary Completion Date : December 6, 2021
Actual Study Completion Date : December 6, 2021

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
1
Patients who received ADVM-043 in a treatment protocol
 Genetic: ADVM-043
Long term follow-up of subjects that received ADVM-043


Outcome Measures
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Primary Outcome Measures :
  1. 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


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients who received ADVM-043 in a treatment protocol
Criteria

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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03804021


Locations
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United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Sponsors and Collaborators
Adverum Biotechnologies, Inc.
Investigators
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Principal Investigator: Charlton Strange, MD Medical University of South Carolina, Charleston, SC, USA
Principal Investigator: Mark Brantly, MD University of Florida, Gainesville, FL, USA
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Adverum Biotechnologies, Inc.
ClinicalTrials.gov Identifier: NCT03804021    
Other Study ID Numbers: ADVM-043-03
First Posted: January 15, 2019    Key Record Dates
Last Update Posted: March 31, 2022
Last Verified: March 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Adverum Biotechnologies, Inc.:
Alpha-1 Antitrypsin Deficiency
alpha1-antitrypsin deficiency
alpha 1-antitrypsin deficiency
alpha1AT
A1AT
ADVM-043
AAVrh.10halpha1AT
AAVrh.10hA1AT
 Gene transfer vector
Gene therapy
Lung disease
Emphysema
COPD
ADVANCE study
Long Term Follow Up
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes