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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. Identifier: NCT03804021
Recruitment Status : Enrolling by invitation
First Posted : January 15, 2019
Last Update Posted : October 4, 2019
Information provided by (Responsible Party):
Adverum Biotechnologies, Inc.

Brief Summary:
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.

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

Detailed Description:
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

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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 : January 11, 2019
Estimated Primary Completion Date : September 30, 2021
Estimated Study Completion Date : December 31, 2021

Group/Cohort Intervention/treatment
Subjects who received a dose of ADVM-043 in a prior clinical study
Genetic: ADVM-043
Long term follow- up of subjects that received ADVM-043

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 will be assessed by collection of Adverse Events of interest and Serious Adverse Events related to ADVM-043

Information from the National Library of Medicine

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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

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)

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 identifier (NCT number): NCT03804021

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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.
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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
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Adverum Biotechnologies, Inc. Identifier: NCT03804021    
Other Study ID Numbers: ADVM043-03
First Posted: January 15, 2019    Key Record Dates
Last Update Posted: October 4, 2019
Last Verified: October 2019

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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.:
alpha1-antitrypsin deficiency
alpha 1-antitrypsin deficiency
Gene transfer vector
Gene therapy
Lung disease
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
Pathologic Processes