Alpha-1 Antitrypsin DeficiencyNCT03804021

Long-Term Follow-up Study of ADVM-043

An observational study of Alpha-1 Antitrypsin Deficiency, run by Adverum Biotechnologies, Inc. at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 (NCT02168686) multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Alpha-1 Antitrypsin Deficiency
How many people: 6 participants
Where: 2 sitesUnited States
Study dates: 27 Dec 2018to 6 Dec 2021
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Study population: Patients who received ADVM-043 in a treatment protocol

Who may be able to join

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

Who cannot join

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)

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Medical University of South Carolina
Charleston, South Carolina, 29425
University of Florida
Gainesville, Florida, 32610

Other trials in United States

About this study

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

Read the 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 (ADVM-043-01; NCT02168686) were enrolled into this LTFU study for continued safety monitoring for a 2-year period.

Conditions: Alpha-1 Antitrypsin Deficiency
Being tested: No Intervention
Keywords: 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
Medical subject headings: alpha 1-Antitrypsin Deficiency, Lung Diseases, Emphysema, Pulmonary Disease, Chronic Obstructive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

No InterventionGenetic
Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

Study groups in NCT03804021
Study group	Role	What they receive
Roll over		Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686) Receives: Genetic: No Intervention

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
All adverse events of special interest and all serious adverse events related to ADVM-043
Measured: 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

27 Dec 20186 Dec 2021

First posted15 Jan 2019
Study start27 Dec 2018
Primary completion6 Dec 2021
Study completion6 Dec 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Alpha-1 Antitrypsin DeficiencyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Other
Time perspective: Prospective
Enrolment: 6 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03804021
Sponsor's protocol ID: ADVM-043-03
Status last verified: Sep 2023
First submitted: 11 Jan 2019
Last updated: 20 Sep 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.
PubMed 34256305

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Enrolling by invitationObservational study
AATD-Liver Scan Study
Alpha-1 Antitrypsin Deficiency, AATD
RecruitingPhase 4
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
Alpha 1-Antitrypsin, COPD
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 3
A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)
Alpha 1 Antitrypsin Deficiency
RecruitingPhase 1
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Alpha 1 Antitrypsin Deficiency

All Alpha-1 Antitrypsin Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency
ClinicalTrials.gov ID: NCT03804021
Acronym / sponsor ID: ADVM-043-03
Conditions: Alpha-1 Antitrypsin Deficiency
Keywords: Alpha-1 Antitrypsin Deficiency, alpha1-antitrypsin deficiency, alpha 1-antitrypsin deficiency, alpha1AT, A1AT, ADVM-043, AAVrh.10halpha1AT, AAVrh.10hA1AT
Study type: Observational
Recruitment status: Completed
Enrolment: 6 participants (actual)
Lead sponsor: Adverum Biotechnologies, Inc.Industry (pharma, biotech, devices)
Study start: 27 Dec 2018 (actual)
Primary completion: 6 Dec 2021 (actual)
Study completion: 6 Dec 2021 (actual)
First posted: 15 Jan 2019 (actual)
Results: Posted 20 Sep 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Observational model: Other
Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
Roll over	–	Genetic: No InterventionSubjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult
Study population: Patients who received ADVM-043 in a treatment protocol

Key inclusion criteria (1 listed)

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

Key exclusion criteria (3 listed)

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)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
No InterventionSubjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)	Genetic	Roll over

Primary outcome

Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
Time frame: 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686)
All adverse events of special interest and all serious adverse events related to ADVM-043

Results and safety

Results posted to the registry on 20 Sep 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Study ADVM-043-01 Part A Dose Level 1	2	2
Study ADVM-043-01 Part A Dose Level 2	2	2
Study ADVM-043-01 Part A Dose Level 3	2	2

Primary outcome: Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043

Time frame: 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686). Number. Unit: participants

Measure	Study ADVM-043-01 Part A Dose Level 1 n = 2	Study ADVM-043-01 Part A Dose Level 2 n = 2	Study ADVM-043-01 Part A Dose Level 3 n = 2
Deaths	0	0	0
Serious Adverse Events	0	0	0
Other Adverse Events	0	0	0

Population analysed: Subjects who received ADVM-043 in Study ADVM-043-01

Adverse events

Time frame: 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Study ADVM-043-01 Part A Dose Level 1	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Study ADVM-043-01 Part A Dose Level 2	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Study ADVM-043-01 Part A Dose Level 3	0 of 2 (0%)	0 of 2 (0%)	1 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 34256305Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.

Related trials for Alpha-1 Antitrypsin Deficiency

Enrolling by invitationObservational study
AATD-Liver Scan Study
Alpha-1 Antitrypsin Deficiency, AATD
RecruitingPhase 4
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
Alpha 1-Antitrypsin, COPD
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 3
A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)
Alpha 1 Antitrypsin Deficiency
RecruitingPhase 1
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Alpha 1 Antitrypsin Deficiency

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Charlton Strange, MD principal investigator

Medical University of South Carolina, Charleston, SC, USA

Mark Brantly, MD principal investigator

University of Florida, Gainesville, FL, USA

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Alpha-1 Antitrypsin Deficiency, run by Adverum Biotechnologies, Inc. at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Sep 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 (time frame: 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03804021, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Sep 2024.