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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Roll over | Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Genetic | Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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Patients who received ADVM-043 in a treatment protocol
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| Name | Affiliation | Role |
|---|---|---|
| Charlton Strange, MD | Medical University of South Carolina, Charleston, SC, USA | Principal Investigator |
| Mark Brantly, MD | University of Florida, Gainesville, FL, USA | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | United States | ||
| Medical University of South Carolina |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34256305 | Derived | 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. |
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Completion of Study ADVM-043-01
This is a long-term follow-up study for subjects who previously participated in Study ADVM-043-01.
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| ID | Title | Description |
|---|---|---|
| FG000 | Study ADVM-043-01 Part A Dose Level 1 | Single IV infusion of ADVM-043 at 8E13 total vg |
| FG001 | Study ADVM-043-01 Part A Dose Level 2 | Single IV infusion of ADVM-043 at 4E14 total vg |
| FG002 | Study ADVM-043-01 Part A Dose Level 3 | Single IV infusion of ADVM-043 at 1.2E15 total vg |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
[Not Specified]
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| ID | Title | Description |
|---|---|---|
| BG000 | Study ADVM-043-01 Part A Dose Level 1 | Single IV infusion of ADVM-043 at 8E13 total vg |
| BG001 | Study ADVM-043-01 Part A Dose Level 2 | Single IV infusion of ADVM-043 at 4E14 total vg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | Subjects who received ADVM-043 in Study ADVM-043-01 | Posted | Number | participants | 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) |
|
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
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Study ADVM-043-01 Part A Dose Level 1 | Follow-up after single IV infusion of ADVM-043 8E13 total vg |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Development Officer | Adverum Biotechnology, Inc. | 650-649-1413 | sseyedkazemi@adverum.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 15, 2019 | Jul 20, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 2, 2022 | May 14, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| D008171 | Lung Diseases |
| D004646 | Emphysema |
| D029424 | Pulmonary Disease, Chronic Obstructive |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
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| Charleston |
| South Carolina |
| 29425 |
| United States |
| BG002 | Study ADVM-043-01 Part A Dose Level 3 | Single IV infusion of ADVM-043 at 1.2E15 total vg |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG002 | Study ADVM-043-01 Part A Dose Level 3 | Follow-up after single IV infusion of ADVM-043 1.2E15 total vg |
|
|
| 0 |
| 2 |
| 0 |
| 2 |
| 0 |
| 2 |
| EG001 | Study ADVM-043-01 Part A Dose Level 2 | Follow-up after single IV infusion of ADVM-043 4E14 total vg | 0 | 2 | 0 | 2 | 0 | 2 |
| EG002 | Study ADVM-043-01 Part A Dose Level 3 | Follow-up after single IV infusion of ADVM-043 1.2E15 total vg | 1 | 2 | 0 | 2 | 0 | 2 |
Disclosure restrictions on the PIs are that Sponsor can review communications by the PI prior to public release and can embargo communications regarding trial results for varying time periods from the time submitted to the sponsor for review. The Sponsor may require changes to the communication after review and may delay publication upon request by the Sponsor to allow the Sponsor to seek/obtain patent protection.
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D008173 | Lung Diseases, Obstructive |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |