Alpha-1 Antitrypsin Deficiency
Conditions
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
Brief 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.
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.
Interventions
GENETICNo Intervention
Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)
Sponsors
Adverum Biotechnologies, Inc.
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion 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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events of Special Interest and of 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) | All adverse events of special interest and all serious adverse events related to ADVM-043 |
Countries
United States
Participant flow
Recruitment details
This is a long-term follow-up study for subjects who previously participated in Study ADVM-043-01.
Pre-assignment details
Completion of Study ADVM-043-01
Participants by arm
| Arm | Count |
|---|---|
| Study ADVM-043-01 Part A Dose Level 1 Single IV infusion of ADVM-043 at 8E13 total vg | 2 |
| Study ADVM-043-01 Part A Dose Level 2 Single IV infusion of ADVM-043 at 4E14 total vg | 2 |
| Study ADVM-043-01 Part A Dose Level 3 Single IV infusion of ADVM-043 at 1.2E15 total vg | 2 |
| Total | 6 |
Baseline characteristics
| Characteristic | Study ADVM-043-01 Part A Dose Level 1 | Total | Study ADVM-043-01 Part A Dose Level 3 | Study ADVM-043-01 Part A Dose Level 2 |
|---|---|---|---|---|
| Age, Continuous | 40.5 years | 51.7 years | 61.0 years | 53.5 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 2 Participants | 6 Participants | 2 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 2 Participants | 6 Participants | 2 Participants | 2 Participants |
| Region of Enrollment United States | 2 participants | 6 participants | 2 participants | 2 participants |
| Sex: Female, Male Female | 0 Participants | 3 Participants | 2 Participants | 1 Participants |
| Sex: Female, Male Male | 2 Participants | 3 Participants | 0 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 2 | 1 / 2 |
| other Total, other adverse events | 0 / 2 | 0 / 2 | 0 / 2 |
| serious Total, serious adverse events | 0 / 2 | 0 / 2 | 0 / 2 |
Outcome results
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
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)
Population: Subjects who received ADVM-043 in Study ADVM-043-01
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Study ADVM-043-01 Part A Dose Level 1 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Serious Adverse Events | 0 participants |
| Study ADVM-043-01 Part A Dose Level 1 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Deaths | 0 participants |
| Study ADVM-043-01 Part A Dose Level 1 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Other Adverse Events | 0 participants |
| Study ADVM-043-01 Part A Dose Level 2 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Serious Adverse Events | 0 participants |
| Study ADVM-043-01 Part A Dose Level 2 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Deaths | 0 participants |
| Study ADVM-043-01 Part A Dose Level 2 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Other Adverse Events | 0 participants |
| Study ADVM-043-01 Part A Dose Level 3 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Deaths | 0 participants |
| Study ADVM-043-01 Part A Dose Level 3 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Other Adverse Events | 0 participants |
| Study ADVM-043-01 Part A Dose Level 3 | Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 | Serious Adverse Events | 0 participants |