Trial Outcomes & Findings for Long-Term Follow-up Study of ADVM-043 (NCT NCT03804021)
NCT ID: NCT03804021
Last Updated: 2024-09-20
Results Overview
All adverse events of special interest and all serious adverse events related to ADVM-043
Recruitment status
COMPLETED
Target enrollment
6 participants
Primary outcome timeframe
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)
Results posted on
2024-09-20
Participant Flow
This is a long-term follow-up study for subjects who previously participated in Study ADVM-043-01.
Completion of Study ADVM-043-01
Participant milestones
| Measure |
Study ADVM-043-01 Part A Dose Level 1
Single IV infusion of ADVM-043 at 8E13 total vg
|
Study ADVM-043-01 Part A Dose Level 2
Single IV infusion of ADVM-043 at 4E14 total vg
|
Study ADVM-043-01 Part A Dose Level 3
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
2
|
|
Overall Study
COMPLETED
|
2
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Long-Term Follow-up Study of ADVM-043
Baseline characteristics by cohort
| Measure |
Study ADVM-043-01 Part A Dose Level 1
n=2 Participants
Single IV infusion of ADVM-043 at 8E13 total vg
|
Study ADVM-043-01 Part A Dose Level 2
n=2 Participants
Single IV infusion of ADVM-043 at 4E14 total vg
|
Study ADVM-043-01 Part A Dose Level 3
n=2 Participants
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Continuous
|
40.5 years
n=99 Participants
|
53.5 years
n=107 Participants
|
61.0 years
n=206 Participants
|
51.7 years
n=7 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
6 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
6 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=99 Participants
|
2 participants
n=107 Participants
|
2 participants
n=206 Participants
|
6 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: 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
All adverse events of special interest and all serious adverse events related to ADVM-043
Outcome measures
| Measure |
Study ADVM-043-01 Part A Dose Level 1
n=2 Participants
Follow-up after single IV infusion of ADVM-043 8E13 total vg
|
Study ADVM-043-01 Part A Dose Level 2
n=2 Participants
Follow-up after single IV infusion of ADVM-043 4E14 total vg
|
Study ADVM-043-01 Part A Dose Level 3
n=2 Participants
Follow-up after single IV infusion of ADVM-043 1.2E15 total vg
|
|---|---|---|---|
|
Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
Deaths
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
Serious Adverse Events
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
Other Adverse Events
|
0 participants
|
0 participants
|
0 participants
|
Adverse Events
Study ADVM-043-01 Part A Dose Level 1
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Study ADVM-043-01 Part A Dose Level 2
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Study ADVM-043-01 Part A Dose Level 3
Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER