Trial Outcomes & Findings for A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts. (NCT NCT04543357)
NCT ID: NCT04543357
Last Updated: 2025-02-26
Results Overview
Development of NAb to AAV9 was defined by an increase of \>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \>=1 and \<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \>=6.
TERMINATED
8 participants
Day 56 after the interventional study participant was dosed
2025-02-26
Participant Flow
This was a low-interventional study to assess development of neutralizing Antibodies (NAbs) to adeno associated virus serotype 9 (AAV9) in household contacts of Duchenne Muscular Dystrophy (DMD) Patients Treated with fordadistrogene movaparvovec.
Participant milestones
| Measure |
Participants With Potential Fordadistrogene Movaparvovec Exposure
People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \[NCT04281485\] and C3391002 \[not listed on ClinicalTrials.gov\]) were enrolled.
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|---|---|
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Overall Study
STARTED
|
8
|
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Overall Study
COMPLETED
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8
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.
Baseline characteristics by cohort
| Measure |
Participants With Potential Fordadistrogene Movaparvovec Exposure
n=8 Participants
People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \[NCT04281485\] and C3391002 \[not listed on ClinicalTrials.gov\]) were enrolled.
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|---|---|
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Age, Continuous
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41.0 Years
STANDARD_DEVIATION 9.35 • n=99 Participants
|
|
Sex: Female, Male
Female
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6 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
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2 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
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1 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
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7 Participants
n=99 Participants
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PRIMARY outcome
Timeframe: Day 28 after the interventional study participant was dosedPopulation: "Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with a NAb titer undetectable (ie, titer \<1) or detectable, but negative test (ie, titer \>=1 and \<4) at Baseline were included in the analysis.
Development of NAb to AAV9 was defined by an increase of \>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \>=1 and \<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \>=6.
Outcome measures
| Measure |
Participants With Potential Fordadistrogene Movaparvovec Exposure
n=3 Participants
People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \[NCT04281485\] and C3391002 \[not listed on ClinicalTrials.gov\]) were enrolled.
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|---|---|
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Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
|
0 Participants
|
PRIMARY outcome
Timeframe: Day 56 after the interventional study participant was dosedPopulation: "Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with a NAb titer undetectable (ie, titer \<1) or detectable, but negative test (ie, titer \>=1 and \<4) at Baseline were included in the analysis.
Development of NAb to AAV9 was defined by an increase of \>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \>=1 and \<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \>=6.
Outcome measures
| Measure |
Participants With Potential Fordadistrogene Movaparvovec Exposure
n=3 Participants
People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \[NCT04281485\] and C3391002 \[not listed on ClinicalTrials.gov\]) were enrolled.
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|---|---|
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Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 28 after the interventional study participant was dosedPopulation: "Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with an ADA titer negative at Baseline were included in the analysis.
Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \<50) for ADA to AAV9 at Baseline.
Outcome measures
| Measure |
Participants With Potential Fordadistrogene Movaparvovec Exposure
n=7 Participants
People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \[NCT04281485\] and C3391002 \[not listed on ClinicalTrials.gov\]) were enrolled.
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|---|---|
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Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
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0 Participants
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SECONDARY outcome
Timeframe: Day 56 after the interventional study participant was dosedPopulation: "Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with an ADA titer negative at Baseline were included in the analysis.
Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \<50) for ADA to AAV9 at Baseline.
Outcome measures
| Measure |
Participants With Potential Fordadistrogene Movaparvovec Exposure
n=7 Participants
People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \[NCT04281485\] and C3391002 \[not listed on ClinicalTrials.gov\]) were enrolled.
|
|---|---|
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Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
|
0 Participants
|
Adverse Events
Participants With Potential Fordadistrogene Movaparvovec Exposure
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 Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER