Clinical trial NCT04543357: A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.
Official title: A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Household Contacts
Keywords: seroconversion
viral shedding
DNA vector shedding
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2020-09-10
Enrollment: 8
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This single center study will include approximately 50 to 250 participants who live or work in the same household as a DMD patient in one of the fordadistrogene movaparvovec interventional studies. Up to 5 participants from the same household may be enrolled.

Brief summary: This study will include male and female participants who live or work in the same household as a patient in one of the fordadistrogene movaparvovec interventional studies. Up to 5 participants from the same household may be enrolled. The objective is to estimate the likelihood of NAb seroconversion to AAV9 in household contacts of a patient in one of the interventional studies who is treated with fordadistrogene movaparvovec gene therapy.

Detailed description: This single center study will include approximately 50 to 250 participants and is designed to estimate the likelihood of NAb seroconversion to AAV9 because of exposure to shed viral vector material released by a DMD patient treated with fordadistrogene movaparvovec in an interventional study. Eligible participants will undergo a blood draw provided by a Home Health Care Vendor at three home visits.

The total duration of participation in this study is about 4 months, including up to 48 days for the screening/baseline period and about 56 days after the DMD patient of the same household is dosed with the investigational gene therapy in the interventional study.

To maintain the blind in the blinded interventional studies, all C3391007 study participants and investigators as well as the sponsor will remain blinded to the Cohort assignment of the interventional study patient until the interventional study becomes unblinded.

Eligibility criteria: Inclusion Criteria:

* Males or females who weigh at least 9 kg.
* Anticipated to be living or working in the same household as a patient in one of the fordadistrogene movaparvovec interventional studies for at least 4 months.
* Anticipated to have \> 10 hours of contact per week and expected to have direct contact with the interventional study patient.
* The interventional patient is dosed in the interventional study.

Exclusion Criteria:

* Prior treatment with gene therapy utilizing AAV vectors of any serotype.

Primary outcomes:
measure: 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
timeFrame: Day 28 after the interventional study participant was dosed
measure: Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
timeFrame: Day 56 after the interventional study participant was dosed
Secondary outcomes:
measure: 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
timeFrame: Day 28 after the interventional study participant was dosed
measure: 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
timeFrame: Day 56 after the interventional study participant was dosed
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: