NCT04543357

A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.

A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 8 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 0 Years
• Healthy volunteers: Accepted

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.

Conditions

• Household Contacts

Interventions

Type	Name	Description
OTHER	Blood Draw	Blood Samples for NAb and ADA to AAV9

Timeline

• Start date: 2022-08-17
• Primary completion: 2024-02-27
• Completion: 2024-02-27
• First posted: 2020-09-10
• Last updated: 2025-02-26
• Results posted: 2025-02-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study