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

Terminated

• Conditions: Household Contacts
• Interventions: Other: Blood Draw

• Registration Number: NCT04543357
• Lead Sponsor: Pfizer

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.

Study Timeline

• Study First Submitted: 2020-08-07
• Study First Submitted QC: 2020-09-02
• Study First Posted: 2020-09-10
• Study Start: 2022-08-17
• Primary Completion: 2024-02-27
• Study Completion: 2024-02-27
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-02
• Last Update Posted: 2024-08-06

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 8

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.

Read More

Exclusion Criteria

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

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
All participants	Blood Draw	Household contacts of a DMD patient in an interventional study of fordadistrogene movaparvovec.

Primary Outcome Measures

Name	Time	Method
To quantify the proportion of participants (previously seronegative for NAb to AAV9) who develop NAb to AAV9 (ie, seroconversion).	Day 56 after the interventional study patient is dosed	Development of NAb to AAV9 (ie, NAb seroconversion).

Secondary Outcome Measures

Name	Time	Method
To quantify the proportion of participants (previously seronegative for ADA to AAV9) who develop ADA to AAV9.	Day 56 after the interventional study patient is dosed	Development of ADA to AAV9 (ie, ADA seroconversion).

Trial Locations

Locations (1)

Kennedy Krieger Institute; 🇺🇸 Baltimore, Maryland, United States