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

Household Contacts Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04543357
• Other study ID #: C3391007
• Secondary ID: ARISEN
• Status: Recruiting
• Start date: August 17, 2022
• Est. completion date: December 24, 2025

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: December 24, 2025
• Est. primary completion date: December 24, 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 0 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Household Contacts

Intervention

Other:
• Blood Draw
Blood Samples for NAb and ADA to AAV9

Locations

Country	Name	City	State
United States	Kennedy Krieger Institute	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To quantify the proportion of participants (previously seronegative for NAb to AAV9) who develop NAb to AAV9 (ie, seroconversion).	Development of NAb to AAV9 (ie, NAb seroconversion).	Day 28 after the interventional study patient is dosed
Primary	To quantify the proportion of participants (previously seronegative for NAb to AAV9) who develop NAb to AAV9 (ie, seroconversion).	Development of NAb to AAV9 (ie, NAb seroconversion).	Day 56 after the interventional study patient is dosed
Secondary	To quantify the proportion of participants (previously seronegative for ADA to AAV9) who develop ADA to AAV9.	Development of ADA to AAV9 (ie, ADA seroconversion).	Day 28 after the interventional study patient is dosed
Secondary	To quantify the proportion of participants (previously seronegative for ADA to AAV9) who develop ADA to AAV9.	Development of ADA to AAV9 (ie, ADA seroconversion).	Day 56 after the interventional study patient is dosed

External Links

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