A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
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 Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| All participants Household contacts of a DMD patient in an interventional study of fordadistrogene movaparvovec. |
Other: Blood Draw
Blood Samples for NAb and ADA to AAV9
|
Outcome Measures
Primary Outcome Measures
- To quantify the proportion of participants (previously seronegative for NAb to AAV9) who develop NAb to AAV9 (ie, seroconversion). [Day 28 after the interventional study patient is dosed]
Development of NAb to AAV9 (ie, NAb seroconversion).
- 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
- To quantify the proportion of participants (previously seronegative for ADA to AAV9) who develop ADA to AAV9. [Day 28 after the interventional study patient is dosed]
Development of ADA to AAV9 (ie, ADA seroconversion).
- 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).
Eligibility Criteria
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.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Kennedy Krieger Institute | Baltimore | Maryland | United States | 21205 |
Sponsors and Collaborators
- Pfizer
Investigators
- Study Director: Pfizer CT.gov Call Center, Pfizer
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- C3391007
- ARISEN