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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04543357
Recruitment Status : Recruiting
First Posted : September 10, 2020
Last Update Posted : June 2, 2023
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Description
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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
Household Contacts Other: Blood Draw

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
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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial
Actual Study Start Date : August 17, 2022
Estimated Primary Completion Date : September 11, 2023
Estimated Study Completion Date : September 11, 2023
Groups and Cohorts
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Group/Cohort 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
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Primary Outcome Measures :
  1. To quantify the proportion of participants (previously seronegative for NAb to AAV9) who develop NAb to AAV9 (ie, seroconversion). [ Time Frame: Day 28 after the interventional study patient is dosed ]
    Development of NAb to AAV9 (ie, NAb seroconversion).

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


Secondary Outcome Measures :
  1. To quantify the proportion of participants (previously seronegative for ADA to AAV9) who develop ADA to AAV9. [ Time Frame: Day 28 after the interventional study patient is dosed ]
    Development of ADA to AAV9 (ie, ADA seroconversion).

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


Biospecimen Retention:   Samples Without DNA
Blood

Eligibility Criteria
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Ages Eligible for Study:   0 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04543357


Contacts
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Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
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United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Additional Information:

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT04543357    
Other Study ID Numbers: C3391007
ARISEN ( Other Identifier: Alias Study Number )
First Posted: September 10, 2020    Key Record Dates
Last Update Posted: June 2, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Pfizer:
seroconversion
viral shedding
DNA vector shedding