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

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ClinicalTrials.gov Identifier: NCT04543357
Recruitment Status : Not yet recruiting
First Posted : September 10, 2020
Last Update Posted : August 19, 2021
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
This study will include male and female participants who live or work in the same household as a participant in the Phase 3 interventional study, C3391003. 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 participant in study C3391003 who is treated with PF-06939926 gene therapy.

Condition or disease Intervention/treatment
Household Contacts Other: Blood Draw

Detailed Description:

This single center study to include approximately 50 to 250 participants is designed to estimate the likelihood of NAb seroconversion to AAV9 because of exposure to shed viral vector material released by a DMD participant treated with PF-06939926 in study C3391003. Eligible participants will undergo a blood draw provided by a Home Health Care Vendor at three home visits.

To maintain the blinded nature of study C3391003, all C3391007 study participants and investigators as well as the sponsor will remain blinded to the Cohort assignment of the C3391003 study participant in the same household until C3391003 becomes unblinded. Additionally, the detailed individual results will be considered sensitive clinical data and will not be shared with the participants, investigators, or the Sponsor until study C3391003 becomes unblinded.

The total duration of participation in the study is up to 4 months, including up to 48 days for the screening/baseline period and up to 66 days after dosing of investigational product in the C3391003 participant of the same household.

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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Other
Time Perspective: Other
Official Title: A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial
Estimated Study Start Date : December 20, 2021
Estimated Primary Completion Date : October 8, 2022
Estimated Study Completion Date : October 8, 2022

Group/Cohort Intervention/treatment
All participants
Household contacts of a participant in study C3391003
Other: Blood Draw
Blood Samples for NAb and ADA to AAV9




Primary Outcome Measures :
  1. To quantify the proportion of participants (previously seronegative for NAb to AAV9) who develop NAb to AAV9 (ie, seroconversion) and have a household contact being treated with PF-06939926 and those treated with placebo in Study C3391003. [ Time Frame: Day 28 after the C3391003 participant 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) and have a household contact being treated with PF-06939926 and those treated with placebo in Study C3391003. [ Time Frame: Day 56 after the C3391003 participant 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 and have a household contact being treated with PF-06939926 and those treated with placebo in Study C3391003. [ Time Frame: Day 28 after the C3391003 participant 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 and have a household contact being treated with PF-06939926 and those treated with placebo in Study C3391003. [ Time Frame: Day 56 after the C3391003 participant is dosed ]
    Development of ADA to AAV9 (ie, ADA seroconversion).


Biospecimen Retention:   Samples Without DNA
Blood


Information from the National Library of Medicine

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Ages Eligible for Study:   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 participant in the Phase 3 interventional study, C3391003. Up to 5 participants from the same household may be enrolled.
Criteria

Inclusion Criteria:

  1. Males or females who weigh at least 9 kg
  2. Anticipated to be living or working in the same household (> or = 10 hours per week and expected to have direct contact with the participant) as a participant in the Phase 3 interventional study, C3391003, for at least 4 months from the time of the screening visit. The participant in C3391003 must have completed their Baseline Visit, but not yet have received IP as part of C3391003
  3. The C3391003 participant is subsequently dosed with study intervention after the Baseline Visit

Exclusion Criteria:

  1. Prior treatment with gene therapy utilizing AAV vectors of any serotype.
  2. Living or working in a household where 5 other participants are already participating in this study (C3391007).

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

Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
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: August 19, 2021
Last Verified: August 2021
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: No
Studies a U.S. FDA-regulated Device Product: No