Household ContactsNCT04543357
A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.
An observational study of Household Contacts, run by Pfizer at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 0 years and olderHealthy volunteers can take part
- Conditions the study is for
- Household Contacts
- What is being tested?
- Blood Draw
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Aug 2022to 27 Feb 2024
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Prior treatment with gene therapy utilizing AAV vectors of any serotype.
Trial locations and contacts
1 site in 1 country.
United States1 site
Kennedy Krieger Institute
Baltimore, Maryland, 21205
About this study
Official title: A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial
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.
Read the 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.
- Conditions
- Household Contacts
- Being tested
- Blood Draw
- Keywords
- seroconversion, viral shedding, DNA vector shedding
- Medical subject headings
- HIV Seropositivity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood DrawOther
Blood Samples for NAb and ADA to AAV9
| Study group | Role | What they receive |
|---|---|---|
| All participants | Household contacts of a DMD patient in an interventional study of fordadistrogene movaparvovec. Receives: Other: Blood Draw |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
Development of NAb to AAV9 was defined by an increase of >=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer >=1 and <4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer <1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer >=6.
Measured: Day 28 after the interventional study participant was dosed
Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
Development of NAb to AAV9 was defined by an increase of >=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer >=1 and <4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer <1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer >=6.
Measured: Day 56 after the interventional study participant was dosed
Secondary outcomes (2)
Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer <50) for ADA to AAV9 at Baseline.
Measured: Day 28 after the interventional study participant was dosed
Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer <50) for ADA to AAV9 at Baseline.
Measured: Day 56 after the interventional study participant was dosed
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2020
- Study start17 Aug 2022
- Primary completion27 Feb 2024
- Study completion27 Feb 2024
Why it stopped: Study was terminated due to lack of efficacy in the Phase 3 study C3391003, Pfizer has decided to terminate any future dosing of fordadistrogene movaparvovec.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Household ContactsStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04543357
- Sponsor's protocol ID
- C3391007
- Other IDs
- ARISEN
- Status last verified
- Feb 2025
- First submitted
- 7 Aug 2020
- Last updated
- 26 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial
- ClinicalTrials.gov ID
- NCT04543357
- Acronym / sponsor ID
- C3391007
- Conditions
- Household Contacts
- Keywords
- seroconversion, viral shedding, DNA vector shedding
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study was terminated due to lack of efficacy in the Phase 3 study C3391003, Pfizer has decided to terminate any future dosing of fordadistrogene movaparvovec.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 17 Aug 2022 (actual)
- Primary completion
- 27 Feb 2024 (actual)
- Study completion
- 27 Feb 2024 (actual)
- First posted
- 10 Sep 2020 (actual)
- Results
- Posted 26 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All participants | – | Other: Blood DrawHousehold contacts of a DMD patient in an interventional study of fordadistrogene movaparvovec. |
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- Prior treatment with gene therapy utilizing AAV vectors of any serotype.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blood DrawBlood Samples for NAb and ADA to AAV9 | Other | All participants |
Primary outcome
Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
Time frame: Day 28 after the interventional study participant was dosed
Development of NAb to AAV9 was defined by an increase of >=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer >=1 and <4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer <1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer >=6.
Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
Time frame: Day 56 after the interventional study participant was dosed
Development of NAb to AAV9 was defined by an increase of >=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer >=1 and <4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer <1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer >=6.
2 secondary outcomes
Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
Time frame: Day 28 after the interventional study participant was dosed
Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
Time frame: Day 56 after the interventional study participant was dosed
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Household Contacts, run by Pfizer at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed (time frame: Day 28 after the interventional study participant was dosed). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2025.