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

NCT04543357 | Terminated Results FDA Drug

• 2 other identifiers: C3391007ARISEN
• Study Type: observational
• Target: 8
• Locations: 1 country
• Sites: 1

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.

Conditions

Household Contacts

Keywords

seroconversion; viral shedding; DNA vector shedding

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  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

  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

  Day 56 after the interventional study participant was dosed

Study Arms (1)

All participants

Household contacts of a DMD patient in an interventional study of fordadistrogene movaparvovec.

Other: Blood Draw

Interventions

Blood Draw OTHER

Blood Samples for NAb and ADA to AAV9

All participants

Eligibility Criteria

• Age: 0 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Prior treatment with gene therapy utilizing AAV vectors of any serotype.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Related Links

• To obtain contact information for a study center near you, click here.

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood

MeSH Terms

Conditions

HIV Seropositivity

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

HIV Infections; Blood-Borne Infections; Communicable Diseases; Infections; Sexually Transmitted Diseases, Viral; Sexually Transmitted Diseases; Lentivirus Infections; Retroviridae Infections; RNA Virus Infections; Virus Diseases; Genital Diseases; Urogenital Diseases; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2020
• First Posted: September 10, 2020
• Study Start: August 17, 2022
• Primary Completion: February 27, 2024
• Study Completion: February 27, 2024
• Last Updated: February 26, 2025
• Results First Posted: February 26, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)