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AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD

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: NCT05683379
Recruitment Status : Recruiting
First Posted : January 13, 2023
Last Update Posted : January 13, 2023
Sponsor:
Information provided by (Responsible Party):
REGENXBIO Inc.

Study Description
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Brief Summary:
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Diagnostic Test: AAV8 DetectCDx

Detailed Description:

This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD). Information collected in this study may be used to identify potential participants for DMD investigational gene therapy clinical trials.

This study consists of:

  • A phone/video interview to provide e-consent and medical history
  • A single home health visit to collect blood sample for antibody testing
  • A phone/video call for communication of AAV8 antibody test results
Study Design
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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Anti-AAV8 Antibody Assessment Study of Boys With Duchenne Muscular Dystrophy Aged 0 to <12 Years
Actual Study Start Date : December 20, 2022
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • Diagnostic Test: AAV8 DetectCDx
    An in vitro diagnostic assay to detect antibodies to AAV8 in human serum specimens.

Outcome Measures
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Primary Outcome Measures :
  1. Prevalence of anti-AAV8 antibodies in patients with DMD [ Time Frame: 90 days ]
    • To evaluate the prevalence of AAV8 antibodies in patients with DMD
    • To identify participants who may be eligible for investigational gene therapy clinical trials in males with DMD


Secondary Outcome Measures :
  1. Prevalence of anti-AAV9 antibodies in patients with DMD [ Time Frame: 90 days ]
    • To evaluate the prevalence of AAV9 antibodies in patients with DMD


Eligibility Criteria
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Ages Eligible for Study:   0 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Primary care clinic, Musculoskeletal care, Neuromusculoskeletal care
Criteria

Inclusion Criteria:

  • Males at least 0 to <12 years of age
  • Diagnosis of DMD
  • Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements

Exclusion Criteria:

  • Prior participation in a gene therapy trial OR recipient of a gene therapy drug
  • Other inclusion/exclusion criteria apply
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): NCT05683379


Contacts
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Contact: Patient Advocacy 866-860-0117 Duchenne@regenxbio.com

Locations
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United States, Georgia
Rare Disease Research Recruiting
Atlanta, Georgia, United States, 30329
Contact: Mandy Fruscione, PharmD    678-883-6897    Mandy.Fruscione@RareDiseaseResearch.com   
Contact: Marcial Almaraz       Marcial.Almaraz@RareDiseaseResearch.com   
Principal Investigator: Han Phan, MD         
Sponsors and Collaborators
REGENXBIO Inc.
More Information
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Responsible Party: REGENXBIO Inc.
ClinicalTrials.gov Identifier: NCT05683379    
Other Study ID Numbers: RGX-202-0101
First Posted: January 13, 2023    Key Record Dates
Last Update Posted: January 13, 2023
Last Verified: January 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Keywords provided by REGENXBIO Inc.:
DMD
Duchenne Muscular Dystrophy
Duchenne
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked