NCT05683379

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).

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P75+ for all trials

Timeline
4mo left

Started Dec 2022

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress91%
Dec 2022Sep 2026

Study Start

First participant enrolled

December 20, 2022

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

January 4, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 13, 2023

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2026

Last Updated

November 25, 2025

Status Verified

November 1, 2025

Enrollment Period

3.7 years

First QC Date

January 4, 2023

Last Update Submit

November 20, 2025

Conditions

Duchenne Muscular Dystrophy

Keywords

DMDDuchenne Muscular DystrophyDuchenne

Outcome Measures

Primary Outcomes (1)

  • Prevalence of anti-AAV8 antibodies in patients with DMD

    * 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

    90 days

Interventions

AAV8 DetectCDxDIAGNOSTIC_TEST

An in vitro diagnostic assay to detect antibodies to AAV8 in human serum specimens.

Eligibility Criteria

Age0 Years - 25 Years
Sexmale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

Primary care clinic, Musculoskeletal care, Neuromusculoskeletal care

You may qualify if:

  • Males at least 0 to \<25 years of age
  • Diagnosis of DMD
  • Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements

You may not qualify if:

  • Prior participation in a gene therapy trial OR recipient of a gene therapy drug

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Rare Disease Research

Atlanta, Georgia, 30329, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Patient Advocacy

CONTACT

(833) 711-0349Duchenne@regenxbio.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 4, 2023

First Posted

January 13, 2023

Study Start

December 20, 2022

Primary Completion (Estimated)

September 1, 2026

Study Completion (Estimated)

September 1, 2026

Last Updated

November 25, 2025

Record last verified: 2025-11

Locations

US(1)