Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin. (SeroDysMyo)

June 3, 2026 updated by: Genethon
This study aims to assess the overall seroprevalence of neutralizing antibodies against different AAVs potentially used in gene therapy in patients with genetic neuromuscular diseases.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

This research aims to determine the seroprevalence and immunological profiles of recent and/or emerging AAV capsids that could be used in future gene therapy programs. It will thus make it possible to: 1/ improve knowledge of the prevalence of anti-AAV antibodies (total and neutralizing) in these specific populations; 2/ identify the clinical, demographic or biological factors likely to influence this seroprevalence (age, sex, type of pathology, immunosuppressive treatment followed); 3/ to characterize the phenomena of cross-reactivity between different serotypes or variants of capsids, a major challenge for the design of new generations of vectors.

The data generated will contribute to a better estimation of the populations potentially eligible for gene therapies, by making it possible to anticipate exclusion rates related to pre-existing immunity. They may also guide the rational selection of alternative capsids that are less immunogenic or evade cross-immunity, as well as the development of immunomodulation or re-assay strategies.

Study Type

Interventional

Enrollment (Estimated)

450

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
    • Île-de-France Region
      • Évry, Île-de-France Region, France, 9100

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease
  2. Informed patient who signed informed consent
  3. No opposition from the holders of parental authority or guardian, for minor patients.
  4. Affiliated/beneficiary of a national health insurance scheme

Exclusion Criteria:

  • Gene or cell therapy treatment prior to blood collection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Screening
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis
Diagnostic test: Dosage of AAV neutralizing antibodies

In each clinical site, the principal investigator will propose to his patients with neuromuscular disease of genetic origin to take a blood sample (4 mL of blood) to determine their level of immunity to certain AAVs used in gene therapy. The collected samples will be prepared and aliquoted on site and then sent frozen to the GENETHON laboratory for analysis.

Only one sample per patient will be analyzed. All samples will be analyzed in batches.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of patients with anti-AAV neutralizing antibodies
Time Frame: Day 1
The primary endpoint is the percentage of patients who tested positive for neutralizing antibodies against multiple AAVs that can be used in gene therapy.
Day 1

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
Time Frame: Day 1
Estimation of seroprevalence (positive or negative) of anti-AAV neutralizing antibodies in different subgroups (age, sex, type of neuromuscular disease, immunosuppressive therapy)
Day 1
To analyze the co-prevalence between several AAV serotypes
Time Frame: Day 1
Quantification of neutralizing antibody titers against various capsids using cellular tests
Day 1
Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method
Time Frame: Day 1
Prevalence of cross-reactions between multiple capsids
Day 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genethon

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

July 31, 2026

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

May 26, 2026

First Submitted That Met QC Criteria

June 3, 2026

First Posted (Actual)

June 8, 2026

Study Record Updates

Last Update Posted (Actual)

June 8, 2026

Last Update Submitted That Met QC Criteria

June 3, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GNT-020-SDM
  • ID RCB N° 2026-A00986-45 (Other Identifier: French health authority (ANSM))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Individual participant data (IPD) will not be shared due to the sensitive nature of the data and the need to protect patient confidentiality, in accordance with applicable data protection regulations.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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