Neuromuscular DeficitsSeroDysMyoNCT07633951
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.
A study testing Dosage of AAV neutralizing antibodies in people with Neuromuscular Deficits and Genetic Disorder, run by Genethon at 1 site in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 to 60 years
- Conditions the study is for
- Neuromuscular Deficits, Genetic Disorder
- What is being tested?
- Dosage of AAV neutralizing antibodies
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 450 participants
- Where
- 1 siteFrance
- Study dates
- 31 Jul 2026to 31 Dec 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 6 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease
- Informed patient who signed informed consent
- No opposition from the holders of parental authority or guardian, for minor patients.
- Affiliated/beneficiary of a national health insurance scheme
Who cannot join
- Gene or cell therapy treatment prior to blood collection
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site
GENETHON
Évry, Île-de-France Region, 9100
Philippe VERON, Master of Sciences, veron@genethon.fr
Céline LABETOULE, Master of Sciences, clabetoule@genethon.fr
About this study
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.
Read the 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.
- Conditions
- Neuromuscular Deficits, Genetic Disorder
- Being tested
- Dosage of AAV neutralizing antibodies
- Keywords
- AAV, Seroprevalence, Nab, Tab, neuromuscular, Genetic disorder
- Medical subject headings
- Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dosage of AAV neutralizing antibodiesDiagnostic test
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis | Other | Not described Receives: Diagnostic Test: Dosage of AAV neutralizing antibodies |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients with anti-AAV neutralizing antibodies
The primary endpoint is the percentage of patients who tested positive for neutralizing antibodies against multiple AAVs that can be used in gene therapy.
Measured: Day 1
Secondary outcomes (3)
To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
Estimation of seroprevalence (positive or negative) of anti-AAV neutralizing antibodies in different subgroups (age, sex, type of neuromuscular disease, immunosuppressive therapy)
Measured: Day 1
To analyze the co-prevalence between several AAV serotypes
Quantification of neutralizing antibody titers against various capsids using cellular tests
Measured: Day 1
Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method
Prevalence of cross-reactions between multiple capsids
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2026
- Study start31 Jul 2026 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DeficitsStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 450 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genethon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07633951
- Sponsor's protocol ID
- GNT-020-SDM
- Other IDs
- ID RCB N° 2026-A00986-45
- Acronym
- SeroDysMyo
- Status last verified
- May 2026
- First submitted
- 26 May 2026
- Last updated
- 8 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT07633951
- Acronym / sponsor ID
- SeroDysMyo / GNT-020-SDM
- Conditions
- Neuromuscular Deficits, Genetic Disorder
- Keywords
- AAV, Seroprevalence, Nab, Tab, neuromuscular, Genetic disorder
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 450 participants (estimated)
- Lead sponsor
- GenethonUniversity, hospital or other
- Study start
- 31 Jul 2026 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 8 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis | Other | Diagnostic Test: Dosage of AAV neutralizing antibodies |
Who may qualify
- Ages
- 6 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease
- Informed patient who signed informed consent
- No opposition from the holders of parental authority or guardian, for minor patients.
- Affiliated/beneficiary of a national health insurance scheme
Key exclusion criteria (1 listed)
- Gene or cell therapy treatment prior to blood collection
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 |
|---|---|---|
| Dosage of AAV neutralizing antibodiesIn 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. | Diagnostic test | Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis |
Primary outcome
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.
3 secondary outcomes
To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
Time frame: Day 1
To analyze the co-prevalence between several AAV serotypes
Time frame: 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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
- Individual participant dataSharing planned: No. 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.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Dosage of AAV neutralizing antibodies in people with Neuromuscular Deficits and Genetic Disorder, run by Genethon at 1 site in France.
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: opening soon, as of the sponsor's last update on 8 Jun 2026. 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 France. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of patients with anti-AAV neutralizing antibodies (time frame: Day 1). 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 8 Jun 2026.