LGMD2CPhase 1NCT05973630
ATA-200 Gene Therapy Trial in Patients With LGMDR5
A Phase 1 study testing ATA-200 in people with LGMD2C, run by Atamyo Therapeutics at 1 site in United States.
Trial summary
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Read more about this study
- Who is it for?
- Ages 6 to 13 years
- Conditions the study is for
- LGMD2C
- What is being tested?
- ATA-200
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Feb 2025to 31 Jan 2031
- Phase
- Phase 1
- 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 13 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
- Ambulant male or female patients aged 6 to less than 12 years of age at screening
- Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support
Who cannot join
- Detectable neutralizing antibodies against AAV8
- Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50%
- Respiratory assistance
- Concomitant medical condition that might interfere with LGMDR5 evolution
- Acute illness within 4 weeks of anticipated IMP administration
- Current participation in another clinical trial with investigational medicinal product
Show all 12Show fewer
- Previous participation in gene and cell therapy trials
- Any condition that would contraindicate immunosuppressant treatment
- Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
- Any vaccination 1 month prior to planned IMP administration
- Serology consistent with HIV exposure or active hepatitis B or C infection
- Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation
Trial locations and contacts
1 site in 1 country.
United States1 site
Child Health Research Institute
Gainsville, Florida, 32610
About this study
Official title: A Phase 1-2, Open-label Study to Evaluate the Safety of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).
Read the detailed description
This is a multicenter Phase 1b assessing the safety and tolerability of intravenous ATA-200 for the treatment of LGMDR5.
The study will enroll ambulant patients with LGMDR5. Patients will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. Patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies.
Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months.
All subjects will be followed up for an additional 4.5 years after completion of the initial evaluation period.
- Conditions
- LGMD2C
- Being tested
- ATA-200
- Medical subject headings
- Limb-girdle muscular dystrophy, type 2C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATA-200Biological
single intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm | Experimental | ATA-200 Dose : 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h Receives: Biological: ATA-200 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Collection of adverse events at each visit
Measured: 0-6 months
Incidence of treatment-emergent adverse events,
Collection of adverse events at each visit
Measured: 0-6 months
Incidence of serious adverse events
Collection of adverse events at each visits
Measured: 0-6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2023
- Study start15 Feb 2025
- Primary completion30 Oct 2026 (estimated)
- Study completion31 Jan 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LGMD2CStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Atamyo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05973630
- Sponsor's protocol ID
- ATA-003-GSAR
- Status last verified
- Jan 2026
- First submitted
- 24 Jul 2023
- Last updated
- 29 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1-2, Open-label Study to Evaluate the Safety of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)
- ClinicalTrials.gov ID
- NCT05973630
- Acronym / sponsor ID
- ATA-003-GSAR
- Conditions
- LGMD2C
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Atamyo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 15 Feb 2025 (actual)
- Primary completion
- 30 Oct 2026 (estimated)
- Study completion
- 31 Jan 2031 (estimated)
- First posted
- 3 Aug 2023 (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.
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).
Detailed description
This is a multicenter Phase 1b assessing the safety and tolerability of intravenous ATA-200 for the treatment of LGMDR5.
The study will enroll ambulant patients with LGMDR5. Patients will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. Patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies.
Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months.
All subjects will be followed up for an additional 4.5 years after completion of the initial evaluation period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment arm | Experimental | Biological: ATA-200ATA-200 Dose : 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h |
Who may qualify
- Ages
- 6 to 13 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
- Ambulant male or female patients aged 6 to less than 12 years of age at screening
- Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support
Key exclusion criteria (12 listed)
- Detectable neutralizing antibodies against AAV8
- Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50%
- Respiratory assistance
- Concomitant medical condition that might interfere with LGMDR5 evolution
- Acute illness within 4 weeks of anticipated IMP administration
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 |
|---|---|---|
| ATA-200single intravenous infusion | Biological | Treatment arm |
Primary outcome
Incidence of adverse events
Time frame: 0-6 months
Collection of adverse events at each visit
Incidence of treatment-emergent adverse events,
Time frame: 0-6 months
Collection of adverse events at each visit
Incidence of serious adverse events
Time frame: 0-6 months
Collection of adverse events at each visits
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 29 Jan 2026.
- Individual participant dataSharing planned: Undecided
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ATA-200 in people with LGMD2C, run by Atamyo Therapeutics at 1 site in United States.
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 3 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 29 Jan 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 United States. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: 0-6 months). The study lists 3 primary outcomes in all. 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 29 Jan 2026.