LGMDR9Phase 1NCT05224505
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
A Phase 1 study testing ATA-100 (AAV9 encoding FKRP gene) in people with LGMDR9, run by Atamyo Therapeutics at 3 sites in 3 countries.
Trial summary
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up Read more about this study
- Who is it for?
- Ages 16 to 99 years
- Conditions the study is for
- LGMDR9
- What is being tested?
- ATA-100 (AAV9 encoding FKRP gene)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 1 Sep 2022to 30 Sep 2029
- 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 16 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Female and male ambulant patients
- 2. Patients ≥ 16 years old
- 3. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- 4. Moderate diaphragmatic muscle impairment
Who cannot join
- 1. Detectable serum neutralizing antibodies against AAV9
- 2. Cardiomyopathy
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
Denmark1 site
Rigshospitalet, University of Copenhagen Blegdamsvej 9
Copenhagen, 2100
France1 site
Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital
Paris, 75013
United Kingdom1 site
Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing
Newcastle upon Tyne, NE1 4LP
About this study
Official title: A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
Read the detailed description
Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.
This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.
Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.
All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.
- Conditions
- LGMDR9
- Being tested
- ATA-100 (AAV9 encoding FKRP gene)
- Medical subject headings
- Muscular Dystrophy, Limb-Girdle, Type 2I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATA-100 (AAV9 encoding FKRP gene)Biological
Single intravenous infusion on Day 0
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Cohort 1: single intravenous injection 9.0E+12 vg/Kg Receives: Biological: ATA-100 (AAV9 encoding FKRP gene) |
| Cohort 2 | Experimental | Cohort 2: single intravenous injection 2.7E+13 vg/Kg Receives: Biological: ATA-100 (AAV9 encoding FKRP gene) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
Incidence of Adverse Events and significant laboratory changes
Measured: Baseline through 12 months
Secondary outcomes (10)
Combined endpoint (global testing)
Change in North Star Assessment for musculary Dystrophy (NSAD) and Forced Vital capacity (FVC %) from baseline
Measured: Baseline through 12 months
Timed Up and Go (TUG) test
Secondary endpoint
Measured: Baseline through 12 months
Change from baseline in velocity as measured by 10MWT
10-meter walk test
Measured: Baseline through 12 months
2-minute walk distance test
Secondary endpoint
Measured: Baseline through 12 months
Cardiac MRI
To measure cardiac function (left ejection fraction)
Measured: Baseline through 12 months
Muscle MRI
To measure change from baseline in fat repartition fraction in thigh and leg skeletal muscles
Measured: Baseline through 12 months
Muscle Biopsy
Quantification of FKRP positive muscle fibers
Measured: Baseline through 12 months
Muscle Biopsy
Percentage of glycosylation
Measured: Baseline through 12 months
Patient reported outcome and quality of life assessment
Quality of Life in genetic Neuromuscular Disease (QoL-gNMD), with a range from 0 to 78, the higher the score the worse the quality of life
Measured: Baseline through 12 months
Patient reported outcome and quality of life assessment
ACTIVLIM, scale measuring level of limitation in performing daily activities (total score ranging from 0 to 44, with the lower score the highest limitation)
Measured: Baseline through 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2022
- Study start1 Sep 2022
- Primary completion1 Feb 2025
- Study completion30 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LGMDR9Studies 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
- Sequential
- Model description
- Single IV administration of ATA-100, open label, 2 sequential cohorts
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 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
- NCT05224505
- Sponsor's protocol ID
- ATA-001-FKRP
- Other IDs
- 2021-004276-33
- Status last verified
- Oct 2025
- First submitted
- 15 Dec 2021
- Last updated
- 6 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)
- ClinicalTrials.gov ID
- NCT05224505
- Acronym / sponsor ID
- ATA-001-FKRP
- Conditions
- LGMDR9
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Atamyo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 1 Feb 2025 (actual)
- Study completion
- 30 Sep 2029 (estimated)
- First posted
- 4 Feb 2022 (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.
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
Detailed description
Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.
This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.
Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.
All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Single IV administration of ATA-100, open label, 2 sequential cohorts
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: ATA-100 (AAV9 encoding FKRP gene)Cohort 1: single intravenous injection 9.0E+12 vg/Kg |
| Cohort 2 | Experimental | Biological: ATA-100 (AAV9 encoding FKRP gene)Cohort 2: single intravenous injection 2.7E+13 vg/Kg |
Who may qualify
- Ages
- 16 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 1. Female and male ambulant patients
- 2. Patients ≥ 16 years old
- 3. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- 4. Moderate diaphragmatic muscle impairment
Key exclusion criteria (2 listed)
- 1. Detectable serum neutralizing antibodies against AAV9
- 2. Cardiomyopathy
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-100 (AAV9 encoding FKRP gene)Single intravenous infusion on Day 0 | Biological | Cohort 1, Cohort 2 |
Primary outcome
Safety and tolerability
Time frame: Baseline through 12 months
Incidence of Adverse Events and significant laboratory changes
10 secondary outcomes
Combined endpoint (global testing)
Time frame: Baseline through 12 months
Timed Up and Go (TUG) test
Time frame: Baseline through 12 months
Change from baseline in velocity as measured by 10MWT
Time frame: Baseline through 12 months
2-minute walk distance test
Time frame: Baseline through 12 months
Cardiac MRI
Time frame: Baseline through 12 months
Muscle MRI
Time frame: Baseline through 12 months
Muscle Biopsy
Time frame: Baseline through 12 months
Muscle Biopsy
Time frame: Baseline through 12 months
Patient reported outcome and quality of life assessment
Time frame: Baseline through 12 months
Patient reported outcome and quality of life assessment
Time frame: Baseline through 12 months
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 6 Oct 2025.
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LGMD2I
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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.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ATA-100 (AAV9 encoding FKRP gene) in people with LGMDR9, run by Atamyo Therapeutics at 3 sites in 3 countries.
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 2 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 6 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Denmark, France, United Kingdom. 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?
Safety and tolerability (time frame: Baseline through 12 months). 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 6 Oct 2025.