Limb Girdle Muscular DystrophyPhase 1/2NCT05230459
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
A Phase 1/2 study testing AB-1003 dose level 1 and AB-1003 dose level 2 in people with Limb Girdle Muscular Dystrophy and Limb-Girdle Muscular Dystrophy Type 2, run by AskBio Inc at 6 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1) Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I
- What is being tested?
- AB-1003 dose level 1 and AB-1003 dose level 2
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 6 sitesUnited States
- Study dates
- 15 May 2023to Dec 2032
- Phase
- Phase 1/2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
- Ability to walk/run 10 meters in <30 seconds.
- Able to understand and comply with all study procedures.
- Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.
Who cannot join
- Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction <40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF >480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.
- Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
- Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
- History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin >upper limit of normal [ULN] and/or elevated AST and ALT >2 ULN).
- Abnormal renal function (GFR <60 ml/min, using the Modification of Diet in Renal Disease equation).
- Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).
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- In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
- Requirement for daytime ventilatory support.
- Change in glucocorticosteroid treatment within 3 months prior to screening visit.
- Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
- Ongoing participation in any other therapeutic clinical trial.
- Neutralizing antibody titer to AAV9 >1:5.
- Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
University of Iowa
Iowa City, Iowa, 52242
University of California - Irvine
Irvine, California, 92697
University of Kansas Medical Center
Kansas City, Kansas, 66160
VCU
Richmond, Virginia, 23298
University of Washington Medical Center
Seattle, Washington, 98195
About this study
Official title: A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
- Conditions
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD
- Being tested
- AB-1003 dose level 1, AB-1003 dose level 2, Placebo
- Keywords
- gene therapy, LGMD2I, LGMD2I/R9, gene augmentation therapy, FKRP, fukutin related protein, FKRP mutation
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle, Limb-girdle muscular dystrophy type 2A, Muscular Dystrophy, Limb-Girdle, Type 2I, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AB-1003 dose level 1Genetic
Single intravenous infusion of AB-1003 gene therapy at dose level 1
AB-1003 dose level 2Genetic
Single intravenous infusion of AB-1003 gene therapy at dose level 2
PlaceboDrug
Single intravenous infusion of Placebo
| Study group | Role | What they receive |
|---|---|---|
| AB-1003 Cohort 1 | Experimental | Not described Receives: Genetic: AB-1003 dose level 1 |
| AB-1003 Cohort 2 | Experimental | Not described Receives: Genetic: AB-1003 dose level 2 |
| Placebo (Cohorts 1 and 2) | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
Measured: 0-52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2022
- Study start15 May 2023
- Primary completionDec 2028 (estimated)
- Study completionDec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb Girdle Muscular DystrophyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- AskBio Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05230459
- Sponsor's protocol ID
- LION-CS101
- Status last verified
- Aug 2026
- First submitted
- 27 Jan 2022
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
- ClinicalTrials.gov ID
- NCT05230459
- Acronym / sponsor ID
- LION-CS101
- Conditions
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD
- Keywords
- gene therapy, LGMD2I, LGMD2I/R9, gene augmentation therapy, FKRP, fukutin related protein, FKRP mutation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (actual)
- Lead sponsor
- AskBio IncIndustry (pharma, biotech, devices)
- Study start
- 15 May 2023 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2032 (estimated)
- First posted
- 9 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.
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AB-1003 Cohort 1 | Experimental | Genetic: AB-1003 dose level 1 |
| AB-1003 Cohort 2 | Experimental | Genetic: AB-1003 dose level 2 |
| Placebo (Cohorts 1 and 2) | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
- Ability to walk/run 10 meters in <30 seconds.
- Able to understand and comply with all study procedures.
- Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.
Key exclusion criteria (13 listed)
- Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction <40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF >480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.
- Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
- Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
- History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin >upper limit of normal [ULN] and/or elevated AST and ALT >2 ULN).
- Abnormal renal function (GFR <60 ml/min, using the Modification of Diet in Renal Disease equation).
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 |
|---|---|---|
| AB-1003 dose level 1Single intravenous infusion of AB-1003 gene therapy at dose level 1 | Genetic | AB-1003 Cohort 1 |
| AB-1003 dose level 2Single intravenous infusion of AB-1003 gene therapy at dose level 2 | Genetic | AB-1003 Cohort 2 |
| PlaceboSingle intravenous infusion of Placebo | Drug | Placebo (Cohorts 1 and 2) |
Primary outcome
Adverse Events
Time frame: 0-52 weeks
Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
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 26 Aug 2026.
- Individual participant dataSharing planned: No
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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.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AB-1003 dose level 1 and AB-1003 dose level 2 in people with Limb Girdle Muscular Dystrophy and Limb-Girdle Muscular Dystrophy Type 2, run by AskBio Inc at 6 sites 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 4 inclusion and 13 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 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Adverse Events (time frame: 0-52 weeks). 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 26 Aug 2026.