A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
NCT ID: NCT05230459
Last Updated: 2025-12-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
10 participants
INTERVENTIONAL
2023-05-15
2032-12-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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AB-1003 Cohort 1
AB-1003 dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 1
AB-1003 Cohort 2
AB-1003 dose level 2
Single intravenous infusion of AB-1003 gene therapy at dose level 2
Placebo (Cohorts 1 and 2)
Placebo
Single intravenous infusion of Placebo
Interventions
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AB-1003 dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 1
AB-1003 dose level 2
Single intravenous infusion of AB-1003 gene therapy at dose level 2
Placebo
Single intravenous infusion of Placebo
Eligibility Criteria
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Inclusion Criteria
2. Ability to ascend 4 stairs between 2.5 and 10 seconds.
3. Ability to walk/run 10 meters in \<30 seconds.
4. Able to understand and comply with all study procedures.
5. 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.
Exclusion Criteria
2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
4. 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).
5. Abnormal renal function (GFR \<60 ml/min, using the Modification of Diet in Renal Disease equation).
6. 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).
7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
8. Requirement for daytime ventilatory support.
9. Change in glucocorticosteroid treatment within 3 months prior to screening visit.
10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
11. Ongoing participation in any other therapeutic clinical trial.
12. Neutralizing antibody titer to AAV9 \>1:5.
13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
18 Years
65 Years
ALL
No
Sponsors
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AskBio Inc
INDUSTRY
Responsible Party
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Locations
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University of California - Irvine
Irvine, California, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
VCU
Richmond, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
Countries
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Central Contacts
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myTomorrows (see link below in reference section)
Role: CONTACT
Facility Contacts
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UCI Alpha Clinic
Role: primary
Ciara Gibbs
Role: primary
Andrea Klempnauer
Role: primary
Mary Yep
Role: primary
Anarosa Rezeq
Role: primary
Piya Modalavalasa
Role: primary
Related Links
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Link to webpage for study and pre-qualification information
Other Identifiers
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LION-CS101
Identifier Type: -
Identifier Source: org_study_id