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Tests treatment safety and results for Limb Girdle Muscular Dystrophy
Official title A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
ClinicalTrials.gov ID: NCT05230459
What this study is testing
What is AB-1003 dose level 1?
AB-1003 dose level 1 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for limb girdle muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- Male and female people 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 people receiving study intervention must use a barrier method...
You likely can't join if
- Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \ 480m/sec), NYHA Class 3 or 4 heart failure, or MRI...
- 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...
- 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...
See the full eligibility criteria
- Male and female people 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 people receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.
- Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \ 480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial...
- 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...
- 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).
- 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 people who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
The study team makes the final eligibility decision.
Where it's taking place
- Irvine, California, United States
- Iowa City, Iowa, United States
- Kansas City, Kansas, United States
- Baltimore, Maryland, United States
- Richmond, Virginia, United States
- Seattle, Washington, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Irvine, California, United States; Iowa City, Iowa, United States; Kansas City, Kansas, United States; Baltimore, Maryland, United States; Richmond, Virginia, United States; Seattle, Washington, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.