Clinical Research Opportunity: LION-CS101 a Phase 1/2 Study of AB-1003 in Adults with LGMD2I/R9

Researchers at AskBio are seeking adults with genetically confirmed limb-girdle muscular dystrophy (LGMD) type 2I/R9 to participate in a phase 1/2 clinical trial (LION-CS101) to evaluate the safety and tolerability of the investigational gene therapy AB-1003 to treat LGMD2I/R9. LGMD2I/R9 is a rare form of LGMD caused by changes in the FKRP gene and is associated with muscle weakness in the arms, legs, and torso. AB-1003 is a gene therapy designed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9. The study is examining whether this treatment is safe and well-tolerated in adults with the disease. Cohort 1 of this study is complete, and the investigators are currently recruiting for Cohort 2.

The study

LION-CS101 is a phase 1/2, double-blind, randomized, placebo-controlled study. This means that participants will be randomly assigned to receive the study drug, AB-1003, or an inactive placebo control. Following the study, an open-label extension period will be open to eligible participants, during which all participants will receive AB-1003. The total duration of the study for participants will be five to seven years. In year one, participants will be required to attend approximately 17 check-ins, including a combination of clinic and home visits. In the subsequent four years, participants will be monitored twice a year for the long-term follow-up. Concierge services to support study visits, including transportation, lodging and meals, may be available for study participants.

The drug will be administered by a single intravenous (in the vein) infusion during a 24-hour hospital stay. The effects of AB-1003 will be evaluated using a number of tests and procedures including but not limited to: physical checkups/assessments, muscle biopsies (needle), blood tests, pulmonary function tests, cardiac MRI, lower extremity MRI, echocardiogram, and quality of life questionnaires.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:

• Male and female individuals between the ages 18 and 65 years with a clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation
• Ability to ascend 4 stairs between 2.5 and 10 seconds
• Ability to walk/run 10 meters in < 30 seconds
• Additional requirements, which will be discussed with the study investigators

Individuals may not be eligible to participate if they are affected by another illness or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

To learn more about the study or inquire about participation, please visit the study website or contact AskBio at AskFirst@AskBio.com.