University of Kansas Medical Center

Eligibility

Eligible Ages

Between 12 Years and 60 Years

Eligible Genders

All

Accepts Healthy Volunteers

No

Criteria

Participants must meet all the following criteria to be enrolled:

1. Have a genetically confirmed diagnosis of LGMD2I/R9 (including review of records of
previous molecular genetic testing) and be clinically affected (defined as
demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern,
or in a distal extremity).

2. Male or female participants 12 to 60 years of age (inclusive).

3. Have a body weight >30 kg.

4. The participant (or parent/guardian) who signs the ICF understands the study
procedures and the participant agrees to participate in the study by giving informed
consent (and assent, if <18 years of age).

5. Female participants of childbearing potential and male participants of reproductive
potential must be willing to use a highly effective method of contraception from time
of consent through 12 weeks after last dose.

6. Willing and able to complete all study procedures, including biopsies, according to
the Schedule of Assessments (see Appendix 1).

Participants must not meet any of the following criteria to be enrolled:

1. Evidence of clinically significant concomitant disease, including:

1. Any significant concomitant medical condition, including mental, cardiac, renal,
pulmonary, hepatic, or endocrine disease other than that associated with
LGMD2I/R9.

2. Moderate to severe renal impairment (estimated glomerular filtration rate [eGFR]
of < 60 mL/min/1.73 m2 based on cystatin C [CysC]), as calculated by the central
laboratory.

3. Any other laboratory, vital sign, ECG abnormality, clinical history, or finding
that, in the Investigator's opinion, is likely to unfavorably alter the
risk-benefit of study participation, confound study results, or interfere with
study conduct or compliance.

4. Surgery for scoliosis or other indication that will significantly impact the
participant's ability to execute clinical assessments planned or expected to be
required to manage curvature within 12 months following the Screening Visit.

2. A participant with a score of zero on any one or more of the primary or key secondary
endpoints at the time of screening. (Participants who previously completed
participation in Study MLB-01-001 and would be excluded due to this criterion may
enroll in this study provided all inclusion and no other exclusion criteria are met.)

3. If pregnant and/or breastfeeding or planning to conceive children within the projected
duration of the study through 12 weeks after the last dose of study treatment.

4. Use of ribose or other sugar alcohol-containing supplement within 90 days of the
Screening Visit.

5. Use of a systemic corticosteroid for the treatment of muscular dystrophy within 90
days of the Screening Visit. (An inhaled corticosteroid or bronchodilator for reactive
airway disease is allowed if the participant is on a stable dose for 30 days prior to
study entry.)

6. Previously received gene therapy to treat LGMD2I/R9.

7. Participants with active suicidal ideation as measured by Columbia-Suicide Severity
Rating Scale during screening with most severe suicide ideation score of 4 (Active
Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active
Suicidal Ideation with Specific Plan and Intent).

8. Presence of a platelet disorder, bleeding disorder, or other contraindication to
muscle biopsy.

9. Actively on an experimental therapy or device or was on an experimental therapy or
device within 90 days of the Screening Visit.

10. In the judgment of the Investigator or Medical Monitor, has any clinically important
ongoing medical condition or laboratory abnormality or condition that might jeopardize
the participant's safety, increase their risk from participation, or interfere with
the study. For COVID-19 infections, Investigator should refer to local guidance.