ABOUT THIS STUDY
1. Male and female patients age ≥ 18
2. Diagnosis of LGMD2I as defined by clinical presentation consistent with LGMD2I and FKRP gene testing showing biallelic alterations known or likely to be pathogenic. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
3. Ability to walk/run 10m
4. Ability to rise from chair
5. Adequate hepatic and renal function on screening laboratory assessments
6. Iron content estimate on the screening liver MRI within the normal range as determined by R2* value (R2* ≤ 139 Hz at 3.0T).
7. Participant must provide written informed consent for participating in study.
8. Participant must possess the ability, per the Principal Investigator (PI), to understand and comply with protocol instruction for the entire duration of the study.
1. Known cognitive impairment or behavioral issues that would impede the ability to
provide informed consent or to follow study instructions.
2. History of major surgical procedure within 6 weeks of signing the informed consent or
planned surgery during the study.
3. Any injury which may impact functional testing. Previous injuries must be fully healed
prior to consent. Prior lower limb fractures must be fully healed and at least 3
months from injury dates.
4. Previous treatment with another investigational product within 30 days or 5
half-lives, (whichever is longer) prior to consenting.
5. Corticosteroid treatment within 3 months prior to consenting.
6. Compromised cardiac function (left ventricular ejection fraction <50%).
7. Unwilling or unable (e.g. metal implants, requires sedation) to undergo examination
with closed MRI without sedation.
8. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein.
9. Female subjects who are pregnant or nursing.
10. Subjects who, are biologically capable of having children who are unwilling or unable
to use highly effective methods of contraception (as outlined in this protocol) during
sexual activity for the duration of the study and through completion of final study
11. Predisposition to iron accumulation. (Serum iron >1.2 X ULN, serum ferritin >1.2
12. Underlying disposition for bleeding disorder on screening laboratory assessment
(PT/INR>1.25 X ULN, aPTT > 1.25 ULN, fecal occult blood is positive)
13. Evidence or history of clinically significant hematological, renal, endocrine,
pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, or allergic disease.
14. Unwillingness or inability to comply with the requirements of this protocol (in the
opinion of the PI) including, but not limited to, the presence of any condition
(physical, mental, or social) that is likely to affect the participant's ability to
return for study visits or adhere to the visit schedule.
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