Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

Inclusion Criteria

A patient must meet all of the following criteria to be eligible:

1. Age 18 to 75 years inclusive;

2. Diagnosis of genetically-confirmed mitochondrial disease with a pathogenic mutation
or defect in their mitochondrial or nuclear DNA;

3. Diagnosis of chronic kidney disease stage 3 or stage 4;

4. Moderate or severe symptomatology of mitochondrial disease;

5. A sexually-active patient agrees to practice acceptable methods of contraception
throughout the protocol period;

6. The patient is not eligible for, or does not have access to, a clinical trial;

7. The patient (or guardian/legal representative) provides signed and dated informed
consent to be treated with probucol in accordance with the expanded access protocol.

Exclusion Criteria

A patient who meets any of the following criteria will not be eligible:

1. A known hypersensitivity or adverse reaction to probucol;

2. A medical condition or laboratory result that, in the opinion of the treating
physician, will interfere with the safe completion of the protocol;

3. An active fungal infection, acute blood, lung, or bladder infection, clinically
significant hepatic or renal dysfunction, and/or viral infection (past or present)
that, in the opinion of the treating physician, may have a clinically significant
effect on their treatment;

4. Taking an investigational drug other than probucol within one month prior to the
first dosing with probucol under the protocol;

5. A medical history or condition that increases the potential for QTc prolongation;

6. A female with a positive pregnancy test result or who is breastfeeding;

7. Has received or is receiving kidney dialysis;

8. Has received or is scheduled to receive an organ transplant;

9. Known or suspected active alcohol and/or substance abuse;

10. A history of or current suicidal ideation, behavior, and/or attempts; or

11. Is unable to swallow tablets or requires gastric feeding.