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.