Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age18-75
SponsorRiboNova Inc.
About this trial
The purpose of this expanded access program is to enable access to R007 (probucol) tablets for the compassionate treatment of adults with mitochondrial disease who also have chronic kidney disease.
Eligibility criteria
Qualifiers
Age 18 to 75 years inclusive;
Diagnosis of genetically-confirmed mitochondrial disease with a pathogenic mutation or defect in their mitochondrial or nuclear DNA;
Diagnosis of chronic kidney disease stage 3 or stage 4;
Moderate or severe symptomatology of mitochondrial disease;
Disqualifiers
A known hypersensitivity or adverse reaction to probucol;
A medical condition or laboratory result that, in the opinion of the treating physician, will interfere with the safe completion of the protocol;
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;
Taking an investigational drug other than probucol within one month prior to the first dosing with probucol under the protocol;
Trial design
Treatments tested in this trial
- Probucol
Treatment groups
No treatment groups listed
Sponsors and collaborators
RiboNova Inc.
Lead sponsor
Children's Hospital of Philadelphia
Collaborator
Source ClinicalTrials.gov