AVAILABLENot Applicable

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

United States

Plain-language summary

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.

Who can participate

Age range18 Years – 75 Years

SexALL

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Inclusion criteria

✓. 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;
✓. A sexually-active patient agrees to practice acceptable methods of contraception throughout the protocol period;
✓. The patient is not eligible for, or does not have access to, a clinical trial;
✓. 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 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;
✕. A medical history or condition that increases the potential for QTc prolongation;
✕. A female with a positive pregnancy test result or who is breastfeeding;
✕. Has received or is receiving kidney dialysis;
✕. Has received or is scheduled to receive an organ transplant;

Trial details

NCT IDNCT06931834

SponsorRiboNova Inc.

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

Contact for this trial

Zarazuela Zolkiplki-Cunningham, MBChB

267-426-4961 MMFP@chop.edu

View on ClinicalTrials.gov More Mitochondrial Disease trials