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

April 11, 2025 updated by: RiboNova Inc.

Expanded Access Treatment Protocol for R007 (Probucol) Tablets in Adults With Genetically-Confirmed Mitochondrial Disease and Chronic Kidney Disease (Intermediate-Size Patient Population)

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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

The protocol for this expanded access program provides for the compassionate treatment with R007 (probucol) tablets of adults who (i) have serious or life-threatening mitochondrial disease and also have chronic kidney disease stages 3 or 4, (ii) do not have access to a satisfactory alternative therapy to treat their condition, and (iii) are not able to enroll in a clinical trial.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Available
        • The Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

RiboNova Inc.

Collaborators

Children's Hospital of Philadelphia

Investigators

  • Principal Investigator: Zarazuela Zolkipli-Cunningham, MBChB, Children's Hospital of Philadelphia

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

April 4, 2025

First Submitted That Met QC Criteria

April 11, 2025

First Posted (Actual)

April 17, 2025

Study Record Updates

Last Update Posted (Actual)

April 17, 2025

Last Update Submitted That Met QC Criteria

April 11, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EP2025010101

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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