Mitochondrial DiseaseNCT06931834
Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
A study testing Probucol in people with Mitochondrial Disease and Chronic Kidney Disease, run by RiboNova Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Mitochondrial Disease, Chronic Kidney Disease
- What is being tested?
- Probucol
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A patient must meet all of the following criteria to be eligible:
- 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.
Who cannot join
- A patient who meets any of the following criteria will not be eligible:
- 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;
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- 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;
- Known or suspected active alcohol and/or substance abuse;
- A history of or current suicidal ideation, behavior, and/or attempts; or
- Is unable to swallow tablets or requires gastric feeding.
Trial locations and contacts
1 site in 1 country.
United States1 site
- Available
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Zarazuela Zolkipli-Cunningham, MBChB, 267-426-4961, MMFP@chop.edu
Sara Nguyen, MPH, 267-426-1986, MMFP@chop.edu
About this study
Official title: 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.
Read the 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.
- Conditions
- Mitochondrial Disease, Chronic Kidney Disease
- Being tested
- Probucol
- Keywords
- Mitochondrial disease, Chronic kidney disease, Myopathy, Genetic disease, Rare disease, Probucol
- Medical subject headings
- Mitochondrial Diseases, Renal Insufficiency, Chronic, Muscular Diseases, Genetic Diseases, Inborn, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ProbucolDrug
Also called R007, Lorelco
Probucol 250 mg tablets orally up to a daily dose of 1,000 mg
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- RiboNova Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital of Philadelphia
- Responsible party
- Sponsor
- Investigators
- Zarazuela Zolkipli-Cunningham, MBChB, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06931834
- Sponsor's protocol ID
- EP2025010101
- Status last verified
- Apr 2025
- First submitted
- 4 Apr 2025
- Last updated
- 17 Apr 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Treatment Protocol for R007 (Probucol) Tablets in Adults With Genetically-Confirmed Mitochondrial Disease and Chronic Kidney Disease (Intermediate-Size Patient Population)
- ClinicalTrials.gov ID
- NCT06931834
- Acronym / sponsor ID
- EP2025010101
- Conditions
- Mitochondrial Disease, Chronic Kidney Disease
- Keywords
- Mitochondrial disease, Chronic kidney disease, Myopathy, Genetic disease, Rare disease, Probucol
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- RiboNova Inc.Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital of Philadelphia
- First posted
- 17 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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;
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ProbucolAlso: R007, LorelcoProbucol 250 mg tablets orally up to a daily dose of 1,000 mg | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Zarazuela Zolkipli-Cunningham, MBChB principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Probucol in people with Mitochondrial Disease and Chronic Kidney Disease, run by RiboNova Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Zarazuela Zolkiplki-Cunningham, MBChB (267-426-4961, MMFP@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2025.