CompletedPhase 2

Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5

France12 participantsStarted 2015-01-08

Plain-language summary

The purpose of this project is to study the efficacy of three candidate molecules (Xenbilox, Tahor and Resveratrol) in order to decrease the production of oxysterols by reducing the synthesis of cholesterol and/or regulate the production of bile acids and/or enabling neuroprotective action within the motor neuron.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * patients that have confirmed through genetic testing their status as carriers of 2 mutations in the CYP7B1 gene * age ≥ 18 years * patients that have signed the informed consent form * presence of health care coverage Exclusion Criteria: * known hypersensitvity to chenodeoxycholic acid, atorvastatin, resveratrol or to any of their byproducts * cholesterol lowering medications other than the study treatment * hepatic failure with transaminases \>3 times the normal level * progressive biliary pathology * chronic diarrhea * serious mental illness * significant comorbid neurological disorder * incapacity to understand information about the protocol * unwilling or unable to participate in any part of the study * participation in another clinical trial during the study period * person deprived of liberty by judicial or administrative decision * adult subject under legal protection or unable to consent * pregnant or breastfeeding women * lack of health care coverage * absence of a signed informed consent form

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment

Timeframe: 2 months

Trial details

NCT IDNCT02314208

SponsorInstitut National de la Santé Et de la Recherche Médicale, France

Sponsor typeOTHER_GOV

Study typeINTERVENTIONAL

Primary completion2017-09-27

View on ClinicalTrials.gov More Spastic Paraplegia, Hereditary trials