Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy i… (NCT01882062) | Clinical Trial Compass
CompletedPhase 2
Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease
France10 participantsStarted 2013-05
Plain-language summary
The purpose of this project is to study the efficacy of an anaplerotic treatment on brain energy profile evolution at an early stage of the Huntington disease.
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:
* 5 \< UHDRS \< 50
* Age \> 18 years
* Ability to undergo MR scanning
* Covered by french social security
Exclusion Criteria:
* Evidence of psychiatric disorder
* Attendant neurological disorder
* Contraindications to MRI (claustrophobia, metallic or material implants)
* Severe head injury
* Unable to understand the protocol
* Pregnancy
* Failure to give informed consent
* Subjects with exclusion criteria required by french law (e.g. subjects who require a legally authorized representative to obtain consent)
* Unwillingness to be informed in case of abnormal MRI
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.
1This trial was testing whether anaplerotic therapy could change specific energy markers in the brain — specifically the ratio of inorganic phosphate to phosphocreatine — what did the results actually show, and does that mean the treatment had a measurable effect on brain energy metabolism in Huntington disease?
2Since this was a Phase 2 proof-of-concept study that is now completed, has the data been published anywhere, and does the evidence from it support moving forward with anaplerotic approaches as a real treatment option for Huntington disease?
3The trial used brain phosphorus MRI spectroscopy to measure brain chemistry — is that kind of imaging something I would need access to if a similar treatment were being considered for me, and is it even available at our hospital?
4Given that this study focused on energy metabolism in the brain as a potential problem in Huntington disease, do you think addressing that pathway is worth pursuing for my specific situation, or are there other treatment approaches right now with stronger evidence?
5Because this trial is completed and was only a proof-of-concept study, does that mean the therapy it was testing is still experimental, and should I be looking at whether any follow-up trials are now recruiting?
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
1
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr