A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (HD)
- Conditions
- neurodegenerative movement disorder10029299Huntington's Disease10028037
- Registration Number
- NL-OMON43815
- Lead Sponsor
- The French National Institute of Health and Medical Research (Inserm)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 50
- positive genetic test with CAG repeat length * 39 in HTT gene
- at least 18 years of age
- signature of informed consent
- UHDRS score between 5 and 40
- Ability to undergo MRI scanning
- hypersensitivity to triheptanoin or to one of its excipients
- additional psychiatric or neurological conditions
- severe head injury
- farticipation in another therapeutical trial ( 3 months exclusion period)
- for women of childbearing age, the absence of two forms of effective contraception (with the exception of those who are abstinent)
- for men, the absence of an effective form of contraception (e.g. a condom) throughout the study period
- pregnancy or breastfeeding
- pnability to understand information about the protocol
- persons deprived of their liberty by judicial or unable to consent
- adult subject under legal protection or unable to consent
- treatment with tetrabenazine, sodium valporate, and pancreatic inhibitors
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>a. An increase in the index of brain energy restoration * as defined by the<br /><br>difference between Pi/PCr ratio during visual stimulation and the mean of<br /><br>Pi/PCr ratio during rest and recovery * using P-MRS after 3 months of treatment.<br /><br>b. A decrease in the rate of caudate atrophy, using volumetric MRI, after 6<br /><br>months of treatment with triheptanoin in early affected HD patients.</p><br>
- Secondary Outcome Measures
Name Time Method