Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02453061 | A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease | PHASE2 | COMPLETED | 100 | — | — | Jun 29, 2015 | Dec 3, 2019 | Jul 15, 2024 | 2 | France, Netherlands |
A decrease in the rate of caudate atrophy, using volumetric MRI
| Arm | Type | Description |
|---|---|---|
| Placebo group | PLACEBO_COMPARATOR | Subjects will receive safflower oil at 1g/kg/day for 6 months (months 0 - 6) and triheptanoin oil at 1g/kg/day for 6 months (months 7 - 12) |
| Triheptanoin group | ACTIVE_COMPARATOR | Subjects will receive triheptanoin oil at 1g/kg/day for 6 months (months 0 - 6) and triheptanoin oil at 1g/kg/day for 6 months (months 7 - 12) |
| Name | Type | Description |
|---|---|---|
| Triheptanoin oil | DRUG | Triheptanoin oil orally administered at 1g/kg/day |
| Placebo | DRUG | Safflower oil orally administered at 1g/kg/day |
Inclusion Criteria: * Positive genetic test with CAG repeat length ≥39 in HTT gene * At least 18 years of age * Signature of informed consent * Covered by social security * UHDRS score between 5 and 40 * Ability to undergo MRI scanning * BMI between 18 and 30 Exclusion Criteria: * Hypersensitivit...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 2 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |