Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Triheptanoin · 9 trials · 13 indications
Heart rate during constant load cycling exercise.
Palmitate oxidation measured via stable isotope technique and indiret calorimetry during constant load exercise.
Subject heart rate will be measured during 20 minutes exercise test performed on a cycle ergometer at a workload corresponding to approximately 60% of maximal oxidative capacity (VO2max).
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.
Amyotrophic lateral sclerosis functional rating scale-revised version (ALSFRS-R) is used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS and each is scored between 0 (no function at all) and 4 (normal function). Thus the overall score for this measure can range from 0 to 48, with higher scores indicating more normal function. The test-retest reliability is greater than 0.88 for all 12 items/activities. The ALSFRS-R declines linearly with time over a wide range during the course of ALS and the minimum clinically significant change in this scale is said to be 20%. The primary analysis in this study is the slope of the ALSFRS-R during treatment compared to pre-treatment. Pre-treatment slope for each participant will be estimated as follows: (48-enrollment ALSFRS-R)/months since symptom onset. This frequently used "pre-slope" method is simple and inexpensive, and can predict disease progression.
Change in lactate levels, comparing results from before and after triheptanoin is initiated - this will be measured by the number of participants who experience any change; measured in mmol/L
Change in pyruvate levels, comparing results from before and after triheptanoin is initiated - this will be measured by the number of participants who experience any change; measured in mg/dl
Change in β-hydroxybutyrate levels, comparing results from before and after triheptanoin is initiated - this will be measured by the number of participants who experience any change; measured in mmol/L
Change in Alanine/Leucine ratios, comparing results from before and after triheptanoin is initiated - this will be measured by the number of participants who experience any change; measured in μmol/L
Change in Alanine/Lysine ratios, comparing results from before and after triheptanoin is initiated - this will be measured by the number of participants who experience any change; measured in μmol/L
Change in Alanine/Proline ratios, comparing results from before and after triheptanoin is initiated - this will be measured by the number of participants who experience any change; measured in μmol/L
Measured by a reduction or alteration of home antiepileptics use, from before and after triheptanoin is initiated
Measured by a reduction in episodes of metabolic decompensation, from before and after triheptanoin is initiated
Measured by a reduction in the frequency of disease related hospitalizations, from before and after triheptanoin is initiated
Reviewing the change in blood glucose levels from baseline to 6 month visit
| Arm | Type | Description |
|---|---|---|
| Triheptanoin | EXPERIMENTAL | Participants will be given prescriptions for triheptanoin in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. Triheptanoin will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. |
| MCT | ACTIVE_COMPARATOR | Participants will be given prescriptions for MCT in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. MCT will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. |
| Active treatment | EXPERIMENTAL | Triheptanoin oil |
| Placebo treatment | PLACEBO_COMPARATOR | Safflower oil |
| Placebo oil | PLACEBO_COMPARATOR | 14 days of diet on a placebo oil including 7 days titration period and 7 days full dose treatment of 1mL/kg/day. |
| Schedule A | EXPERIMENTAL | Subjects previously treated with triheptanoin |
| Schedule B | EXPERIMENTAL | Naïve to triheptanoin |
| Active/Placebo | OTHER | Subjects receive 1-2 grams/kilogram body weight triheptanoin divided into 4 equal doses taken with meals and snack for 6 months crossing over to receive placebo vegetable oil at the same dose and frequency for the next 6 months during the randomization phase. |
| Placebo/Active | OTHER | Subjects receive 1-2 grams/kilogram body weight placebo vegetable oil divided into 4 equal doses taken with meals and snack for 6 months crossing over to receive triheptanoin at the same dose and frequency for the next 6 months during the randomization phase. |
| 1.0 gm/kg/day triheptanoin | EXPERIMENTAL | Up to 24 subjects (8: 4-9 years of age, 8: 10-15 years of age; 8: 16 years of age or older) will take 1.0 gm/kg/day divided into 3 or 4 doses |
| Group 1 | EXPERIMENTAL | Standard care for 1 month, then standard care and Triheptanoin for 5 months. |
| Group 2 | EXPERIMENTAL | Standard care and Triheptanoin for 6 months. |
| Group 3 | NO_INTERVENTION | Healthy controls for biomarkers |
| Name | Type | Description |
|---|---|---|
| Triheptanoin | DRUG | Liquid for oral (PO) or enteral feeding tube administration |
| MCT Oil | DIETARY_SUPPLEMENT | Liquid for oral (PO) or enteral feeding tube administration |
| Placebo Oil | DRUG | Daily treatment with Safflower oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day). |
| Vegetable Oil | OTHER | 1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 g triheptanoin (drug)/kilogram body weight for 6 months. |
Inclusion Criteria for Main Study: * Males and females, from 0 (including newborns) to \< 18 years of age at time of randomization * Confirmed diagnosis of LC-FAOD * Have a caregiver(s) willing and able to assist in all applicable study requirements * Have a legally authorized representative willin...
Triheptanoin is an investigational small molecule being studied for Tarui Disease, Pyruvate Dehydrogenase Complex Deficiency, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS), ALS, and Glycogen Storage Disease Type I. It is in clinical development for these neurological and metabolic conditions.
Triheptanoin is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on NASDAQ under the ticker RARE. The company is conducting clinical trials to evaluate the drug for multiple rare metabolic and neurological disorders.
Triheptanoin is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. Clinical trials are ongoing or completed to evaluate its safety and efficacy for various indications.
Triheptanoin has been studied in several clinical trials, including NCT03506425 for ALS, NCT03642860 for glycogenoses, NCT03665636 for Glycogen Storage Disease Type I, and NCT06340685 for Pyruvate Dehydrogenase Complex Deficiency. These trials are at various stages of completion.
Triheptanoin is the drug name used in clinical trials. No alternative names have been reported for this compound in the available clinical trial information.