Trial · NCT02214160
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 17 of 17
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) | The annualized LC-FAOD MCE rate, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, defined as any visit to the emergency room (ER)/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25 |
| Primary | Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) | The annualized LC-FAOD MCE rate, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, defined as any visit to the emergency room (ER)/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25 |
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days | An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. A serious adverse event (SAE) results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; an important medical event. AEs were graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (mild=1, moderate=2, severe=3, life-threatening=4, death=5). |
| Secondary | Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) | Baseline, Month 12, Month 18, Month 24, Month 30, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) | Baseline, Month 12, Month 24, Month 36 | The Z-scores express the deviation (or how far away) the measure is from the mean LVEF based on the size or age of the pediatric participants: Z-score=0 indicates the participant is exactly the same as the mean of the healthy general population. Z-score=-1 indicates it's 1 standard deviation below the mean of the healthy population. Z-score=+1 indicates it's 1 standard deviation above the mean. |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) | Baseline, Month 12, Month 24, Month 30, Month 36, Month 48, Month 60 | Fractional shortening is calculated by measuring the percentage change in left ventricular diameter during systole. A negative value indicates less ventricular/muscular contractility, and a positive value indicates more ventricular/muscular contractility. |
| Secondary | Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | The Z-scores express the deviation (or how far away) the measure is from the mean LVSF based on the size or age of the pediatric participants: Z-score=0 indicates the participant is exactly the same as the mean of the healthy general population. Z-score=-1 indicates it's 1 standard deviation below the mean of the healthy population. Z-score=+1 indicates it's 1 standard deviation above the mean. |
| Secondary | Annualized Duration Rate of All MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized duration rate of LC-FAOD MCEs, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, and defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. |
| Secondary | Annualized Event Rate of Rhabdomyolysis MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized event rate of LC-FAOD major events of skeletal myopathy (rhabdomyolysis), defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25. |
| Secondary | Annualized Duration Rate of Rhabdomyolysis MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized duration rate of LC-FAOD skeletal myopathy (rhabdomyolysis) MCEs, defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. |
| Secondary | Annualized Event Rate of Cardiomyopathy MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized event rate of LC-FAOD major events inclusive of cardiomyopathy events, defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25. |
| Secondary | Annualized Duration Rate of Cardiomyopathy MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized duration rate of LC-FAOD cardiomyopathy MCEs, defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25 |
| Secondary | Annualized Event Rate of Hypoglycemic MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized event rate of LC-FAOD major events of hepatic (hypoglycemia) events, defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25. |
| Secondary | Annualized Duration Rate of Hypoglycemic MCEs | Post-UX007 treatment through the end of the study (up to 2072 days) | The annualized duration rate of LC-FAOD hepatic (hypoglycemia) MCEs, defined as any visit to the ER/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT02214160/registered