The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.
Age range
6 Months
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.
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.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
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.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Change From Baseline in Time Adjusted-Area Under the Curve (AUC/Time) for Workload During Cycle Ergometry at Week 24
Timeframe: Baseline, Week 24
Change From Baseline in Time-Adjusted-AUC for Respiratory Exchange Ratio (RER) During Cycle Ergometry at Week 24
Timeframe: Baseline, Week 24
Change From Baseline in Actual Duration of Exercise During Cycle Ergometry at Week 24
Timeframe: Baseline, Week 24
Change From Baseline in Distance Traveled During the 12-Minute Walk Test (12MWT) at Week 18
Timeframe: Baseline (last assessment during the 4-week run-in period), Week 18
Change From Baseline in Energy Expenditure Index (EEI) During the 12MWT at Week 18
Timeframe: Baseline (last assessment during the 4-week run-in period), Week 18
Change From Baseline in Percentage of the Predicted 6-Minute Walk Test (6MWT) Distance Walked at Week 18
Timeframe: Baseline (last assessment during the 4-week run-in period), Week 18
Change From Baseline in Physical Summary Score (PHS-10) of the Short Form 10 (SF-10) at Week 24
Timeframe: Baseline, Week 24
Change From Baseline in Psychosocial Summary Score (PSS-10) of the SF10 at Week 24
Timeframe: Baseline, Week 24
Change From Baseline in the Physical Component Summary Scale (PCS-12) at Week 24
Timeframe: Baseline, Week 24
Change From Baseline in the Mental Component Summary Scale (MCS-12) at Week 24
Timeframe: Baseline, Week 24
Annualized Event Rate of All Major Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Duration Rate of All Major Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Event Rate of Major Rhabdomyolysis Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Duration Rate of Major Rhabdomyolysis Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Event Rate of Major Hypoglycemia Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Duration Rate of Major Hypoglycemia Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Event Rate of Major Cardiac Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation
Annualized Duration Rate of Major Cardiac Clinical Events Pre- and Post-Treatment With UX007
Timeframe: 18 months before and after UX007 initiation