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.
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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