RESTORE Study Design
RESTORE: randomized,
double-blind, placebo-controlled crossover trial1
- 14 patients enrolled, 13 randomized to receive either CTEXLI 250 mg or matching placebo 3x/day for 4 weeks1
- A 4-phase study including 2 separate 4-week, double-blind withdrawal periods1*
TID=3 times a day.
*The study also included an 8-week, run-in period of CTEXLI 250 mg TID and an 8-week, open-label period between the 2 double-blind withdrawal periods. Total duration of treatment was 24 weeks.1
Baseline patient characteristics (n=13)1
Age
35 years
Median age at diagnosis
(15-55)
42 years
Median age at baseline
(16-55)
Racial Group
62%
White
23%
Other
15%
Asian
Ethnicity
15%
Hispanic/Latino
54%
Not Hispanic/Latino
31%
Unknown
Gender
62%
Male
39%
Female
RESTORE 24-week crossover study design1-3
Plasma cholestanol and urine 23S-pentol were assessed at multiple time points over 24 weeks of treatment.1
Significant difference between CTEXLI and placebo seen within 4 weeks1
4x
lower level of plasma cholestanol with continued CTEXLI treatment vs placebo (change from baseline to Day 29)1
(CTEXLI –2.3 μ/mL vs placebo 6.2 μ/mL; estimated treatment difference: –8.5 μ/mL [95% CI: –13.2, –3.9])
For each study treatment (placebo or CTEXLI), the mean value at baseline was calculated as the mean of the measurements obtained prior to receiving the study treatment during the double-blind study duration. The mean value at Day 29 was calculated as the mean of the measurements at Day 29 at the end of the study treatment.1
For each patient at each visit, the measurement of urine 23S-pentol was calculated as the geometric mean of the first 3 morning void urine samples collected within 5 days prior to the visit.1
Significant reductions during the 8-week, open-label CTEXLI phase (P=0.0012)3,4
150x
lower level of urine 23S-pentol with continued CTEXLI treatment vs placebo (change from baseline to Day 29)1
(CTEXLI 185 ng/mL vs placebo 29,506 ng/mL; estimated treatment difference: –29,321 ng/mL [95% CI: –45,701, –12,941])
Within 8 weeks of starting the open-label CTEXLI phase, significant reductions in urine 23S-pentol were seen in both groups (P=0.0012).1
Adherence is critical: significant increases in toxic bile acid precursors were seen within 2 weeks of stopping CTEXLI4
These significant increases were seen when patients switched from CTEXLI to placebo in the double-blind withdrawal periods.