Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Blinded CDCA · 1 trial · 1 indication
Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test
| Arm | Type | Description |
|---|---|---|
| Adult Cohort | EXPERIMENTAL | Patients in the adult cohort will participate in a randomized, double-blind, placebo-controlled, 2 period × 2-treatment crossover study with rescue medication and open-label run-in to assess the efficacy and safety of CDCA. |
| Pediatric Cohort | EXPERIMENTAL | Pediatric cohort patients (≥1 month and \<16 years) will participate in a 24-week, open-label cohort with an 8-week titration period and a 16-week treatment period at the tolerated dose. |
| Name | Type | Description |
|---|---|---|
| Blinded CDCA 250 mg TID | DRUG | Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. |
| Placebo | DRUG | Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. |
| Open-Label CDCA 250 mg TID | DRUG | Adult cohort patients will receive open-label 250 mg CDCA TID during the run-in and washout periods of the study or as rescue medication during the double-blind periods, if needed, based on clinical symptoms. |
| Rescue Medication CDCA 250 mg TID | DRUG | CDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results. |
| CDCA Weight-Based Dose TID | DRUG | Patients in the pediatric cohort will complete a weight-based dose titration to a tolerated dose and will maintain that tolerated dose for the remainder of the study. Pediatric cohort dosing of CDCA will not exceed an equivalent dose of 750 mg/day. |
Inclusion Criteria: 1. Male or female at least 1 month or older at screening. 2. Clinical diagnosis of CTX with biochemical confirmation. 3. Women of childbearing potential must agree to the use of one highly reliable method of contraception during the study, plus one additional barrier method duri...