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...
Blinded CDCA is an investigational small molecule being studied for the treatment of cerebrotendinous xanthomatosis (CTX), a rare genetic condition. It is currently in Phase 3 clinical development and is not yet approved by the FDA.
Blinded CDCA is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol MIRM. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with CTX.
Blinded CDCA is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet received regulatory approval and is still being evaluated in clinical trials for the treatment of cerebrotendinous xanthomatosis (CTX).
Blinded CDCA is associated with the clinical trial NCT04270682, titled 'Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)'. This Phase 3 trial has been completed and enrolled 19 participants in the United States and Brazil.
Blinded CDCA is a designation used in clinical trials to refer to the investigational drug being studied. The name indicates that the treatment is blinded, meaning neither the participants nor the researchers know who receives the active drug versus a control during the study.