Ctexli Drug Information
Generic name: CHENODIOL
Bile Acid [EPC]
Uses of Ctexli
CTEXLI is indicated for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.
Dosage & Administration of Ctexli
Important Recommendation Prior to CTEXLI Treatment Initiation Before initiating CTEXLI, obtain baseline liver transaminase (alanine aminotransferase and aspartate aminotransferase ) and total bilirubin levels in all patients.
Recommended Dosage
The recommended dosage of CTEXLI is 250 mg administered orally three times daily. Administer CTEXLI with or without food. Swallow tablets whole.
Missed Dose If a dose of CTEXLI is missed, advise the patient to skip the missed dose and to resume taking the prescribed dose at the next scheduled time. Patients should not take a double dose.
Administration Modification and Monitoring
If liver transaminase (ALT, AST) levels are elevated > 3 times the upper limit of normal (ULN) or total bilirubin level is >2 times ULN, interrupt treatment with CTEXLI until the levels have returned to baseline values. Monitor liver transaminase and total bilirubin levels yearly and as clinically indicated.
Side Effects of Ctexli
Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. CTEXLI is not approved for use in pediatric patients. The dosage of CTEXLI was 250 mg orally three times a day.
The mean (SD) chenodiol exposure during Trial 1 was 139.1 days. In Trial 1, one CTEXLI-treated patient (7%) had increased ALT levels > 3x ULN, which led to treatment interruption.
Postmarketing Experience
The following adverse reactions have been identified during post approval use of chenodiol. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure. • Hepatobiliary Disorders: Hepatotoxicity • Immune System Disorders: Hypersensitivity reactions such as facial swelling, pruritus, rash, urticaria.
Warnings & Cautions for Ctexli
Hepatotoxicity
Chenodiol, including CTEXLI, has been associated with hepatotoxicity. In Trial 1, one CTEXLI-treated patient (7%) had increased ALT levels > 3 times ULN, which led to treatment interruption. Patients with pre-existing liver disease or bile duct abnormalities may be at higher risk for hepatotoxicity during treatment with CTEXLI.
Published reports suggest patients who are poor sulfators of lithocholic acid are more likely to develop chenodiol-induced serum aminotransferase elevations. Obtain baseline liver transaminase (ALT, AST) and total bilirubin levels in all patients prior to treatment initiation with CTEXLI. If liver transaminase levels are elevated > 3 times ULN or total bilirubin level is >2 times ULN, interrupt treatment with CTEXLI until the levels have returned to baseline values.
Monitor liver transaminase and total bilirubin levels yearly and as clinically indicated. For persistent or recurrent liver test abnormalities, consider discontinuing CTEXLI. Inform the patient of the symptoms of hepatotoxicity (e.g., abdominal pain, bruising, dark-colored urine, fatigue, bleeding, jaundice, nausea, and pruritus).
If clinical signs and symptoms consistent with hepatotoxicity occur, have the patient discontinue CTEXLI immediately.
Drug Interactions with Ctexli
Effect of Other Drugs on CTEXLI Co-administration of bile acid sequestering agents, such as cholestyramine and colestipol, or aluminum-based antacids may decrease absorption of CTEXLI in the intestine and may result in decreased efficacy of CTEXLI. Avoid concomitant use of bile acid sequestering agents or aluminum-based antacids with CTEXLI.
Effect of CTEXLI on Other Drugs Due to potential hepatotoxicity, CTEXLI may affect the pharmacodynamics of coumarin and its derivatives, causing unexpected prolongation of the prothrombin time and hemorrhage. If concomitant use of CTEXLI with coumarin or its derivatives is unavoidable, monitor prothrombin time. Adjust the dosage of coumarin or its derivatives in accordance with its approved product labeling.
Pregnancy Safety for Ctexli
Pregnancy Risk Summary Available data from published case reports over decades of use with chenodiol during pregnancy have not identified an increased risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Serious hepatic, renal and adrenal lesions occurred in fetuses of female Rhesus monkeys treated at doses 1 to 2 times the recommended human dose based on body surface area (mg/m 2 ). Hepatic lesions also occurred at doses comparable to the human dose based on body surface area in neonatal baboons born to mothers administered chenodiol during pregnancy ( see Data ).
The animal study findings have not been demonstrated with human use. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.
In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is respectively. Data Animal Data Hepatic lesions were reported in neonatal baboons whose mothers had received 18 to 38 mg/kg of chenodiol throughout pregnancy (0.6 to 1.4 times the recommended human dose based on body surface area). Non-human primates form sulfate conjugates of the known hepatotoxic bacterial metabolite of chenodiol, lithocholic acid, to a lesser extent than reported in humans, which may exaggerate the toxicity of orally dosed chenodiol compared to humans.
However, there is also evidence that the hepatobiliary toxicity is partly due to the parent drug, chenodiol.
Pediatric Use of Ctexli
Pediatric Use The safety and effectiveness of CTEXLI for the treatment of CTX have not been established in pediatric patients.
Overdosage Information for Ctexli
Cases of intentional overdose with chenodiol have been reported: one patient consumed 3 g to 4.5 g of chenodiol and another patient consumed 30 g of chenodiol. Clinical manifestations experienced by these patients included nausea, dizziness, and diarrhea. In the event of an overdose, discontinue CTEXLI, monitor the patient, and institute general supportive measures if needed.
Clinical Studies of Ctexli
The efficacy of CTEXLI for the treatment of patients with CTX was evaluated in Trial 1, which was a randomized, double-blind, placebo controlled, 2-period with 2-treatment crossover trial in patients ≥16 years of age (NCT 04270682). The study also included treatment with CTEXLI 250 mg three times daily during an 8 week run-in period and an 8-week open label period in between the 2 double-blind withdrawal periods. The total duration of study treatment was 24 weeks.
Of the 13 randomized patients, 62% were male and 39% were female. CTEXLI is not approved for use in pediatric patients. The patient population consisted of 62% White, 15% Asian, and 23% Other (In the Other racial group, there was one patient who reported both White and Black).
Ethnicity consisted of 15% Hispanic or Latino, 54% not Hispanic or Latino, and 31% unknown. Plasma cholestanol and urine 23S-pentol were assessed at multiple time points as shown in Figure 1. For plasma cholestanol, the estimated mean change from baseline at day 29 was -2.3 µg/mL when patients continued CTEXLI treatment and 6.2 µg/mL when patients received placebo.
For urine 23S-pentol, the estimated mean change from baseline at day 29 was 185 ng/mL when patients continued CTEXLI treatment and 29506 ng/mL when patients received placebo. Figure 1: Mean (SE) of Observed Plasma Cholestanol by Treatment Sequence (All Randomized Patients) DB = double-blind. Solid line represents treatment with CTEXLI and dashed line represents treatment with placebo.
The mean values of plasma cholestanol at baselines and end of each DB period are annotated in the figure. Table 1: Summary Results for Plasma Cholestanol and Urine 23S Pentol Figure 1: Mean (SE) of Observed Plasma Cholestanol by Treatment Sequence (All Randomized Patients)
| 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; and the mean value at Day 29 was calculated as the mean of the measurements at Day 29 at the end of the study treatment. For each patient at each visit, the measurement of urine 23S-pentol was calculated as the geometric mean of first 3 morning void urine samples collected within 5 days prior to the visit. | |||
| Plasma Cholestanol (µg/mL) | Mean (SD) | CTEXLI (N = 13) | Placebo (N = 13) |
| Baseline | 10.8 (10.0) | 8.8 (7.8) | |
| Day 29 | 8.5 (7.0) | 15.1 (8.8) | |
| Change from Baseline at Day 29 | -2.3 (3.9) | 6.2 (5.6) | |
| Treatment Difference | -8.5 (95% CI: -13.2, -3.9) | ||
| Urine 23S- Pentol (ng/mL) | Baseline | 1811 (1693) | 1773 (1940) |
| Day 29 | 1996 (1341) | 31279 (27595) | |
| Change from Baseline at Day 29 | 185 (1479) | 29506 (27257) | |
| Treatment Difference | -29321 (95% CI: -45701, -12941) | ||
Drug information sourced from the FDA. This content is for informational purposes only and does not constitute medical advice. Consult a healthcare professional before making any medication decisions.
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