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Study to Evaluate Patients With Cerebrotendinous Xanthomatosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04270682
Recruitment Status : Recruiting
First Posted : February 17, 2020
Last Update Posted : February 17, 2020
Sponsor:
Information provided by (Responsible Party):
Retrophin, Inc.

Brief Summary:
The study is made up of two cohorts: a randomized double-blind crossover (placebo withdrawal with rescue) study among patients ≥ 16 years of age (adult cohort) and an open-label dose titration study among pediatric patients ≥1 month and <16 years of age (pediatric cohort)

Condition or disease Intervention/treatment Phase
CTX Drug: Blinded CDCA 250 mg TID Drug: Placebo Drug: Open-Label CDCA 250 mg TID Drug: Rescue Medication CDCA 250 mg TID Drug: CDCA Weight-Based Dose TID Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: Two-cohort study. One cohort is for adult patients with a double-blind placebo withdrawal (with CDCA rescue) crossover. Second cohort will dose titrate pediatric patients into a stable, open-label treatment.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis
Actual Study Start Date : January 31, 2020
Estimated Primary Completion Date : March 24, 2021
Estimated Study Completion Date : March 31, 2021


Arm Intervention/treatment
Experimental: Adult Cohort
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.
Drug: Blinded CDCA 250 mg TID
Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.

Drug: Placebo
Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.

Drug: Open-Label CDCA 250 mg TID
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.

Drug: Rescue Medication CDCA 250 mg TID
CDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results.

Experimental: Pediatric Cohort
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.
Drug: CDCA Weight-Based Dose TID
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.




Primary Outcome Measures :
  1. Change in Urinary Bile Alcohols [ Time Frame: Week 4 and Week 16 ]
    Change from baseline in urine bile alcohols at the end of each DB treatment period.



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Ages Eligible for Study:   1 Month and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 during sexual activity.
  4. Males must be surgically sterile, or males and their sexual partners must together agree to use medically accepted methods of contraception that are considered highly reliable during the course of the study.

Exclusion Criteria:

  1. Genetic testing does not confirm CTX.
  2. Malabsorption disorder or confounding inflammatory gastrointestinal condition (for example, irritable bowel syndrome).
  3. Documented history of heart failure.
  4. Treated with medications which impact bile acid absorption such as bile acid sequestering agents (eg, cholestyramine, colestipol, aluminum-based antacids.
  5. Treated with cholic acid medication.
  6. Female patient who is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
  7. Positive at screening for the human immunodeficiency virus (HIV) or markers indicating acute or chronic hepatitis B infection or hepatitis C infection.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04270682


Contacts
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Contact: Retrophin Medical Information 1-877-659-5518 medinfo@retrophin.com

Locations
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United States, Louisiana
Retrophin Investigational Site Recruiting
New Orleans, Louisiana, United States, 70112
Principal Investigator: Cheno-CTX-301 Investigator         
United States, Washington
Retrophin Investigational Site Recruiting
Seattle, Washington, United States, 98195
Principal Investigator: Cheno-CTX-301 Investigator         
Sponsors and Collaborators
Retrophin, Inc.

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Responsible Party: Retrophin, Inc.
ClinicalTrials.gov Identifier: NCT04270682    
Other Study ID Numbers: Cheno-CTX-301
First Posted: February 17, 2020    Key Record Dates
Last Update Posted: February 17, 2020
Last Verified: February 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Retrophin, Inc.:
Cerebrotendinous xanthomatosis
CTX
Cholestanol
Leukodystrophy
CYP27A1
Additional relevant MeSH terms:
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Xanthomatosis, Cerebrotendinous
Xanthomatosis
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Chenodeoxycholic Acid
Cathartics
Gastrointestinal Agents