Trial record 1 of 1666 for:
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Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
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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. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04270682 |
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Recruitment Status :
Completed
First Posted : February 17, 2020
Last Update Posted : November 22, 2023
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Sponsor:
Mirum Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.
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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 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 18 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 |
| Actual Primary Completion Date : | July 13, 2023 |
| Actual Study Completion Date : | October 4, 2023 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cerebrotendinous xanthomatosis
| Arm | Intervention/treatment |
|---|---|
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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.
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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. |
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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.
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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 :
- Change in Bile Alcohols. [ Time Frame: Week 4 and Week 16 ]Change from baseline in 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:
- Male or female at least 1 month or older at screening.
- Clinical diagnosis of CTX with biochemical confirmation.
- 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.
- 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:
- Genetic testing does not confirm CTX.
- Malabsorption disorder or confounding inflammatory gastrointestinal condition (for example, irritable bowel syndrome).
- Documented history of heart failure.
- Treated with medications which impact bile acid absorption such as bile acid sequestering agents (eg, cholestyramine, colestipol, aluminum-based antacids.
- Treated with cholic acid medication.
- Female patient who is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
- 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
Locations
| United States, Colorado | |
| Travere Investigational Site | |
| Aurora, Colorado, United States, 80045 | |
| United States, Florida | |
| Travere Investigational Site | |
| Orlando, Florida, United States, 32806 | |
| United States, Iowa | |
| Travere Investigational Site | |
| Iowa City, Iowa, United States, 52242 | |
| United States, Louisiana | |
| Travere Investigational Site | |
| New Orleans, Louisiana, United States, 70112 | |
| Travere Investigational Site | |
| New Orleans, Louisiana, United States, 70121 | |
| United States, New York | |
| Travere Investigational Site | |
| Great Neck, New York, United States, 11021 | |
| United States, Ohio | |
| Travere Investigational Site | |
| Columbus, Ohio, United States, 43221 | |
| United States, Texas | |
| Travere Investigational Site | |
| Austin, Texas, United States, 78723 | |
| United States, Washington | |
| Travere Investigational Site | |
| Seattle, Washington, United States, 98195 | |
| Brazil | |
| Travere Investigational Site | |
| Fortaleza, CE, Brazil, 60430-270 | |
| Travere Investigational Site | |
| Porto Alegre, RS, Brazil, 90035-903 | |
| Travere Investigational Site | |
| São Paulo, SP, Brazil, 04024-002 | |
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
| Responsible Party: | Mirum Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT04270682 |
| Other Study ID Numbers: |
Cheno-CTX-301 |
| First Posted: | February 17, 2020 Key Record Dates |
| Last Update Posted: | November 22, 2023 |
| Last Verified: | November 2023 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Mirum Pharmaceuticals, Inc.:
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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 |