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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)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | Primary Analysis of Change from Baseline Plasma Cholestanol (Natural Log-transformed) at the End of the Two Double-Blind Periods- Paired T-test | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
| Change From Baseline Plasma 7αC4 During the Two Double-Blind Periods |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Travere Investigational Site | Aurora | Colorado | 80045 | United States | ||
| Travere Investigational Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | Sequence AB | Patients in the adult cohort participated 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. Patients randomized to sequence AB received blinded 250 mg CDCA TID for DB1 and Placebo TID for DB2. Open-Label CDCA 250 mg TID: Adult cohort patients received 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: CDCA 250 mg TID was provided as rescue medication during the double-blind periods, if needed, based on laboratory results. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 4, 2023 | Jul 12, 2024 |
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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.
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| Placebo | Drug | Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. |
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| 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. |
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| 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. |
|
Primary Analysis of Change from Baseline Plasma 7αC4 (Natural Log-Transformed) During the Two Double-Blind Periods |
| Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
| Proportion of Participants Who Received Rescue Treatment During Two Double-Blind Periods | Proportion of Participants Who Received Rescue Treatment during Two Double-Blind Periods - Prescott's Method | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
| Orlando |
| Florida |
| 32806 |
| United States |
| Travere Investigational Site | Iowa City | Iowa | 52242 | United States |
| Travere Investigational Site | New Orleans | Louisiana | 70112 | United States |
| Travere Investigational Site | New Orleans | Louisiana | 70121 | United States |
| Travere Investigational Site | Great Neck | New York | 11021 | United States |
| Travere Investigational Site | Columbus | Ohio | 43221 | United States |
| Travere Investigational Site | Austin | Texas | 78723 | United States |
| Travere Investigational Site | Seattle | Washington | 98195 | United States |
| Travere Investigational Site | Fortaleza | Ceará | 60430-270 | Brazil |
| Travere Investigational Site | Porto Alegre | Rio Grande do Sul | 90035-903 | Brazil |
| Travere Investigational Site | São Paulo | São Paulo | 04024-002 | Brazil |
| FG001 | Sequence BA | Patients in the adult cohort participated 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. Patients randomized to sequence BA received blinded Placebo TID for DB1 and 250 mg CDCA TID for DB2. Open-Label CDCA 250 mg TID: Adult cohort patients received 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: CDCA 250 mg TID was provided as rescue medication during the double-blind periods, if needed, based on laboratory results. |
| FG002 | Pediatric Cohort | Pediatric cohort patients (≥1 month and <16 years) participated in a 24-week, open-label cohort with an 8-week titration period and a 16-week treatment period at the tolerated dose. CDCA Weight-Based Dose TID: Patients in the pediatric cohort completed a weight-based dose titration to a tolerated dose and maintained that tolerated dose for the remainder of the study. Pediatric cohort dosing of CDCA was not to exceed an equivalent dose of 750 mg/day. |
| COMPLETED |
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| NOT COMPLETED |
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Adult Cohort: Start of Run-In Period [Day-56/R1W1, First Day] Pediatric Cohort: Start of Treatment (Day 1)
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| ID | Title | Description |
|---|---|---|
| BG000 | Sequence AB | 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. Patients randomized to sequence AB will receive blinded CDCA TID for 4 weeks or until open-label rescue medication criteria are triggered 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. Placebo: 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: 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. |
| BG001 | Sequence BA | 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. Patients randomized to sequence BA will receive placebo TID for 4 weeks or until either the blinded and/or open-label rescue medication criteria are triggered. 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. Placebo: 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: 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: CDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results. |
| BG002 | 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. 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. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Thirteen of 14 enrolled participants (92.9%) were randomized and completed the study. One participant was enrolled, but was not randomized, and withdrew from the study. | Mean | Standard Deviation | years |
| ||||||||||||||
| Sex: Female, Male | Thirteen of 14 enrolled participants (92.9%) were randomized and completed the study. One participant was enrolled, but was not randomized, and withdrew from the study. | Count of Participants | Participants |
| |||||||||||||||
| Race/Ethnicity, Customized | Thirteen of 14 enrolled participants (92.9%) were randomized and completed the study. One participant was enrolled, but was not randomized, and withdrew from the study. | Count of Participants | Participants | No |
| ||||||||||||||
| Region of Enrollment | Thirteen of 14 enrolled participants (92.9%) were randomized and completed the study. One participant was enrolled, but was not randomized, and withdrew from the study. | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test | All adult cohort participants who were randomized and took at least one dose of randomized study medication. | Posted | Mean | Standard Deviation | natural log of ng/mL | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
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| Secondary | Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | Primary Analysis of Change from Baseline Plasma Cholestanol (Natural Log-transformed) at the End of the Two Double-Blind Periods- Paired T-test | All adult cohort participants who were randomized and took at least one dose of randomized study medication, and who had been on CDCA for at least 2 months prior to the open-label run-in period. | Posted | Mean | Standard Deviation | natural log of μg/mL | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
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| Secondary | Change From Baseline Plasma 7αC4 During the Two Double-Blind Periods | Primary Analysis of Change from Baseline Plasma 7αC4 (Natural Log-Transformed) During the Two Double-Blind Periods | All adult cohort participants who were randomized and took at least one dose of randomized study medication. | Posted | Mean | Standard Deviation | natural log of ng/mL | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
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| Secondary | Proportion of Participants Who Received Rescue Treatment During Two Double-Blind Periods | Proportion of Participants Who Received Rescue Treatment during Two Double-Blind Periods - Prescott's Method | All adult cohort participants who were randomized and took at least one dose of randomized study medication. | Posted | Number | participants | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 |
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AEs (including SAEs) were captured from the time informed consent was signed to the patient's final visit. In addition, AEs (including SAEs) were to be collected for all patients via a follow-up phone call 30 (±7) days after the last dose of study medication to assess patient safety. Adult Cohort: 24 weeks duration Pediatric Cohort: 16 week duration
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | CDCA | Patients in the adult cohort ; blinded CDCA 250 mg TID in DB1 or DB2 Treatment-Emergent Adverse Events (TEAEs) were defined as those that start from Open-label Period 1 throughout the study, or existing AEs that worsen during or after Open-label Period 1. | 0 | 13 | 0 | 13 | 13 | 13 |
| EG001 | Placebo | Patients in the adult cohort ; Placebo 250 mg TID in DB1 or DB2 Treatment-Emergent Adverse Events (TEAEs) were defined as those that start from Open-label Period 1 throughout the study, or existing AEs that worsen during or after Open-label Period 1. | 0 | 13 | 1 | 13 | 13 | 13 |
| EG002 | 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. | 0 | 5 | 0 | 5 | 5 | 5 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gait Disturbance | General disorders | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Upper Respiratory Tract Infection | Infections and infestations | Systematic Assessment |
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| Gastroenteritis | Gastrointestinal disorders | Systematic Assessment |
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| Gastroenteritis Viral | Gastrointestinal disorders | Systematic Assessment |
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| Muscular Weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
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| Gait Disturbance | General disorders | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
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| Skin Abrasion | Injury, poisoning and procedural complications | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | Systematic Assessment |
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| Aminotransferase Increased | Investigations | Systematic Assessment |
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| Bilirubin Increase | Investigations | Systematic Assessment |
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| Athralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Tremor | Nervous system disorders | Systematic Assessment |
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| Hypertension | Vascular disorders | Systematic Assessment |
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| Ataxia | Nervous system disorders | Systematic Assessment |
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| Cerebral disorder | Nervous system disorders | Systematic Assessment |
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| Disturbance in attention | Nervous system disorders | Systematic Assessment |
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| Dizzyness | Nervous system disorders | Systematic Assessment |
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| Paraesthesia | Nervous system disorders | Systematic Assessment |
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| Neck Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Blood bilirubin increased | Infections and infestations | Systematic Assessment |
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| Electroencephalogram abnormal | Investigations | Systematic Assessment |
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| Tandem gait test abnormal | Investigations | Systematic Assessment |
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| Oedema peripheral | General disorders | Systematic Assessment |
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| Hot Flush | Vascular disorders | Systematic Assessment |
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| Hypoacusis | Ear and labyrinth disorders | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | Systematic Assessment |
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| Dysuria | Renal and urinary disorders | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | Systematic Assessment |
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| Acarodermatitis | Infections and infestations | Systematic Assessment |
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| Lip Infection | Infections and infestations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mirum Clinical Trials | Mirum Pharmaceuticals | 650-667-4085 | medinfo@mirumpharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 17, 2023 | Jul 12, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D019294 | Xanthomatosis, Cerebrotendinous |
| ID | Term |
|---|---|
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D014973 | Xanthomatosis |
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| Male |
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| White |
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| Other |
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| Multiple |
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| Brazil |
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| Pooled change from baseline to last non missing postbaseline Double Blind record |
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