CTXPhase 3NCT04270682
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
A Phase 3 study testing Blinded CDCA 250 mg TID, Open-Label CDCA 250 mg TID and Rescue Medication CDCA 250 mg TID in people with CTX, run by Mirum Pharmaceuticals, Inc. at 12 sites in 2 countries.
Trial 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) Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- CTX
- What is being tested?
- Blinded CDCA 250 mg TID, Open-Label CDCA 250 mg TID and Rescue Medication CDCA 250 mg TID
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 31 Jan 2020to 4 Oct 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
12 sites in 2 countries.
12 sites in 2 countries
United States9 sites
Travere Investigational Site
Aurora, Colorado, 80045
Travere Investigational Site
Austin, Texas, 78723
Travere Investigational Site
Columbus, Ohio, 43221
Travere Investigational Site
Great Neck, New York, 11021
Travere Investigational Site
Iowa City, Iowa, 52242
Travere Investigational Site
New Orleans, Louisiana, 70112
Travere Investigational Site
New Orleans, Louisiana, 70121
Travere Investigational Site
Orlando, Florida, 32806
Travere Investigational Site
Seattle, Washington, 98195
Brazil3 sites
Travere Investigational Site
Fortaleza, Ceará, 60430-270
Travere Investigational Site
Porto Alegre, Rio Grande do Sul, 90035-903
Travere Investigational Site
São Paulo, São Paulo, 04024-002
About this study
Official title: A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis
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)
- Conditions
- CTX
- Being tested
- Blinded CDCA 250 mg TID, Placebo, Open-Label CDCA 250 mg TID, Rescue Medication CDCA 250 mg TID, CDCA Weight-Based Dose TID
- Keywords
- Cerebrotendinous xanthomatosis, CTX, Cholestanol, Leukodystrophy, CYP27A1
- Medical subject headings
- Xanthomatosis, Cerebrotendinous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blinded CDCA 250 mg TIDDrug
Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.
PlaceboDrug
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 TIDDrug
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 TIDDrug
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 TIDDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Blinded CDCA 250 mg TID; Drug: Placebo; Drug: Open-Label CDCA 250 mg TID; Drug: Rescue Medication CDCA 250 mg TID |
| 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. Receives: Drug: CDCA Weight-Based Dose TID |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16
Secondary outcomes (3)
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
Measured: 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
Primary Analysis of Change from Baseline Plasma 7αC4 (Natural Log-Transformed) During the Two Double-Blind Periods
Measured: 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
Measured: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study start31 Jan 2020
- Primary completion13 Jul 2023
- Study completion4 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for CTXStudies of the same condition, open and under way.
- More trials run by Mirum Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- 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 blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04270682
- Sponsor's protocol ID
- Cheno-CTX-301
- Status last verified
- Oct 2024
- First submitted
- 12 Feb 2020
- Last updated
- 28 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis
- ClinicalTrials.gov ID
- NCT04270682
- Acronym / sponsor ID
- Cheno-CTX-301
- Conditions
- CTX
- Keywords
- Cerebrotendinous xanthomatosis, CTX, Cholestanol, Leukodystrophy, CYP27A1
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Jan 2020 (actual)
- Primary completion
- 13 Jul 2023 (actual)
- Study completion
- 4 Oct 2023 (actual)
- First posted
- 17 Feb 2020 (actual)
- Results
- Posted 28 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Adult Cohort | Experimental | Drug: Blinded CDCA 250 mg TID, Drug: Placebo, Drug: Open-Label CDCA 250 mg TID, Drug: Rescue Medication CDCA 250 mg TIDPatients 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 | Drug: CDCA Weight-Based Dose TIDPediatric 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. |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blinded CDCA 250 mg TIDAdult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. | Drug | Adult Cohort |
| PlaceboAdult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. | Drug | Adult Cohort |
| Open-Label CDCA 250 mg TIDAdult 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 | Adult Cohort |
| Rescue Medication CDCA 250 mg TIDCDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results. | Drug | Adult Cohort |
| CDCA Weight-Based Dose TIDPatients 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. | Drug | Pediatric Cohort |
Primary outcome
Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods
Time frame: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16
Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test
3 secondary outcomes
Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods
Time frame: 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
Time frame: 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
Time frame: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for CTX
Active, not recruitingPhase 2
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
Graft-versus-host Disease
Active, not recruitingPhase 2
Brentuximab Vedotin and Combination Chemotherapy in Treating Patients With CD30-Positive Peripheral T-cell Lymphoma
Adult T-Cell Leukemia/Lymphoma, Anaplastic Large Cell Lymphoma, ALK-Negative
RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
RecruitingPhase 1/2
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
RecruitingPhase 3
Adding an Immunotherapy Drug, MEDI4736 (Durvalumab), to the Usual Chemotherapy Treatment (Paclitaxel, Cyclophosphamide, and Doxorubicin) for Stage II-III Breast Cancer
Anatomic Stage II Breast Cancer AJCC v8, Anatomic Stage III Breast Cancer AJCC v8
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Blinded CDCA 250 mg TID, Open-Label CDCA 250 mg TID and Rescue Medication CDCA 250 mg TID in people with CTX, run by Mirum Pharmaceuticals, Inc. at 12 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods (time frame: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2024.