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Bile Acid Synthesis Modulator
Open-Label CDCA 250 mg TID for Cerebral Thrombosis Xanthomatosis
Phase 3
Waitlist Available
Research Sponsored by Mirum Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 4 and week 16
Awards & highlights
Study Summary
This trial tests the effectiveness of an experimental drug for treating migraines in adults and children.
Eligible Conditions
- Cerebral Thrombosis Xanthomatosis
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ week 4 and week 16
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 4 and week 16
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in Bile Alcohols.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Pediatric CohortExperimental Treatment1 Intervention
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.
Group II: Adult CohortExperimental Treatment4 Interventions
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Blinded CDCA 250 mg TID
2020
Completed Phase 3
~20
Placebo
1995
Completed Phase 3
~2670
Open-Label CDCA 250 mg TID
2020
Completed Phase 3
~20
Rescue Medication CDCA 250 mg TID
2020
Completed Phase 3
~20
CDCA Weight-Based Dose TID
2020
Completed Phase 3
~20
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Who is running the clinical trial?
Mirum Pharmaceuticals, Inc.Lead Sponsor
29 Previous Clinical Trials
1,628 Total Patients Enrolled
Travere Therapeutics, Inc.Lead Sponsor
20 Previous Clinical Trials
177,618 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You are currently taking cholic acid medication.You have been diagnosed with heart failure in the past.You have a condition that affects how your body absorbs nutrients or an inflammatory condition in your digestive system.You are taking medications that interfere with the absorption of bile acids, like cholestyramine or antacids containing aluminum.
Research Study Groups:
This trial has the following groups:- Group 1: Adult Cohort
- Group 2: Pediatric Cohort
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What are the risks associated with taking Open-Label CDCA 250 mg TID?
"Open-Label CDCA 250 mg TID is considered safe, as it has progressed to Phase 3 clinical trials. This means that, although more research is needed to confirm efficacy, there is enough data supporting its safety from multiple rounds of testing."
Answered by AI
Are there other ongoing research projects involving Open-Label CDCA 250 mg TID?
"There is currently one active trial for Open-Label CDCA 250 mg TID in Phase 3. Most of the studies related to this topic are based in Porto Alegre, RS, but there are 8 locations running these sorts of studies."
Answered by AI
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