Recent Updates
Recently added Catalysts

Cholic Acids

Phase 3

Infantile Refsum's Disease | Small molecule | Rare Disease |Mirum Pharmaceuticals, Inc.|Last Updated: Oct 3, 2023

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment85

FDA Designations

No designations recorded

Clinical trial landscape

Cholic Acids · 1 trial · 5 indications

Phase 3 1
NCT00007020Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidInfantile Refsum's Disease
COMPLETED85 Analytics
PHASE3COMPLETED
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Infantile Refsum's Disease
NCT0000702085
Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years

Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)

Secondary Endpoints

Change in Liver Function Tests (LFTs) Measured in Serum
Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
Liver Histology
At baseline (if no historical data were available) and between 1 and 6 months following treatment start.
Height and Weight
Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cholic AcidOTHER -

Interventions

NameTypeDescription
Cholic AcidsDRUG10-15 mg/kg body weight/day taken orally.
Unlock Study Design Details

Eligibility Criteria

SexALL
Healthy VolunteersNo
Study Sites1

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Clinical or biochemical evidence of liver disease, unexplained fat-soluble vitamin malabsorption, or peroxisomal dysfunction that compromises bile acid biosynthesis Inclusion criteria for enrollment were: * Infants \< age 3 months * Children p...

Countries:United States
Unlock Eligibility Criteria

Frequently asked questions about Cholic Acids

What is Cholic Acids used for?

Cholic Acids is an investigational small molecule being developed for Infantile Refsum's Disease, a rare inherited disorder. It is also being studied in related conditions including Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, and Cholestasis. The drug is currently in Phase 3 clinical development.

Who makes Cholic Acids?

Cholic Acids is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol MIRM. The company is focused on rare disease therapies and is advancing Cholic Acids through clinical trials for Infantile Refsum's Disease.

What phase is Cholic Acids in?

Cholic Acids is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The drug is being studied for Infantile Refsum's Disease and related peroxisomal disorders, with one completed Phase 3 trial supporting its development.

What clinical trials is Cholic Acids in?

Cholic Acids has one completed clinical trial, NCT00007020, titled "Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid." This Phase 3 study enrolled 85 participants with conditions including Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, and Cholestasis.

Is Cholic Acids an approved drug?

Cholic Acids is an investigational drug and is not FDA approved. It is currently in Phase 3 clinical development for Infantile Refsum's Disease. The drug has completed one clinical trial, but approval status has not been established.