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NCT Number: NCT01115582

Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis

This is a study in a small population of children who have inborn errors of bile acid synthesis who are currently taking established doses of the currently used cholic acid capsules prepared at the Cincinnati Children's Hospital Pharmacy. The study is designed to compare the efficacy of these currently used capsules with the efficacy of the same treatment provided in a cholic acid capsule that is made by a company that will be marketed after FDA approval.

At baseline, patients receive established doses of cholic acid capsules prepared at the Cincinnati Children's Hospital Medical Center Pharmacy. During the study, patients receive the same treatment provided in the to-be-marketed (TBM) cholic acid capsule. Hence, patients serve as their own controls, with baseline values presenting the reference value (CCHMC cholic acid capsule) and values after 30 days treatment presenting the value for the investigational treatment (TBM cholic acid capsule).

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Key information

Conditions

Inborn Errors of Bile Acid Synthesis Digestive System Diseases Liver Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

About this study

Bile acids are end products of cholesterol metabolism. Individuals with inborn errors of bile acid synthesis lack the enzymes needed to synthesize the primary bile acids cholic acid and chenodeoxycholic acid (CDCA). These conditions are serious and account for approximately 1% of cases presenting as idiopathic cholestatic liver disease. The liver disease associated with these inborn errors in bile acid synthesis is progressive and, if untreated, may lead to death from cirrhosis and liver failure.

Monotherapy with cholic acid is considered the most appropriate therapeutic strategy to treat inborn errors in bile acid synthesis because it provides a stimulus for bile flow and inhibits endogenous production and accumulation of potentially hepatotoxic and cholestatic bile acid precursors, while additionally facilitating the absorption of fats and fat-soluble vitamins. At therapeutic doses, adverse effects are not generally observed and as such, cholic acid has become the treatment of choice at the Cincinnati Children's Hospital since 1994.

This study will bridge data on the effectiveness of a standardized manufactured preparation to data obtained from patients originally treated with the currently used cholic acid capsules formulated in the CCHMC Pharmacy before being switched to the manufactured preparation.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • must have stable transaminase levels within 2 times the upper limits of the normal range.
  • must have a diagnosis of an inborn error of bile acid synthesis.
  • must have signed the written informed consent/assent document before study start.
  • must be currently receiving currently used cholic acid therapy under IND 45,470.
  • must be willing and able to comply with all study assessments and procedures.
  • must be able to make two visits (Visit 1 and Visit 2) to the study site.

Exclusion criteria

  • is not currently receiving cholic acid therapy for inborn errors of bile acid synthesis under IND 45,470.
  • is unable or unwilling to comply with study requirements.

Treatment and study plan

Cholic Acid

Drug

The IUPAC name for cholic acid is 3 alpha,7alpha,12 alpha-trihydroxy-5 beta-cholanoic acid. The international nonproprietary name (INN) is cholic acid.

Each patient will be given a box containing a 1 month supply of study drug. Each bottle will contain 90 capsules; each capsule will contain either 50 or 250 mg of manufactured cholic acid depending upon the child's weight. The study drug will be taken orally, in divided doses (as determined by the investigator), for a total daily dose of 10-15 mg/kg body weight. Parents of infants and young children who are unable to swallow the TBM cholic acid capsule will be instructed to sprinkle the contents of the capsule over 1-2 teaspoons of plain applesauce and feed it to the child.

Primary outcomes

  1. Serum Transaminases

    Time frame: At baseline and after 30 days of treatment

    Concentration of serum alanine transaminase (ALT) and aspartate transaminase (AST)

  2. Serum and Urine Bile Acids

    Time frame: At baseline (BL) and after 30 days of treatment (D30)

    Concentration of bile acids in serum (S) and urine (U). (abbreviations: chol.=cholenoic; monohydro=monohydroxy; dihydro=monohydro)

Secondary outcomes

  1. Adverse Events

    Time frame: Total of 30 days, i.e. from the time point the patients entered into the study up to the end of treatment

    Total number of patients with any adverse events

  2. Blood Pressure

    Time frame: At baseline and after 30 days of treatment

    Systolic blood pressure (SBP) and diastolic blood pressure (DBP)

  3. Physical Examination

    Time frame: At baseline (BL) and after 30 days of treatment (D30)

    Total number of patients with abnormal findings from general physical examination

  4. Total Bilirubin

    Time frame: At baseline and after 30 days of treatment

    Concentration of total bilirubin in serum

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Open Label, Single Center, Nonrandomized Study Comparing Efficacy of To Be Marketed Cholic Acid With That of the Currently Used Formulation of Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis

Important dates

Study start
2010
Primary completion
2010
Study completion
2010
First posted
May 4, 2010
Registry last updated
Oct 3, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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