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Completed

NCT Number: NCT01438411

Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

The primary purpose of the study is to evaluate the therapeutic efficacy and safety of cholic acid in subjects with identified inborn errors of bile acid synthesis.

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Key information

Conditions

Bile Acid Synthesis Defect

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

This is a Phase 3, open-label, single center, nonrandomized study. This continuation protocol will consist of eligible subjects who have previously received cholic acid through the Cincinnati Children's Hospital Medical Center (CCHMC) Compassionate Use (91-10-10), CAC-001-01 study protocols and newly diagnosed subjects.

New subjects will be infants, children, adolescents identified from urine samples obtained from the clinical services of programs across the U.S., Canada, South America, Europe, and Asia. Subject or their legal representative will receive information regarding the study, and the principle investigator (PI) or designee will obtain informed consent. Serum and urine samples will be collected and sent to CCHMC to measure complete bile acid profile analysis. Clinical records including medical history, physical exams, vital signs, and laboratory assessments performed as standard of care will be reviewed to ensure subject eligibility and determine baseline values.

Subjects who have participated in Protocols conducted under IND 45,470 will be consented to continue to receive cholic acid capsules under this continuation protocol. Subjects will serve as their own controls and no placebo will be utilized.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

Subjects who received cholic acid through CCHMC protocols 91-10-10 or CAC-002-01 and meet the following criteria are eligible for study participation.

  • The subject and/or parent/legal guardian must have provided informed consent prior to study start.
  • The subject must have a diagnosis of an inborn error of bile acid synthesis.
  • The subject must be willing and able to comply with all study assessments and procedures.
  • Subjects with other organ dysfunction will not be excluded

Treatment and study plan

Cholic Acid

Drug

10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid Capsules

Other names: Cholic, Cholic Acid Capsules

Primary outcomes

  1. Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.

Secondary outcomes

  1. Evaluation of Serum Transaminases: ALT

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes in ALT were evaluated in terms of elevations above the upper limit of normal (ULN) and were categorized as: <ULN; ≥1 ULN but <2 ULN; ≥2 ULN but <3 ULN; and ≥3x ULN. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented.

  2. Evaluation of Serum Transaminases: AST

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes in AST were evaluated in terms of elevations above the upper limit of normal (ULN) and were categorized as: <ULN; ≥1 ULN but <2 ULN; ≥2 ULN but <3 ULN; and ≥3x ULN. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented.

  3. Clinical Laboratory Results: Bilirubin

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for bilirubin. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.

  4. Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for GGT. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.

  5. Clinical Laboratory Results: Alkaline Phosphatase

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for alkaline phosphatase. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.

  6. Clinical Laboratory Results: Prothrombin Time

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for prothrombin time. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.

  7. Physical Examinations: Height

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes in height percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.

  8. Physical Examinations: Body Weight

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Changes in body weight percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.

  9. Incidence of Adverse Events

    Time frame: At baseline, then every 12 months for an average of 3.5 years

    Number (%) of patients with any AE

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study title

An Open Label, Single Center, Nonrandomized Continuation Study of Cholic Acid Capsules to Treat Subjects With Inborn Errors of Bile Acid Synthesis

Important dates

Study start
2010
Primary completion
2016
Study completion
2016
First posted
Sep 22, 2011
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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