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

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ClinicalTrials.gov Identifier: NCT01438411
Recruitment Status : Completed
First Posted : September 22, 2011
Last Update Posted : August 16, 2016
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Bile Acid Synthesis Defect Drug: Cholic Acid Phase 3

Detailed Description:

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.


Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 53 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Single Center, Nonrandomized Continuation Study of Cholic Acid Capsules to Treat Subjects With Inborn Errors of Bile Acid Synthesis
Study Start Date : January 2010
Primary Completion Date : July 2016
Study Completion Date : July 2016


Arms and Interventions

Arm Intervention/treatment
Cholic Acid
Active drug
Drug: Cholic Acid
10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid Capsules
Other Names:
  • Cholic
  • Cholic Acid Capsules


Outcome Measures

Primary Outcome Measures :
  1. Change in evaluation of Atypical Bile Acid Metabolites via GC-MS, LC-MS and/or LSIMS analysis [ Time Frame: Comparison between baseline and follow-up visits (every month x 3; then every 3-6 months x 12 months and then an average of yearly visits) ]

Secondary Outcome Measures :
  1. Incidence and Severity of Adverse Events [ Time Frame: Comparison between baseline and follow-up visits (every month x 3; then every 3-6 months x 12 months and then an average of yearly visits) ]
  2. Clinical Laboratory Results [ Time Frame: Standard of Care (an average of every 6-12 months) ]
    Clinical evaluations as standard of care will be reviewed. These would also include Vitamins A, E, E and prothrombin time.

  3. Physical Examinations [ Time Frame: Standard of Care (an average of every 6-12 months) ]
    Physical examinations could include height and weight as well as assessment of vital signs.

  4. Evaluation of Serum Transaminases (AST and ALT) [ Time Frame: Comparison of baseline values and follow-up visits (Every month x 3; every 6-9 months for the next 12 and then yearly) ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01438411


Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Retrophin, Inc.
Investigators
Principal Investigator: James E Heubi, MD Children's Hospital Medical Center, Cincinnati
Principal Investigator: Kenneth Setchell, PhD Children's Hospital Medical Center, Cincinnati
More Information

Responsible Party: Retrophin, Inc.
ClinicalTrials.gov Identifier: NCT01438411     History of Changes
Other Study ID Numbers: CAC-002-01
First Posted: September 22, 2011    Key Record Dates
Last Update Posted: August 16, 2016
Last Verified: August 2016

Keywords provided by Retrophin, Inc.:
Cholic Acid
Inborn Error
Bile Acid Metabolism
Inborn Error of Bile Acid Synthesis

Additional relevant MeSH terms:
Bile Acids and Salts
Cholic Acids
Gastrointestinal Agents