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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2013 by Asklepion Pharmaceuticals, LLC
Sponsor:
Information provided by (Responsible Party):
Asklepion Pharmaceuticals, LLC
ClinicalTrials.gov Identifier:
NCT01438411
First received: September 20, 2011
Last updated: May 21, 2013
Last verified: May 2013
  Purpose

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 Intervention Phase
Bile Acid Synthesis Defect
Drug: Cholic Acid
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Further study details as provided by Asklepion Pharmaceuticals, LLC:

Primary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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) ] [ Designated as safety issue: Yes ]
  • Clinical Laboratory Results [ Time Frame: Standard of Care (an average of every 6-12 months) ] [ Designated as safety issue: Yes ]
    Clinical evaluations as standard of care will be reviewed. These would also include Vitamins A, E, E and prothrombin time.

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

  • 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) ] [ Designated as safety issue: No ]

Estimated Enrollment: 45
Study Start Date: January 2010
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01438411

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Donna Buckley, M.Ed    513-636-8549    donna.buckley@cchmc.org   
Principal Investigator: James E Heubi, MD         
Principal Investigator: Kenneth Setchell, PhD         
Sponsors and Collaborators
Asklepion Pharmaceuticals, LLC
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

No publications provided

Responsible Party: Asklepion Pharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT01438411     History of Changes
Other Study ID Numbers: CAC-002-01
Study First Received: September 20, 2011
Last Updated: May 21, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Asklepion Pharmaceuticals, LLC:
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
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 31, 2014