Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
|Study Design:||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|
- 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 ]
- 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 ]
|Study Start Date:||January 2010|
|Estimated Study Completion Date:||June 2016|
|Estimated Primary Completion Date:||June 2016 (Final data collection date for primary outcome measure)|
Drug: Cholic Acid
10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid Capsules
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01438411
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||James E Heubi, MD||Children's Hospital Medical Center, Cincinnati|
|Principal Investigator:||Kenneth Setchell, PhD||Children's Hospital Medical Center, Cincinnati|