- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01438411
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
An Open Label, Single Center, Nonrandomized Continuation Study of Cholic Acid Capsules to Treat Subjects With Inborn Errors of Bile Acid Synthesis
Study Overview
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 Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
-
-
Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: Cholic Acid
Active drug
|
10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid Capsules
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
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.
|
At baseline, then every 12 months for an average of 3.5 years
|
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.
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At baseline, then every 12 months for an average of 3.5 years
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Physical Examinations: Body Weight
Time Frame: At baseline, then every 12 months for an average of 3.5 years
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Changes in body weight percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.
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At baseline, then every 12 months for an average of 3.5 years
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Incidence of Adverse Events
Time Frame: At baseline, then every 12 months for an average of 3.5 years
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Number (%) of patients with any AE
|
At baseline, then every 12 months for an average of 3.5 years
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Kenneth Setchell, PhD, Children's Hospital Medical Center, Cincinnati
- Principal Investigator: James E Heubi, MD, Children's Hospital Medical Center, Cincinnati
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CAC-002-01
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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