NCT01438411

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
53

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2010

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2010

Completed
1.7 years until next milestone

First Submitted

Initial submission to the registry

September 20, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 22, 2011

Completed
4.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
4.1 years until next milestone

Results Posted

Study results publicly available

August 21, 2020

Completed
Last Updated

October 3, 2023

Status Verified

September 1, 2023

Enrollment Period

6.5 years

First QC Date

September 20, 2011

Results QC Date

June 29, 2020

Last Update Submit

September 29, 2023

Conditions

Bile Acid Synthesis Defect

Keywords

Cholic AcidInborn ErrorBile Acid MetabolismInborn Error of Bile Acid Synthesis

Outcome Measures

Primary Outcomes (1)

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

    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 Outcomes (9)

  • Evaluation of Serum Transaminases: ALT

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

  • Evaluation of Serum Transaminases: AST

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

  • Clinical Laboratory Results: Bilirubin

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

  • Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)

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

  • Clinical Laboratory Results: Alkaline Phosphatase

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

  • +4 more secondary outcomes

Study Arms (1)

Cholic Acid

OTHER

Active drug

Drug: Cholic Acid

Interventions

Cholic AcidDRUG

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

Also known as: Cholic, Cholic Acid Capsules
Cholic Acid

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

  • Heubi JE, Setchell KDR. Open-label Phase 3 Continuation Study of Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis. J Pediatr Gastroenterol Nutr. 2020 Apr;70(4):423-429. doi: 10.1097/MPG.0000000000002618.

    PMID: 31899729DERIVED

MeSH Terms

Interventions

Cholic Acid

Intervention Hierarchy (Ancestors)

Cholic AcidsBile Acids and SaltsSteroidsFused-Ring CompoundsPolycyclic CompoundsCholanes

Results Point of Contact

Title
Retrophin Medical Information
Organization
Retrophin, Inc.
medinfo@retrophin.com
1-877-659

Study Officials

  • James E Heubi, MD

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

  • Kenneth Setchell, PhD

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 20, 2011

First Posted

September 22, 2011

Study Start

January 1, 2010

Primary Completion

July 1, 2016

Study Completion

July 1, 2016

Last Updated

October 3, 2023

Results First Posted

August 21, 2020

Record last verified: 2023-09

Locations

US(1)