Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Retrophin, Inc.
ClinicalTrials.gov Identifier:
NCT01115582
First received: January 19, 2010
Last updated: October 17, 2016
Last verified: July 2016
Results First Received: July 20, 2016  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Inborn Errors of Bile Acid Synthesis
Intervention: Drug: Cholic acid

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
In this single-arm study, patients served as their own controls: Reference was presented by the baseline value, when patients received cholic acid capsules prepared by the Cincinnati Children's Hospital Medical Center; Investigational Treatment was the to-be-marketed (TBM) cholic acid capsule administered to patients for 30 days treatment duration.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The study was performed in patients with inborn defects of bile acid synthesis currently receiving cholic acid capsules prepared by the Cincinnati Children’s Hospital Medical Center (CCHMC) under IND 45,470. The study planned to include 25 patients; but only 16 patients fulfilled eligibility criteria and were willing to travel to the CCHMC.

Reporting Groups
  Description
Cholic Acid All patients entered and treated

Participant Flow:   Overall Study
    Cholic Acid
STARTED   16 
COMPLETED   16 
NOT COMPLETED   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Cholic Acid All patients entered and treated

Baseline Measures
   Cholic Acid 
Overall Participants Analyzed 
[Units: Participants]
 16 
Age 
[Units: Years]
Mean (Standard Deviation)
 7.8  (4.6) 
Gender 
[Units: Participants]
 
Female   5 
Male   11 
Region of Enrollment 
[Units: Participants]
 
United States   16 


  Outcome Measures
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1.  Primary:   Serum Transaminases   [ Time Frame: At baseline and after 30 days of treatment ]

2.  Primary:   Serum and Urine Bile Acids   [ Time Frame: At baseline (BL) and after 30 days of treatment (D30) ]

3.  Secondary:   Adverse Events   [ Time Frame: Total of 30 days, i.e. from the time point the patients entered into the study up to the end of treatment ]

4.  Secondary:   Blood Pressure   [ Time Frame: At baseline and after 30 days of treatment ]

5.  Secondary:   Physical Examination   [ Time Frame: At baseline (BL) and after 30 days of treatment (D30) ]

6.  Secondary:   Total Bilirubin   [ Time Frame: At baseline and after 30 days of treatment ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Retrophin Medical Information
Organization: Retrophin, Inc.
phone: 1-877-659 ext 5518
e-mail: medinfo@retrophin.com


Publications:
Jacquemin E., Gerhardt M, Cresteil D, Fabre M, Taburet AM, Hadchouel M, Trivin F, Stechell KDR and Bernard O. Long-term effects of bile acid therapy in children with defects of primary bile acid synthesis: 3ß-Hydroxy-C27-steroid dehydrogenase/isomerase and Delta4-3-Oxosteroid 5ß-reductase deficiencies. In: van Berge Henegouwen GP, et al. (eds): Falk Symposium No 120: Biology of Bile Acids in Health and Disease. Kluwer Academic Publishers, Dordrecht/Boston/London; 2001:278-282.
Setchell KDR, et al. A unique case of cerebrotendinous xantomatosis presenting in infancy with cholestatic liver disease further highlights bile acid synthetic defects as an important category of metabolic liver disease. In: van Berge Henegouwen GP et al. (ed): Falk Symposium No. 120: Biology of Bile Acids in Health and Disease. Boston: Kluwer Academic Publishers; 2001.


Responsible Party: Retrophin, Inc.
ClinicalTrials.gov Identifier: NCT01115582     History of Changes
Other Study ID Numbers: CAC-001-01
Study First Received: January 19, 2010
Results First Received: July 20, 2016
Last Updated: October 17, 2016