Ignite Creation Date:
2024-05-05 @ 11:20 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00007020
Status:
COMPLETED
Last Update Posted:
2023-10-03
First Post:
2000-12-06
Brief Title:
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Sponsor:
Mirum Pharmaceuticals Inc
Organization:
Mirum Pharmaceuticals Inc
Study Overview
Official Title:
Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism
Status:
COMPLETED
Status Verified Date:
2023-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
OBJECTIVES
I To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism
II To assess the safety and tolerability of cholic acid
I To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism
II To assess the safety and tolerability of cholic acid
Detailed Description:
Investigational Plan
A Phase III open label single arm nonrandomized non-comparative compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism
The study was begun with a single study site at Cincinnati Childrens Hospital Medical Center CCHMC but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the centers screeningdiagnostic program
Patients who were screened were contacted and evaluated with respect to the inclusionexclusion criteria Signed informed consent by the patient andor parentslegal guardian was obtained as soon as it is confirmed that the patient met inclusionexclusion criteria and the parentsguardian would agree for the child to participate in the study
The primary interventions for the study were
1 Administration of study drug
2 Collection of baseline physical exam vital signs blood and urine samples for laboratory tests
3 Collection of periodic physical exam vital signs blood and urine samples for laboratory tests during the period of administration of the study drug
4 Collection of any adverse event information
Time and Events Schedule
Baseline
1 Confirm eligibility
2 Obtain written informed consent from patient andor parentslegal guardian
3 Collect demographic data and disease and medication history including family history
Baseline and Ongoing
4 Obtain body weight
5 Record adverse events
6 Obtain blood and urine samples for laboratory tests
7 Initiate study drug therapy monitor study drug therapy and adjust dose as needed
A Phase III open label single arm nonrandomized non-comparative compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism
The study was begun with a single study site at Cincinnati Childrens Hospital Medical Center CCHMC but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the centers screeningdiagnostic program
Patients who were screened were contacted and evaluated with respect to the inclusionexclusion criteria Signed informed consent by the patient andor parentslegal guardian was obtained as soon as it is confirmed that the patient met inclusionexclusion criteria and the parentsguardian would agree for the child to participate in the study
The primary interventions for the study were
1 Administration of study drug
2 Collection of baseline physical exam vital signs blood and urine samples for laboratory tests
3 Collection of periodic physical exam vital signs blood and urine samples for laboratory tests during the period of administration of the study drug
4 Collection of any adverse event information
Time and Events Schedule
Baseline
1 Confirm eligibility
2 Obtain written informed consent from patient andor parentslegal guardian
3 Collect demographic data and disease and medication history including family history
Baseline and Ongoing
4 Obtain body weight
5 Record adverse events
6 Obtain blood and urine samples for laboratory tests
7 Initiate study drug therapy monitor study drug therapy and adjust dose as needed
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| NCRR-M01RR08084-0009 | OTHER | National Center For Research Resources NCRR | None |
| CCHMC-91-10-10 | OTHER | None | None |