Compassionate Treatment of Patients With Inborn Errors of Bile Acid

Status Completed

Clinical Trial 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

Clinical Trial 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 Children's 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 center's screening/diagnostic program. Patients who were screened were contacted and evaluated with respect to the inclusion/exclusion criteria. Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian 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 and/or parents/legal 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 Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00007020
Study type	Interventional
Source	Mirum Pharmaceuticals, Inc.
Contact
Status	Completed
Phase	Phase 3
Start date	January 1992
Completion date	December 2009

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04510090` - Evaluate the Safety, Tolerability, and PK of EP547 in Healthy Subjects and Subjects With Cholestatic or Uremic Pruritus	Phase 1
Completed	`NCT02917408` - Retrospective Study About Primary Biliary Cholangitis During January 2001 to July 2016 at West China Hospital
Completed	`NCT00738101` - Compassionate Use of an Intravenous Fish Oil Emulsion in the Treatment of Liver Injury in Infants	N/A
Completed	`NCT00004414` - Sincalide (Cholecystokinin Octapeptide) Versus Placebo in Neonates at High Risk for Developing Parenteral Nutrition Associated Cholestasis	N/A
Completed	`NCT03662282` - Omegaven as Alternative Parenteral Fat Nutrition	Phase 3
Recruiting	`NCT02334293` - Omegaven® as Parenteral Nutrition	N/A
Recruiting	`NCT01252043` - Retrospective Review of CT and MR in Pediatric Patients With Cholestasis	N/A
Completed	`NCT00846963` - Ursodiol for Treating Parenteral Nutrition Associated Cholestasis in Neonates	Phase 2/Phase 3
Completed	`NCT00007033` - Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease	N/A
Completed	`NCT01194063` - Use of Omegaven Fish Oil Emulsion for Parenteral Nutrition Associated Liver Disease in Infants and Children	Phase 3
Recruiting	`NCT01998620` - Efficacy and Safety of S-adenosyl-L-methionine in Treatment of Chronic Hepatitis B Patients With Cholestasis	Phase 4
Completed	`NCT00080236` - Safety and Efficacy Study of a Caspase Inhibitor in Patients Undergoing Liver Transplantation	Phase 2
Completed	`NCT00058890` - Gabapentin to Treat Itch in Patients With Liver Disease	Phase 3
Active, not recruiting	`NCT00004315` - Phase II Pilot Study to Compare the Bioavailability of Buffered, Enteric-Coated Ursodiol With Unmodified Ursodiol for Chronic Cholestatic Liver Disease and Cystic Fibrosis-Associated Liver Disease	Phase 2
Active, not recruiting	`NCT02922751` - FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
Completed	`NCT04604652` - Open-Label Study of HTD1801 in Adult Subjects With Primary Biliary Cholangitis	Phase 2
Terminated	`NCT02267707` - Pharmacokinetic and Safety Study of Nab®-Paclitaxel (ABI-007) Plus Gemcitabine in Subjects With Advanced Pancreatic Cancer Who Have Cholestatic Hyperbilirubinemia	Phase 1
Completed	`NCT02357576` - Standard Lipid Therapy vs IVFE Minimization for Prevention of PNALD	Phase 3
Completed	`NCT02721277` - SMOFlipid to Lessen the Severity of Neonatal Cholestasis	Phase 1/Phase 2
Terminated	`NCT02767648` - Inter-regional Cohort of Neonatal and Infant Cholestasis in the Greater Southwest Region	N/A

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms