Ignite Creation Date:
2024-05-06 @ 4:01 AM
Last Modification Date:
2024-10-26 @ 11:42 AM
Study NCT ID:
NCT02430077
Status:
COMPLETED
Last Update Posted:
2023-12-13
First Post:
2015-04-24
Brief Title:
Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients
Sponsor:
Abhimanyu Garg
Organization:
University of Texas Southwestern Medical Center
Study Overview
Official Title:
Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients
Status:
COMPLETED
Status Verified Date:
2024-08
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:
Lipodystrophies are rare disorders characterized by selective loss of adipose tissue and predisposition to insulin resistance and its metabolic complications Hepatic steatosis is a common complication in patients with partial and generalized lipodystrophiesDespite aggressive management of diabetes and hyperlipidemia hepatic steatosis and its complications present a therapeutic challenge in many patients Due to this large disease burden it is important to assess the efficacy and safety of novel therapies for hepatic steatosis in patients with lipodystrophiesThere are however no systematic studies evaluating various therapeutic interventions for reducing hepatic steatosis in patients with lipodystrophies A variety of drugs have been investigated in nonlipodystrophic patients with non-alcoholic hepatic steatosis and steatohepatitis NASH or non-alcoholic fatty liver disease NAFLD Recent data support the activation of the farnesoid X receptor FXR NR1H4 a nuclear hormone receptor regulated by bile acids for treatment of NASH and NAFLD FXR activates transcription of several genes particularly the atypical nuclear receptor small heterodimer partner SHP NR0B2 and thus can influence triglyceride metabolism within hepatocytesBoth cholic acid CA and chenodeoxycholic acid CDCA are ligands for FXR however UDCA which is the 7 hydroxy β-epimer of CDCA does not activate FXR Obeticholic acid OCA is a first-in-class selective FXR agonist which has approximately 100 fold greater FXR-agonistic activity in the nanomolar range as compared to CDCA It therefore appears that FXR modulation offers interesting therapeutic possibilities in treating hepatic steatosis This study is primarily designed to study efficacy of OCA a strong FXR ligand in reducing hepatic triglyceride levels in patients with hepatic steatosis and Familial Partial Lipodystrophy FPLD If proven to be effective it may reduce morbidity and mortality as a result of sequelae of hepatic steatosis in patients with lipodystrophies
Detailed Description:
This study will be a randomized placebo-controlled cross-over trial Patients who are considered eligible for the study will undergo screening evaluation to determine their eligibility for the trial For those who are found to be eligible during the baseline period they will continue their usual diet and other lifestyle measures without changing any medications for 1 month in order to establish a baseline state Three blood samples will be obtained during this period at the Clinical and Translational Research Center Following the baseline period the patients will receive obeticholic acid OCA or an identical placebo in the dose of 25 mgday for a period of 4 months and then will receive the other treatment OCA or placebo for 4 months There will be a wash-out period of 4 months in-between the two study periods
Patients will be educated to maintain their usual physical activities and diet during the study The subjects will be admitted to the Clinical and Translational Research Center for the baseline evaluations at the beginning of the two study periods and at the end of four months during each study period
Patients will be educated to maintain their usual physical activities and diet during the study The subjects will be admitted to the Clinical and Translational Research Center for the baseline evaluations at the beginning of the two study periods and at the end of four months during each study period
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