Ignite Creation Date:
2024-05-06 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 1:52 PM
Study NCT ID:
NCT04685200
Status:
RECRUITING
Last Update Posted:
2024-07-01
First Post:
2020-12-24
Brief Title:
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary Integrative Research Approach
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary Integrative Research Approach
Status:
RECRUITING
Status Verified Date:
2024-09-17
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:
Background
Primary sclerosing cholangitis is a rare chronic liver disease It affects the bile ducts of the
liver It can result in bile duct infections cirrhosis cancer and end stage liver disease Researchers want to learn more about this disease
Objective
To understand the biological causes of primary sclerosing cholangitis
Eligibility
Adults age 18 and older who have primary sclerosing cholangitis
Design
Participants will be screened with a medical history physical exam and blood tests
Participants will give blood saliva urine and stool samples They will have nasal swabs They will complete surveys
Participants will get an intravenous IV catheter A plastic tube is inserted into an arm vein
Participants will have a colonoscopy A tube with a video camera at the end is inserted into the rectum
Participants will have an upper endoscopy A scope with a light and camera at its tip is used to look inside the upper digestive tract
Participants will have a liver biopsy entering through the chest wall or a neck vein Blood is drawn from a blood vessel that carries blood to the liver A liver tissue sample is taken
Participants will have magnetic resonance imaging or spectroscopy They will get a contrast agent through an IV
Participants may have an optional bone marrow aspiration A large needle is inserted into the hip to withdraw marrow
Participants will have a liver ultrasound
Participants will complete a 3-day food diary They will have a nutrition assessment
Participants may give contact details for people who live with them to also take part in this study
Participation will last for 12 months
Primary sclerosing cholangitis is a rare chronic liver disease It affects the bile ducts of the
liver It can result in bile duct infections cirrhosis cancer and end stage liver disease Researchers want to learn more about this disease
Objective
To understand the biological causes of primary sclerosing cholangitis
Eligibility
Adults age 18 and older who have primary sclerosing cholangitis
Design
Participants will be screened with a medical history physical exam and blood tests
Participants will give blood saliva urine and stool samples They will have nasal swabs They will complete surveys
Participants will get an intravenous IV catheter A plastic tube is inserted into an arm vein
Participants will have a colonoscopy A tube with a video camera at the end is inserted into the rectum
Participants will have an upper endoscopy A scope with a light and camera at its tip is used to look inside the upper digestive tract
Participants will have a liver biopsy entering through the chest wall or a neck vein Blood is drawn from a blood vessel that carries blood to the liver A liver tissue sample is taken
Participants will have magnetic resonance imaging or spectroscopy They will get a contrast agent through an IV
Participants may have an optional bone marrow aspiration A large needle is inserted into the hip to withdraw marrow
Participants will have a liver ultrasound
Participants will complete a 3-day food diary They will have a nutrition assessment
Participants may give contact details for people who live with them to also take part in this study
Participation will last for 12 months
Detailed Description:
Study Description
We hypothesize that primary sclerosing cholangitis PSC develops as a consequence of a genetically driven aberrant immune response to commensal or pathogenic bacteria and that unique genetic-immunemicrobial associations may underlie development of distinct disease patterns We intend to conduct a thorough radiologic endoscopic histologic and microbiological investigation of patients with PSC in order to determine potential associations
Objectives
Primary Objective
The ultimate goal of this study is to generate understanding of how factors driving pathogenesis in PSC interact by capturing and integrating collated datasets from across relevant biologic systems and interpreting those in the context of phenotypic presentation in one exceptionally well-characterized set of patients
Secondary Objectives
1 To collect comprehensive data on distinct patterns of disease expression through single cell sequencing and microRNA and transcriptome profiling
2 To conduct extensive phenotypic characterization of cellular and humoral immune responses as well as microbiome signatures at multiple anatomic sites
3 To evaluate metabolic signatures or biomarkers for PSC diagnosis and prognostication
4 To generate a humanized mouse model of each subject s disease by obtaining bone marrow aspirate
Endpoints
Primary Endpoint
1 Identification of immune signatures at multiple levels and from different anatomical sites and tissues
2 Characterization of microbiome signatures taxonomic and functional as well as identification of specific species
3 Identification of metabolomic signatures from different anatomical sites and tissues as well as identification of different biomarkers correlating with disease progression
4 MicroRNA profiling of portal and systemic blood
We hypothesize that primary sclerosing cholangitis PSC develops as a consequence of a genetically driven aberrant immune response to commensal or pathogenic bacteria and that unique genetic-immunemicrobial associations may underlie development of distinct disease patterns We intend to conduct a thorough radiologic endoscopic histologic and microbiological investigation of patients with PSC in order to determine potential associations
Objectives
Primary Objective
The ultimate goal of this study is to generate understanding of how factors driving pathogenesis in PSC interact by capturing and integrating collated datasets from across relevant biologic systems and interpreting those in the context of phenotypic presentation in one exceptionally well-characterized set of patients
Secondary Objectives
1 To collect comprehensive data on distinct patterns of disease expression through single cell sequencing and microRNA and transcriptome profiling
2 To conduct extensive phenotypic characterization of cellular and humoral immune responses as well as microbiome signatures at multiple anatomic sites
3 To evaluate metabolic signatures or biomarkers for PSC diagnosis and prognostication
4 To generate a humanized mouse model of each subject s disease by obtaining bone marrow aspirate
Endpoints
Primary Endpoint
1 Identification of immune signatures at multiple levels and from different anatomical sites and tissues
2 Characterization of microbiome signatures taxonomic and functional as well as identification of specific species
3 Identification of metabolomic signatures from different anatomical sites and tissues as well as identification of different biomarkers correlating with disease progression
4 MicroRNA profiling of portal and systemic blood
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
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 |
|---|---|---|---|
| 000130-DK | None | None | None |