Identify Predictors That Distinguish Between Tofacitinib Responders and Non-responders Based on Genotype and Cellular and Molecular Profiles From Pinch Biopsies, Blood and Stool Samples

NCT03663400 | Completed

• 1 other identifier: 18-00630
• Study Type: observational
• Target: 47
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective cohort study enrolling ulcerative colitis patients who initiate tofacitinib therapy. Investigators will collect clinical data, blood and stool samples prior to initiation of tofacitinib and, at minimum, monthly after the start of therapy. They will collect tissue from colonoscopies prior to initiation of therapy and within 6 months on therapy. Clinical characteristics and response to treatment will then be associated with genotype, blood immune profiles, stool microbiota, and cellular and molecular profiles of the biopsies to generate a treatment response model. Using predictors identified in our model, we will then attempt to validate the model and findings with the OCTAVE (Pfizer), SPARC (CCF), and RISK (CCF) data.

Conditions

Ulcerative Colitis

Outcome Measures

Primary Outcomes (1)

• GWAS analysis pretreatment

  GWAS analysis by Illumina BeadChip to generate comprehensive genotype data on all 50 patients.

  1 Day

Study Arms (1)

Patients Treated with Tofacitinib

Microbiota profiling by 16S sequencing RNA-seq transcriptional profiling of the blood and biopsy samples Immunological profiling by multi-parameter flow cytometry

Diagnostic Test: GWAS analysis by Illumina BeadChip

Interventions

GWAS analysis by Illumina BeadChip DIAGNOSTIC_TEST

will generate comprehensive genotype data for patients. Samples will be used for transcriptional profiling, microbiota profiling by 16S analysis and immunological profiling by flow cytometry

Patients Treated with Tofacitinib

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients 18-70 with moderate to severe ulcerative colitis defined as a total mayo score between 6 and 12, and an endoscopic subscore of 2 or 3.

You may qualify if:

• Total mayo score between 6 and 12
• Endoscopic subscore of 2 or 3

You may not qualify if:

• Recent use of antibiotic therapy (\<4 weeks)
• Current extreme diet (parenteral nutrition, specific carbohydrate diet).
• Active infection or malignancy.
• Significant underlying liver or renal disease.

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

New York University School of Medicine

New York, New York, 10016, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Microbiota profiling by 16S sequencing RNA seq transcriptional profiling oof blood and biopsy Immunological profiling by multi parameter flow cytometry

MeSH Terms

Conditions

Colitis, Ulcerative

Condition Hierarchy (Ancestors)

Colitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Inflammatory Bowel Diseases; Colonic Diseases; Intestinal Diseases

Study Officials

• David Hudesman, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 14, 2018
• First Posted: September 10, 2018
• Study Start: December 4, 2018
• Primary Completion: March 24, 2022
• Study Completion: March 24, 2022
• Last Updated: August 12, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Beginning 3 months and ending 5 years following article publication.
• Access Criteria: Researchers who provide a methodologically sound proposal.

Locations

US (1)