Primary Sclerosing Cholangitis in Children

Recruiting at 9 trial locations

Overseen By Melissa Sexton, BBA, CCRP

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Arbor Research Collaborative for Health

Disqualifiers: Liver transplant, Immunodeficiency, Cystic fibrosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

1000 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to learn more about primary sclerosing cholangitis (PSC), a rare liver disease that damages bile ducts in children. Researchers seek to understand PSC's progression, its impact on daily life, and possible causes. Children and young adults with a confirmed PSC diagnosis, who have not undergone a liver transplant or have certain other medical conditions, may qualify. Participants will provide medical information and samples to aid scientists in further exploring PSC. As an unphased trial, this study allows participants to contribute to groundbreaking research that could lead to better understanding and future treatments for PSC.

Why are researchers excited about this trial?

Researchers are excited about new treatments for Primary Sclerosing Cholangitis (PSC) in children because current options mainly focus on managing symptoms rather than addressing the root cause of the disease. Unlike standard treatments like ursodeoxycholic acid, which primarily help with bile flow and liver function, these new investigational treatments aim to target the underlying inflammation and immune response that drive PSC. By addressing these root causes, the new treatments hold promise for halting disease progression and offering a more effective solution for young patients.

Who Is on the Research Team?

Katrina Loh, MD

Principal Investigator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Lisa Henn, PhD

Principal Investigator

Arbor Research Collaborative for Health

Cara Mack, MD

Principal Investigator

Medical College of Wisconsin-Milwaukee

John Magee, MD

Principal Investigator

University of Michigan

Ed Doo, MD

Principal Investigator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Are You a Good Fit for This Trial?

Inclusion Criteria

Patients with the clinical diagnosis of large or small duct PSC made at any time prior to enrollment are screened for eligibility to participate in this prospective cohort study. The site PI will determine eligibility following review of MRCP or ERCP images with the site radiologist to confirm presence of an abnormal cholangiogram at the time of diagnosis of large duct PSC. Liver histopathology obtained at the time of diagnosis of small duct PSC will be reviewed with the site pathologist prior to enrollment.

Aged 2 through 25 years at time of screening.

Focal structuring of the bile duct(s)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Assessment

Participants undergo liver stiffness measurement (LSM) and may have a research MRI if eligible

1 visit

1 visit (in-person)

Longitudinal Observation

Annual assessments including liver stiffness measurement (LSM), collection of biological samples, and administration of questionnaires

up to 10 years

Annual visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after the main observational period

1 year

What Are the Treatments Tested in This Trial?

Who Is Running the Clinical Trial?

Arbor Research Collaborative for Health

Lead Sponsor

Trials

21

Recruited

35,500+

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborator

Trials

2,513

Recruited

4,366,000+