Management of Steatorrhea in Children and Young Adults With Alagille Syndrome After Liver Transplantation

NCT ID: NCT07335523

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-03-31
• Study Completion Date: 2028-03-31

Brief Summary

The goal of this clinical trial is to learn if PERTZYE® works to treat steatorrhea in children and adults with Alagille Syndrome (ALGS) post liver transplant. It will also learn about the safety of PERTZYE®. The main questions it aims to answer are:

• Does PERTZYE improve dietary fat absorption?
• Does PERTZYE improve stool frequency and consistency?
• Does PERTZYE improve abdominal signs and symptoms?
• What medical problems do participants have when taking PERTZYE? Researchers will compare the results when participants are taking and stop taking PERTZYE.

During the study, participants will:

• Consume a high fat diet
• Take PERTZYE every day with meals and snacks for up to 4 weeks
• Have scheduled checkups by phone or video by clinical staff
• Keep a diary of their symptoms and PERTZYE dosing
• Collect stool for laboratory testing

Conditions

Steatorrhea

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PERTZYE

PERTZYE (pancrelipase) delayed-release capsules, for oral administration

Group Type: EXPERIMENTAL

Pertzye

Intervention Type: DRUG

PERTZYE (pancrelipase) delayed-release capsules

Interventions

Pertzye

PERTZYE (pancrelipase) delayed-release capsules

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male and female participants ages 7-50 years inclusive.
• Confirmed diagnosis of ALGS
• Status post OLT due to ALGS complications.
• Participants currently managed with pancreatic enzyme products (PEPs) are allowed pending discontinuation of PEPs for 5 days prior to beginning the 72-hour stool collections. Participants must discontinue PEPs for the entire study period until completion.
• Agree to adhere to a high fat diet at least 2 days prior to and during the 72-hour stool collections and monitor dietary consumption through the use of a diet diary.
• Presence of steatorrhea confirmed by baseline CFA ≤ 90%.
• Have a consistent caregiver for the duration of the study if applicable for enrollment of a child.
• Capable of giving written informed consent and assent (if age appropriate).

Exclusion Criteria

• Surgical disruption of the enterohepatic circulation (e.g., biliary diversion).
• Diets high in medium-chain triglyceride (MCT) oil.
• Use of bile acids, bile acid uptake inhibitors, lipid-binding resins, synthetic fat substitutes (e.g. Olestra), and diaper rash ointments (note that H2 antagonists and proton pump inhibitors [PPIs] are permitted if on a stable and consistent regimen throughout the duration of the study).
• History or presence of other concomitant liver diseases such as liver transplant rejection with cholestasis, hepatitis B or C virus infection, HIV, primary sclerosing cholangitis (PSC), alcoholic liver disease, autoimmune hepatitis, metabolic-associated fatty liver disease (MASLD)/non-alcoholic steatohepatitis (NASH) or significant chronic allograft rejection.
• Currently pregnant or breastfeeding.
• Cancers except for in situ carcinoma or cancers treated at least 5 years prior to screening with no evidence of recurrence.
• Participation in a clinical trial and received an investigational product within the last 30 days.
• Use of anti-diarrheal, anti-spasmodic, and/or cathartic laxatives. Change in osmotic laxatives within the past 3 months.

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alagille Syndrome Alliance

UNKNOWN

Sponsor Role: collaborator

Digestive Care, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Digestive Care Customer Service

Role: CONTACT

customerservice@pertzye.com

877-882-5950

Other Identifiers

102824-001

Identifier Type: -

Identifier Source: org_study_id