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

Digestive Care

Status and phase

Begins enrollment this month

Phase 2

Conditions

Steatorrhea

Treatments

Drug: Pertzye

Study type

Interventional

Funder types

Industry

Other

Identifiers

NCT07335523

102824-001

Details and patient eligibility

About

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

Enrollment

30 estimated patients

Sex

All

Ages

7 to 50 years old

Volunteers

No Healthy Volunteers

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.