Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis

NCT05719311 | Completed Phase 2 Results FDA Drug

• 1 other identifier: FWB-CF-2.03
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 3

Brief Summary

Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. The goals of this clinical study are to evaluate the safety and efficacy of a novel formulation of a non-porcine lipase, called adrulipase, in patients with EPI due to cystic fibrosis. The main question\[s\] the study aims to answer are:

1. 1.Is the novel formulation of adrulipase safe to use at the doses being evaluated in the clinical study.
2. 2.Is adrulipase as effective, or more effective, compared to the pig enzymes the patients currently use.

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Outcome Measures

Primary Outcomes (2)

• Safety of Adrulipase

  Number of subjects reporting 1 or more adverse events.

  End of 3-week treatment period.

• Efficacy of Adrulipase: Coefficient of Fat Absorption (CFA)

  The primary efficacy endpoint is the CFA that will be assessed at the end of the 3-week treatment period. CFAs for adrulipase will be compared to the CFAs of PERT obtained at baseline/eligibility using descriptive methods.

  End of 3-week treatment period.

Secondary Outcomes (2)

• Stool Weight

  Change between two time points: initial PERT confinement and end of 3-week treatment period.

• Coefficient of Nitrogen Absorption (CNA)

  Change between two time points: initial PERT confinement and end of 3-week treatment period

Study Arms (1)

Adrulipase

EXPERIMENTAL

Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase.

Drug: adrulipase

Interventions

adrulipase DRUG

Enteric microgranule formulation of adrulipase.

Also known as: MS1819

Adrulipase

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride \>60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype.
• On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range.
• CFA = or \> 80% at screening while on stable PERT
• A fair or better nutritional status as defined by:
• BMI ≥16.0 kg/m2 for female patients ≥18 years of age, or
• BMI ≥16.5 kg/m2 for male patients ≥18 years of age
• Fecal elastase \<100 µg/g of stool at screening
• Standard-of-Care medications including CFTR modulators are allowed

You may not qualify if:

• History or diagnosis of fibrosing colonopathy
• Any chronic diarrheal illness unrelated to pancreatic insufficiency
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level
• ×upper limit of normal (ULN), or total bilirubin level ≥1.5 ×ULN at Screening
• Feeding via an enteral tube during 6 months before screening
• Forced expiratory volume ≤30% at the Screening visit
• Changes in gastric acid suppressant therapy during the one month prior to screening for patients already on suppressant therapy.

Sponsors & Collaborators

• Entero Therapeutics: lead

Study Sites (3)

Central Florida Pulmonary Group

Orlando, Florida, 32803, United States

Location

The Cystic Fibrosis Institute

Northfield, Illinois, 60093, United States

Location

Childrens Lung Specialists

Las Vegas, Nevada, 89109, United States

Location

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: VP, Regulatory, QA & Compliance
• Organization: Entero Therapeutics

AChandler@Enterothera.com

9194491484

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2023
• First Posted: February 8, 2023
• Study Start: February 1, 2023
• Primary Completion: June 20, 2023
• Study Completion: July 28, 2023
• Last Updated: September 19, 2024
• Results First Posted: September 19, 2024

Record last verified: 2023-07

Locations

US (3)