Safety and preliminary clinical activity of a novel pancreatic enzyme preparation in pancreatic insufficient cystic fibrosis patients.

Abstract:

OBJECTIVES:Currently available pancreatic enzyme products are crude porcine products with few data available regarding their efficacy, safety, and manufacture. We conducted a phase 1 study of a novel pancreatic enzyme product, TheraCLEC-Total (TCT), a proprietary formulation of microbial-derived lipase, protease, and amylase, to determine its safety and preliminary efficacy in cystic fibrosis. METHODS:We conducted an open-label, dose-ranging study in 23 subjects diagnosed with pancreatic insufficiency with cystic fibrosis. The subjects received TCT containing lipase dose of 100, 500, 1000, 2500, or 5000 USP U/kg per meal with each meal or snack for 3 days. The clinical and laboratory parameters and adverse events (AEs) were monitored. RESULTS:There were no serious AEs. Most AEs were mild, although gastrointestinal complaints were common. TCT increased the coefficient of fat and nitrogen absorption in all groups except in the low-dose group. At the other dosing levels, the mean coefficient of fat and nitrogen absorption increases were 19.1% +/- 24.9% and 17.8% +/- 13.6%, respectively, whereas the mean stool weight decreased by 517 +/- 362 g. CONCLUSIONS:TCT was well tolerated in this short-term exposure study. The preliminary efficacy data demonstrate lipase and protease activity with little difference seen with lipase doses greater than 500 USP U/kg per meal. These data support a larger randomized phase 2 trial.

journal_name

Pancreas

journal_title

Pancreas

authors

Borowitz D,Goss CH,Stevens C,Hayes D,Newman L,O'Rourke A,Konstan MW,Wagener J,Moss R,Hendeles L,Orenstein D,Ahrens R,Oermann CM,Aitken ML,Mahl TC,Young KR Jr,Dunitz J,Murray FT

doi

10.1097/01.mpa.0000202952.10612.21

subject

Has Abstract

pub_date

2006-04-01 00:00:00

pages

258-63

issue

3

eissn

0885-3177

issn

1536-4828

pii

00006676-200604000-00004

journal_volume

32

pub_type

杂志文章,多中心研究

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