Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis

J Pediatr. 2006 Nov;149(5):658-662. doi: 10.1016/j.jpeds.2006.07.030.

Abstract

Objectives: We studied a novel pancreatic enzyme product, ALTU-135, a proprietary formulation of microbially derived lipase, protease, and amylase, to determine its efficacy and safety in treatment of pancreatic insufficiency (PI) in patients with cystic fibrosis (CF).

Study design: Ambulatory subjects with CF-PI (n = 117) had baseline coefficient of fat and nitrogen absorption (CFA and CNA, respectively) determined in an inpatient setting while not receiving pancreatic enzyme replacement therapy. Subjects were then randomized to treatment with ALTU-135 containing 5000 (low), 25,000 (mid), or 100,000 (highest) units of lipase (1:1:0.15 of lipase:protease:amylase) for 28 days. After 14 days, CFA and CNA were re-measured. The primary outcomes were change from baseline in CFA and CNA between treatments.

Results: Treatment CFA was significantly greater in the mid and highest dose groups compared with that in the low dose group (P = .0229 and P =.0041, respectively); findings were similar for CNA. Subjects with baseline CFA < or = 40% and > 40% in the 2 higher dose groups had a mean increase of 31 and 8 percentage points in CFA, respectively (P < .0001).

Conclusion: ALTU-135 was efficacious during the 1-month study period at the dose of 25,000 units of lipase, 25,000 units of protease, and 3750 units of amylase.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Amylases / administration & dosage
  • Amylases / adverse effects
  • Amylases / therapeutic use*
  • Analysis of Variance
  • Blood Glucose / metabolism
  • Child
  • Cystic Fibrosis / complications*
  • Cystic Fibrosis / enzymology
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Exocrine Pancreatic Insufficiency / complications
  • Exocrine Pancreatic Insufficiency / drug therapy*
  • Exocrine Pancreatic Insufficiency / enzymology*
  • Fats / analysis
  • Fats / metabolism
  • Feces / chemistry
  • Female
  • Follow-Up Studies
  • Humans
  • Intestinal Absorption / drug effects
  • Lipase / administration & dosage
  • Lipase / adverse effects
  • Lipase / therapeutic use*
  • Male
  • Nitrogen / analysis
  • Nitrogen / metabolism
  • Peptide Hydrolases / administration & dosage
  • Peptide Hydrolases / adverse effects
  • Peptide Hydrolases / therapeutic use*
  • Treatment Outcome

Substances

  • Blood Glucose
  • Fats
  • liprotamase lipase
  • Lipase
  • Amylases
  • Peptide Hydrolases
  • Nitrogen