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


METHODS
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).


CONCLUSIONS
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.
Borowitz
Drucy
D
Women and Children's Hospital of Buffalo, Division of Pediatric Pulmonology, State University of New York at Buffalo, Buffalo, NY 14222, USA. dborowitz@upa.chob.edu
Goss
Christopher H
CH
Limauro
Stacey
S
Konstan
Michael W
MW
Blake
Kathryn
K
Casey
Susan
S
Quittner
Alexandra L
AL
Murray
Frederick T
FT
D000293:Adolescent; D000328:Adult; D000681:Amylases / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D000704:Analysis of Variance; D001786:Blood Glucose / Q000378:metabolism; D002648:Child; D003550:Cystic Fibrosis / Q000150:complications* / Q000201:enzymology; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D010188:Exocrine Pancreatic Insufficiency / Q000150:complications / Q000188:drug therapy* / Q000201:enzymology*; D005223:Fats / Q000032:analysis / Q000378:metabolism; D005243:Feces / Q000737:chemistry; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007408:Intestinal Absorption / Q000187:drug effects; D008049:Lipase / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D009584:Nitrogen / Q000032:analysis / Q000378:metabolism; D010447:Peptide Hydrolases / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D001786:Blood Glucose; D005223:Fats; C515846:liprotamase lipase; D008049:Lipase; D000681:Amylases; D010447:Peptide Hydrolases; D009584:Nitrogen