Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)

Forest Laboratories

Status and phase

Completed

Phase 3

Conditions

Pancreatic Insufficiency

Cystic Fibrosis

Treatments

Drug: Ultrase® MT12

Study type

Interventional

Funder types

Industry

Identifiers

NCT00880100

UMT12CF08-01

Details and patient eligibility

About

Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).

Full description

This is a multicenter, explorative, phase IIIb, open-label study in patients with CF and PI. The study consists of a screening visit (visit 1), followed by a baseline phase of 9 days (plus a 5-day window if necessary) during which the regular pancreatic enzyme will be maintained and 10 stool samples will be collected over 5 days, for baseline evaluation of steatorrhea. Afterward, a treatment phase of 19 days (plus a 5-day window if necessary) with Ultrase® MT12 will follow (the usual pancreatic enzyme will be replaced by Ultrase® MT12). Over the last 5 days of the treatment phase, 10 additional stool samples will be collected, for evaluation of steatorrhea.

Enrollment

49 patients

Sex

All

Ages

2 to 6 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Male or female patients aged 2 to 6 years inclusively
Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
Patients receiving current treatment of PI with pancreatic enzymes
The parent or legal guardian signed informed consent form (ICF) and is mentally able to understand and comply with the study procedures

Exclusion criteria

Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20) for PI in the last 30 days
Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
Patients with use of any prohibited medication or product at study entry and during the course of the study
Patients with chronic use of narcotics
Patients with use of bowel stimulants and/or laxatives more than once a week
Patients with presence of acute pancreatitis or exacerbation of chronic pancreatic disease
Patients with presence of an acute infection that needed to be treated with oral or intravenous (IV) broad-spectrum antibiotics
Patients having history of significant bowel resection; small bowel resection for meconium ileus at birth and appendectomy were accepted. Patients with Presence of dysmotility disorders
Patients with presence of chronic or severe abdominal pain
Patients unable to comply with diet requirement
Patients receiving enteral tube feeding overnight at study entry or who will need to receive enteral tube feeding overnight during the course of the study
Patients with history of or a current diagnosis of clinically significant portal hypertension
Patients with presence of poorly controlled diabetes according to the Investigator's clinical judgment
Patients having any condition or pre-study laboratory abnormality or history of any illness which, in the opinion of the Investigator, might have put the patient at risk, prevented the patient from completing the study, or otherwise affect the outcome of the study
Patient with use of any investigational drug within 30 days prior to the date of signature of the ICF