A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - ND

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2007-000171-41-IT
• Date of registration: 07/01/2008
• Prospective Registration: No
• Primary sponsor: ALTUS PHARMACEUTICALS INC
• Public title: A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - ND
• Scientific title: A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - ND
• Date of first enrolment: 31/12/2007
• Target sample size: 176
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-000171-41
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Italy

Key inclusion & exclusion criteria

Inclusion criteria:
1.7 or more year-old 2. Females of childbearing potential must be willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence). 3.Diagnosis of CF based upon the following criteria: a. two clinical features consistent with CF; and b.either genotype with two identifiable mutations consistent with CF, c. or sweat chloride >60 mEq/L by quantitative pilocarpine iontophoresis. 4. Clinically stable with no evidence of acute upper or lower respiratory tract infection. 5. PI determined by fecal elastase <100 microg/g stool measured at Screening visit. 6. Able to take pancreatic enzyme supplementation in the form of capsules. 7. Able to perform the testing (e.g. stool collections) and inpatient stays required for this study, as judged by the Inestigator. 8. Willing and able to proide informed consent or assent. 9. Baseline CFA ≤80%
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Patient <7 years old. 2. CFA >80% at Baseline. 3. Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control (IUD; oral, transdermal or parental contraceptives; abstinence) during the study. 4. History of fibrosing colonopathy. 5. History of liver transplant, lung transplant or significant surgical resection of the bowel. Significant resection of the bowel is defined as any resection of the terminal ileum, or ileo-cecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (e.g., increased, or new, need for supplementation compared to pre-op in order to maintain the same nutritional status) should also be excluded. 6. Any acute or chronic diarrheal illness unrelated to PI (e.g. infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease). 7. Unable to discontinue enteral tube feedings during the study. 8. Known hypersensitivity to food additives. 9. Inability to consume the PP diets, in the judgment of the Investigator. 10. Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to the Screening Visit. 11. ALT or AST level > 5x ULN, or total bilirubin level > 1.5x ULN at the Screening Visit or at Baseline (except for patients with Gilbert Syndrome). 12. Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF?. 13. DIOS in the last six months prior to the Screening Visit. 14. Unable to discontinue the use of pancreatic enzymes. 15. Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the patient. 16. Patient is unlikely to complete the study, as determined by the Investigator. ? Eligible patients with ALT or AST level > 3x to 5x ULN at the Screening Visit or at Baseline will be tested for viral hepatitis. Other diagnostic testing may be performed at the discretion of the Investigator.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency.
MedDRA version: 9.1 Level: LLT Classification code 10011766 Term: Cystic fibrosis pancreatic

Intervention(s)

Product Name: ALTU-135
Product Code: ALTU-135
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Multienzymes (lipase, protease etc.)
CAS Number: 9001-62-1
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 32500-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use

Product Name: ALTUS-135
Product Code: ALTUS-135
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Multienzymes (lipase, protease etc.)
CAS Number: 9000-90-2
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 3750-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use

Product Name: ALTU-135
Product Code: ALTU-135
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Multienzymes (lipase, protease etc.)
CAS Number: 9074-07-1
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 25000-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: To determine the efficacy of ALTU-135 for the treatment of protein malabsorption in patient with CF-related exocrine PI. To determine the efficacy of ALTU-135 treatment in improving the absorption of carbohydrates in patients with CF-related exocrine PI. To determine the efficacy of ALTU-135 treatment in decreasing stool weight and frequency in patients with CF-related exocrine PI.

Main Objective: To determine the efficacy of ALTU_135 for the treatment of the fat malabsorption in patients with CF-related exocrine PI.

Primary end point(s): Mean change in CFA from Baseline Period to Double-Blind Treatment Period. To determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI.

Secondary ID(s)

0000726