An Open-Label Clinical Study Evaluating the Long-Term Safety of ALTU-135 for the Treatment of Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 200

Inclusion Criteria

= 7 years of age
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 either
? A genotype with two identifiable mutations consistent with CF, 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 µg/g measured at the Screening Visit unless
previously documented
6. Able to take pancreatic enzyme supplementation in the form of capsules
7. Able to perform the testing and procedures required for this study, as judged by the Investigator
8. Willing and able to provide informed consent or assent

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. < 7 years of age
2. Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence) during the study
3. Participation in an investigational study of a drug, biologic, or device not currently
approved for marketing within 30 days prior to the Screening Visit except for the
Phase III Efficacy Study (Protocol 0000726)
4. History of fibrosing colonopathy
5. History of liver transplant, lung transplant or significant surgical resection of the bowel
Note: 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 chronic diarrheal illness unrelated to PI (e.g., sprue or inflammatory bowel disease)
7. ALT or AST level > 5 x ULN, or total bilirubin level > 1.5x ULN at the Screening Visit
or at Baseline (except for patients with Gilbert Syndrome)
8. Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF
9. Unable to discontinue enteral tube feedings during the study
10. 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.
11. Baseline CFA = 93% from the Phase III efficacy study (Protocol 0000726)
12. Patient is unlikely to complete the study, as determined by the Investigator

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety and tolerability of ALTU-135 treatment in patients with Cystic Fibrosis related exocrine Pancreatic Insufficiency;Secondary Objective: ;Primary end point(s): To evaluate the long-term safety and tolerability of ALTU-135 treatment in patients with CYstic Fibrosis related exocrine Pancretic Insufficiency

Secondary Outcome Measures

Name	Time	Method

Related Trials