Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis (SPAN)
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
About this trial
This is an interventional treatment trial for Exocrine Pancreatic Insufficiency
Eligibility Criteria
Inclusion Criteria: A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride >60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype. On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range. CFA = or > 80% at screening while on stable PERT A fair or better nutritional status as defined by: BMI ≥16.0 kg/m2 for female patients ≥18 years of age, or BMI ≥16.5 kg/m2 for male patients ≥18 years of age Fecal elastase <100 µg/g of stool at screening Standard-of-Care medications including CFTR modulators are allowed Exclusion Criteria: History or diagnosis of fibrosing colonopathy Any chronic diarrheal illness unrelated to pancreatic insufficiency Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level 5 ×upper limit of normal (ULN), or total bilirubin level ≥1.5 ×ULN at Screening Feeding via an enteral tube during 6 months before screening Forced expiratory volume ≤30% at the Screening visit Changes in gastric acid suppressant therapy during the one month prior to screening for patients already on suppressant therapy.
Sites / Locations
- Central Florida Pulmonary GroupRecruiting
- The Cystic Fibrosis InstituteRecruiting
- Childrens Lung SpecialistsRecruiting
Arms of the Study
Arm 1
Experimental
Adrulipase
Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase.