Clinical trial NCT04966897: Assessment of a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
Official title: Randomized, Double-blind, Cross-over Trial Assessing a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Keywords: nutritional supplement
cystic fibrosis
PERT-free
pancreatic enzyme
enzyme modified oil
exocrine pancreatic insufficiency
Lead sponsor: GlycosBio, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Baylor College of Medicine
First posted: 2021-07-19
Enrollment: 10
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: Single center, randomized, double-blind, cross-over trial with a single blind stage evaluation assessing the GBNS for safety, tolerability and blood lipid levels compared to a standard nutritional supplement with PERT. Phase 1: After an overnight fast, subjects in Arm 1 administered PERT placebo with the GBNS and those in Arm 2 will be administered standard nutritional supplement with PERT. Blood samples collected at baseline and hourly over 6 hours for all subjects. Subjects will return for crossover treatment no fewer than 4 days and no more than 14 days after with the same steps as the above but with patients switching treatment arms. Phase 2: Starting the day following the 2nd treatment the 6 day, single-blind safety stage begins with subjects randomized to GBNS with PERT-placebo or the standard nutritional supplement with PERT. One week after the end of the Home Trial (+/- 3 days) patients will return for an end of study visit.
Primary purpose: TREATMENT

Brief summary: This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.

Detailed description: This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.

The study hypothesis is that the PERT-free nutritional supplement will be equivalent to, or superior to, a standardized nutritional shake with regard to fat absorption, as indicated by blood lipid (triglyceride) levels while maintaining tolerability and safety without the use of PERT. The objective is to evaluate efficacy, safety, and tolerability of a "PERT-free" nutritional shake compared to a standard nutritional shake used concomitantly with PERT. Measurements include components of a standard lipid blood panel (triglycerides, cholesterol, HDL cholesterol, LDL cholesterol, and VLDL cholesterol), absorption of fat-soluble vitamins (vitamin D) and symptoms associated with EPI. Other objectives include evaluating palatability and patients' impressions of the products.

Eligibility criteria: Inclusion Criteria:

* Provision of signed and dated informed consent form.
* Stated willingness to comply with all study procedures and availability for the duration of the study.
* Male or female, aged 12 years or older.
* Diagnosis of cystic fibrosis
* Currently receiving treatment with a commercially available pancreatic enzyme product for more than 3 months.
* Clinically stable condition without evidence of acute respiratory disease within 1 month of enrollment.
* Stable body weight defined as no more than 5% decline within 3 months of enrollment.
* Females of child-bearing potential should agree to continue using a medically acceptable method of birth control throughout the study and for 30 days immediately after the last dose of study drug. Medically acceptable methods of birth control include bilateral tubal ligation or the use of either a contraceptive implant, a contraceptive injection (Depo-Provera™), an intrauterine device, or an oral contraceptive taken within the past 3 months where the subject agrees to continue using during the study or to adopt another birth control method, or a double-barrier method which consists of a combination of any two of the following: diaphragm, cervical cap, condom, or spermicide.
* Ability to take oral medication and oral liquid nutritional supplements and be willing to adhere to the study interventions.
* Agreement to adhere to Lifestyle Considerations as defined by the study protocol throughout the duration of the study.

Exclusion Criteria:

* Evidence of cardiovascular, respiratory (except underlying disease), urogenital, gastrointestinal/hepatic (except underlying disease), hematologic/immunologic, head, ears, eyes, nose, throat, dermatologic/connective tissue, musculoskeletal, metabolic/nutritional (except underlying disease), endocrine (except controlled diabetes mellitus), neurologic/psychiatric, milk, nut or soy allergies, recent major surgery, or other relevant diseases as revealed by history, physical examination and/or laboratory assessments, which could limit participation in or completion of the study.
* History of acute abdomen in the last year.
* History of fibrosing colonopathy.
* History of distal intestinal obstruction syndrome (DIOS) within 6 months prior to enrollment.
* Solid organ transplant or surgery affecting the large bowel other than appendectomy.
* Small bowel surgery that significantly affected absorptive capacity (e.g. gastrectomy or pancreatectomy).
* Intestinal inflammatory diseases including chronic diarrheal illness unrelated to pancreatic insufficiency.
* Celiac disease or Crohn's disease.
* Receiving enteral tube feeds for ≥50% of daily calorie intake.
* Pregnancy or lactation.
* Any type of malignancy involving the digestive tract in the last 5 years.
* Known allergy to pancreatin or inactive ingredients (excipients) of pancreatin capsules.
* Suspected non-compliance or non-cooperation.
* Intake of experimental drugs within 30 days prior to study start.
* Mental disability or any other lack of fitness, in the Investigator's opinion, to preclude subject's participation in or ability to complete the study.
* Diagnosis of human immunodeficiency virus in medical history.
* Listed for lung transplantation or other solid organ transplant or documented forced expiratory volume (FEV) ≤25%.
* Use of lipid lowering therapy including statins, fibrates, niacin, and proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors that cannot be held at least 14 days prior to Day 1 and through Day 15 of the study.

Primary outcomes:
measure: Change in serum triglyceride concentration
timeFrame: 6 hours
measure: Maximum serum triglyceride concentration
timeFrame: 6 hours
measure: Area under the curve (AUC) for triglyceride serum levels
timeFrame: 6 hours
Secondary outcomes:
measure: Serum glucose
timeFrame: 6 hours
measure: Total cholesterol
timeFrame: 6 hours
measure: HDL cholesterol
timeFrame: 6 hours
measure: LDL cholesterol
timeFrame: 6 hours
measure: VLDL cholesterol
timeFrame: 6 hours
measure: Incidence of gastrointestinal (GI) symptoms
timeFrame: 6 hours
measure: Number of stools and other abdominal symptoms
timeFrame: 6 days
Other outcomes:
measure: Palatability
timeFrame: 6 days
Locations:
city: Houston
country: United States
facility: Clinical Research Center (CRC) at Texas Children's Hospital
state: Texas
Study officials:
References: