Assessment of a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis

NCT04966897 | Completed

• 1 other identifier: GLYCOS-001-CF17
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Keywords

nutritional supplement; cystic fibrosis; PERT-free; pancreatic enzyme; enzyme modified oil; exocrine pancreatic insufficiency

Outcome Measures

Primary Outcomes (3)

• Change in serum triglyceride concentration

  Maximum change from baseline for serum triglyceride concentration based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.

  6 hours

• Maximum serum triglyceride concentration

  Maximum serum triglyceride concentration during the 6 hour period following administration of the oral nutritional supplement.

  6 hours

• Area under the curve (AUC) for triglyceride serum levels

  Incremental area under the curve (AUC) for triglyceride serum levels during the 6 hour time period following following administration of the oral nutritional supplement.

  6 hours

Secondary Outcomes (7)

• Serum glucose

  6 hours

• Total cholesterol

  6 hours

• HDL cholesterol

  6 hours

• LDL cholesterol

  6 hours

• VLDL cholesterol

  6 hours

Other Outcomes (1)

• Palatability

  6 days

Study Arms (2)

GBNS + PERT placebo

EXPERIMENTAL

GBNS + PERT placebo (drink volume sufficient to supply 0.5 g of MAG per kg of body weight plus PERT placebo capsules according to patient body weight.

Dietary Supplement: GlycosBio Nutritional Supplement + PERT placebo

Standard Nutritional Supplement + PERT

ACTIVE COMPARATOR

Standard nutritional supplement + PERT (drink volume sufficient to supply 0.5 g of TAG per kg of body weight plus PERT capsules according to body weight).

Dietary Supplement: Standard Nutritional Supplement + PERT

Interventions

GlycosBio Nutritional Supplement + PERT placebo DIETARY_SUPPLEMENT

The lipid (fat) in the GlycosBio Nutritional Supplement (GBNS) was provided in the form of a re-structured lipid monoacylglyceride (MAG) produced by the study sponsor. MAGs are readily absorbed by enterocytes without the need of digestion by pancreatic lipases. The "MAG oil" is produced enzymatically from almond oil. The product also contains essential amino acids, carbohydrates as simple sugars and fat-soluble vitamins and minerals. The GBNS to be administered in a volume sufficient to supply 0.5 g of MAG per kg of body weight

GBNS + PERT placebo

Standard Nutritional Supplement + PERT DIETARY_SUPPLEMENT

A 5.9% fat triacylglycerol-based commercially available nutritional supplement (Boost Plus®, Nestlé Health Science, Bridgewater, NJ) in a volume sufficient to supply 0.5 g of TAG per kg of body weight over the same time period and PERT capsules (24,000 iu/capsule) at a dose of 2,500 iu of lipase activity per gram of fat ingested.

Standard Nutritional Supplement + PERT

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• GlycosBio, Inc.: lead
• Baylor College of Medicine: collaborator

Study Sites (1)

Clinical Research Center (CRC) at Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: July 8, 2021
• First Posted: July 19, 2021
• Study Start: August 31, 2018
• Primary Completion: May 31, 2019
• Study Completion: September 30, 2019
• Last Updated: July 22, 2021

Record last verified: 2021-07

Locations

US (1)