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

Cystic Fibrosis Clinical Trial

Official title:

Randomized, Double-blind, Cross-over Trial Assessing a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04966897
• Other study ID #: GLYCOS-001-CF17
• Status: Completed
• Phase: N/A
• Start date: August 31, 2018
• Est. completion date: September 30, 2019

Study information

• Verified date: July 2021
• Source: GlycosBio, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: September 30, 2019
• Est. primary completion date: May 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency
• Fibrosis

Intervention

Dietary Supplement:
• GlycosBio Nutritional Supplement + PERT placebo
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
• Standard Nutritional Supplement + PERT
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.

Locations

Country	Name	City	State
United States	Clinical Research Center (CRC) at Texas Children's Hospital	Houston	Texas

Sponsors (2)

Lead Sponsor	Collaborator
GlycosBio, Inc.	Baylor College of Medicine

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Palatability	Differences in the palatability of each oral nutritional supplement assessed via a participant survey.	6 days
Primary	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
Primary	Maximum serum triglyceride concentration	Maximum serum triglyceride concentration during the 6 hour period following administration of the oral nutritional supplement.	6 hours
Primary	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	Serum glucose	Maximum change from baseline for serum serum glucose based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.	6 hours
Secondary	Total cholesterol	Maximum change from baseline for serum total cholesterol based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.	6 hours
Secondary	HDL cholesterol	Maximum change from baseline for serum HDL cholesterol based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.	6 hours
Secondary	LDL cholesterol	Maximum change from baseline for serum LDL cholesterol based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.	6 hours
Secondary	VLDL cholesterol	Maximum change from baseline for serum VLDL cholesterol based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.	6 hours
Secondary	Incidence of gastrointestinal (GI) symptoms	Incidence of gastrointestinal (GI) symptoms including nausea, heartburn, abdominal pain, steatorrhea, bloating and other reported symptoms over a 6 hour period following administration of the oral nutritional supplement.	6 hours
Secondary	Number of stools and other abdominal symptoms	Average daily number of stools and the percentage of days with 1) hard or formed/normal or soft stools, 2) no steatorrhea, 3) no abdominal pain, and 4) no bloating during the 6 days during Phase II (home phase) of trial.	6 days