Cystic FibrosisNCT04966897
Assessment of a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
A study testing Standard Nutritional Supplement + PERT in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by GlycosBio, Inc. at 1 site in United States.
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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- What is being tested?
- Standard Nutritional Supplement + PERT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2018to 30 Sep 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Clinical Research Center (CRC) at Texas Children's Hospital
Houston, Texas, 77030
About this study
Official title: Randomized, Double-blind, Cross-over Trial Assessing a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
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.
Read the 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.
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Being tested
- GlycosBio Nutritional Supplement + PERT placebo, Standard Nutritional Supplement + PERT
- Keywords
- nutritional supplement, cystic fibrosis, PERT-free, pancreatic enzyme, enzyme modified oil, exocrine pancreatic insufficiency
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlycosBio Nutritional Supplement + PERT placeboDietary 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
Standard Nutritional Supplement + PERTDietary 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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). Receives: Dietary Supplement: Standard Nutritional Supplement + PERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 hours
Maximum serum triglyceride concentration
Maximum serum triglyceride concentration during the 6 hour period following administration of the oral nutritional supplement.
Measured: 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.
Measured: 6 hours
Secondary outcomes (7)
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.
Measured: 6 hours
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.
Measured: 6 hours
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.
Measured: 6 hours
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.
Measured: 6 hours
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.
Measured: 6 hours
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.
Measured: 6 hours
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.
Measured: 6 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2021
- Study start31 Aug 2018
- Primary completion31 May 2019
- Study completion30 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- 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.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- GlycosBio, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Baylor College of Medicine
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04966897
- Sponsor's protocol ID
- GLYCOS-001-CF17
- Status last verified
- Jul 2021
- First submitted
- 8 Jul 2021
- Last updated
- 22 Jul 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-blind, Cross-over Trial Assessing a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT04966897
- Acronym / sponsor ID
- GLYCOS-001-CF17
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Keywords
- nutritional supplement, cystic fibrosis, PERT-free, pancreatic enzyme, enzyme modified oil, exocrine pancreatic insufficiency
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- GlycosBio, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Baylor College of Medicine
- Study start
- 31 Aug 2018 (actual)
- Primary completion
- 31 May 2019 (actual)
- Study completion
- 30 Sep 2019 (actual)
- First posted
- 19 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| GBNS + PERT placebo | Experimental | Dietary Supplement: GlycosBio Nutritional Supplement + PERT placeboGBNS + 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. |
| Standard Nutritional Supplement + PERT | Active comparator | Dietary Supplement: Standard Nutritional Supplement + PERTStandard 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). |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GlycosBio Nutritional Supplement + PERT placeboThe 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 | Dietary supplement | GBNS + PERT placebo |
| Standard Nutritional Supplement + PERTA 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. | Dietary supplement | Standard Nutritional Supplement + PERT |
Primary outcome
Change in serum triglyceride concentration
Time frame: 6 hours
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.
Maximum serum triglyceride concentration
Time frame: 6 hours
Maximum serum triglyceride concentration during the 6 hour period following administration of the oral nutritional supplement.
Area under the curve (AUC) for triglyceride serum levels
Time frame: 6 hours
Incremental area under the curve (AUC) for triglyceride serum levels during the 6 hour time period following following administration of the oral nutritional supplement.
7 secondary outcomes
Serum glucose
Time frame: 6 hours
Total cholesterol
Time frame: 6 hours
HDL cholesterol
Time frame: 6 hours
LDL cholesterol
Time frame: 6 hours
VLDL cholesterol
Time frame: 6 hours
Incidence of gastrointestinal (GI) symptoms
Time frame: 6 hours
Number of stools and other abdominal symptoms
Time frame: 6 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Standard Nutritional Supplement + PERT in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by GlycosBio, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in serum triglyceride concentration (time frame: 6 hours). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2021.