A 90 Day, Phase 4,Open Labeled Exploratory Study of RELiZORB
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|ClinicalTrials.gov Identifier: NCT03530852|
Recruitment Status : Not yet recruiting
First Posted : May 21, 2018
Last Update Posted : July 23, 2018
|Condition or disease||Intervention/treatment||Phase|
|Short Bowel Syndrome Malabsorption||Device: Relizorb||Not Applicable|
Short bowel syndrome (SBS) is often due to the loss of large amounts of small intestine that compromises digestive absorption. The treatments include (1) a high-calorie diet that includes vitamins, minerals, carbohydrates, proteins and fats; (2) injections of vitamins and minerals; (3) administration of drugs to slow the normal movement of the intestine or to increase the surface area of the intestinal lining; and (4) feeding through the vein (i.e., parenteral nutrition or PN). Many patients cannot wean from PN due to reduced intestinal length or function. Patients on long-term PN frequently experience serious metabolic complications, sepsis, hepatic biliary disorders including cholestasis, and fibrosis and can progress to liver failure. Full intestinal feeding (enteral nutrition) without PN is the optimal way to prevent the above complications.
Enterally administered long chain triglycerides in patients with SBS, especially those with hepatic dysfunction, are not well tolerated due to bile acid malabsorption, which leads to decreased micelle formation and fat digestion. The dietary fat is unable to be emulsified by the bile acids and acted on by lipases before exiting the patient as stool. Switching to other forms of fat such as medium-chain triglycerides (MCTs) that do not require micelles for absorption may be better tolerated in patients with bile acid or pancreatic insufficiency but are not optimal as they increase the osmotic load in the intestine. This may increase the chance of stool dumping; moreover, MCTs do not contain essential fatty acids (FAs). The ability to provide the essential FAs such as those present in enteral formulas in a form that does not require the formation of micelles for absorption, would allow patients with SBS and those who are no longer PN dependent to receive adequate nutrition and continue to maintain the same growth trajectory as when they received the majority of their nutrition parenterally.
RELiZORB is a digestive enzyme cartridge connected in-line with enteral feed tubing sets designed to mimic the function of pancreatic lipase. It is hypothesized that by using an external lipase device (RELiZORB) enteral nutrition will be better absorbed, and PN dependence reduced as enteral autonomy is increased. This product uniquely eliminates the need for intestinal emulsification and lipase activity and eliminates the risk of drugs, including lipases, allowing absorption at the time the diet enters the gut. The device has been shown to digest >90% of fat in most enteral formulas.
This is a phase 4, open label single center clinical trial to determine the safety, tolerability, and bioavailability of the RELiZORB enzyme cartridge with enteral nutrition when used daily for 90 days in pediatric subjects with SBS, aged 5 years - 18 years, who are PN dependent. The change in PN calories from baseline, assessed at Day 7, 14, 28, 60, and 90, will be assessed by area under the curve and presented with a 95% confidence interval. The number (percent) of treatment-emergent adverse events, grade 2 or above, will be tabulated. Changes in growth, fecal fat/protein, plasma FAs, PN volume, and enteral/oral nutrition will be described.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A 90 Day, Phase 4, Open Labeled Exploratory Study of RELiZORB to Evaluate Safety, Tolerability, and Nutrient Absorption in Children With Short Bowel Syndrome Who Are Dependent on Parenteral Nutrition|
|Estimated Study Start Date :||August 15, 2018|
|Estimated Primary Completion Date :||June 15, 2021|
|Estimated Study Completion Date :||September 30, 2021|
Experimental: Relizorb treatment
Patients will have tube feeds placed through chamber and evaluate wean from parenteral nutrition
Tube feeds run across device to digest fats.
- Effectiveness of the RELiZORB enzyme cartridge on the absorption of enteral nutrition based on PN calories [ Time Frame: 90 days ]To determine the effectiveness of the RELiZORB enzyme cartridge on the absorption of enteral nutrition when used daily for a total 90 days of treatment, in pediatric subjects with SBS who are PN dependent, aged 5 years - 18 years, by measuring the change in PN calories from baseline, assessed at days 7, 14, 28, 60, and day 90. Each subject will be on the study for a total of 90 days.
- Effectiveness of the RELiZORB enzyme cartridge on the absorption of enteral nutrition based on body weight change [ Time Frame: 90 days ]• To determine the effectiveness of the RELiZORB enzyme cartridge on the absorption of enteral nutrition when used daily for a total 90 days of treatment, in pediatric subjects with SBS who are PN dependent, aged 5 years - 18 years, by measuring the change in body weight from baseline, assessed at days 7, 14, 28, 60, and day 90. Each subject will be on the study for a total of 90 days.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03530852
|Contact: Mark Puder, MD, PhDfirstname.lastname@example.org|
|United States, Massachusetts|
|Boston Children's Hospital||Not yet recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Mark Puder, MD, PhD 617-355-1838 email@example.com|