DRI Pilot Clinical Trial to Test a New Site for a BioHub Platform
The Diabetes Research Institute has received approval from the Food and Drug Administration (FDA) to proceed with a Phase I/II clinical trial that will test the omentum, an apron-like lining inside the abdomen, as a possible location in the body for a DRI BioHub. In this pilot trial, researchers will transplant donor islet cells into the omentum within a “biodegradable scaffold,” one of the approaches originally conceived as a potential BioHub platform.
The biodegradable scaffold is made using a patient's own plasma, the liquid part of the blood. Researchers combine the islet cells with the plasma, apply it to the omentum, and then add thrombin, a commonly used, clinical-grade enzyme. When mixed together, these substances create a gel-like material that sticks to the omentum and holds the islets in place. This section of the omentum will be folded over and stitched, creating a pouch around the biodegradable scaffold mixture.
Over time, the body will absorb the plasma/thrombin gel leaving the islet cells intact, while new blood vessels are formed to support the cells’ survival and function. Patients involved in this pilot trial will be given the immunosuppressive drugs currently used for other clinical islet transplantation studies.
The DRI is now screening participants for this pilot clinical trial and for pilot clinical trials that will be conducted in the future to have as many patients on the waiting list as possible to maximize the chances of donor matching and the utilization of pancreases that become available.
After printing out the screening form, please complete it and return it to the DRI Clinical Cell Transplant Program by faxing it to 305-243-1058. For additional information on the trials and/or eligibility criteria, please call 305-243-5321 or send an e-mail to: firstname.lastname@example.org
Frequently Asked Questions (FAQ)
1. Is this pilot clinical trial testing the DRI BioHub?
This is a Phase I/II clinical trial representing the first stage in testing the omentum, an apron-like lining covering the inside of the abdomen, as an alternative site within the body to house transplanted islet cells. It will also test one of the platforms being considered for a DRI BioHub, a “biodegradable scaffold.” The BioHub is a platform technology comprised of several components that work together in the DRI’s attempt to mimic the native pancreas.
2. When will this pilot trial begin?
The DRI's Clinical Cell Transplant Team is actively screening patients and anticipates that this Phase I/Phase II clinical trial will begin before the end of this year.
3. What are the criteria for this pilot trial?
Some of the initial criteria for participation include:
- Age 18-60- More than 5 years with type 1 diabetes
- Severe hypoglycemia
- Hypoglycemic unawareness - unable to sense low blood sugar levels
Additional inclusion and exclusion criteria will be reviewed during the screening process.
4. Will anti-rejection drugs be needed in this trial?
Yes. While the DRI’s goal is to eliminate the use of these systemic drugs, this first trial is designed to test the new omental pouch site. To compare the results of the previously used liver site to the omentum (abdominal lining), DRI researchers must limit the variables that might confuse the results. Therefore, in this initial step, they will use the same immunosuppression, islet processing and matching criteria as in previous islet transplant studies.
5. Will everyone who participates be given the same treatment or will a placebo be involved?
Each person selected for participation will receive the same treatment as indicated in the approved protocol. Organ and tissue transplantation studies do not use placebo procedures in their testing of a new treatment. They do compare the current trial results with other previously conducted trial protocols to determine the effects of a new therapy.
6. How many patients will participate in this clinical trial?
Pilot trials are designed to be the first step in the clinical research process and provide important data for larger studies. This pilot trial will be limited to no more than six participants.
7. If I participated in a previous trial, would I be eligible for this pilot trial?
It depends on what type of trial you participated in. Individuals who previously received a transplant will not be eligible for this initial pilot trial. If you participated in any other clinical trial, please call the DRI's Clinical Cell Transplant Program at 305-243-5321 to discuss your eligibility.
8. If I submitted a screening application in the past, am I already among the pool to be considered for islet transplantation pilot trials?
If you have been identified as an eligible candidate for islet transplantation within the past year, you should contact the DRI's Clinical Cell Transplant Program at 305-243-5321 to confirm your interest and ask if additional information is needed to update your application. If it's been more than a year, you will need to resubmit the application with current information.
9. After submitting the application, how long will it take for me to learn whether or not I am eligible to participate in this pilot clinical trial?
A member of the DRI's Clinical Cell Transplant Program will be notifying you of your status within two weeks of your application submission. Each applicant will be contacted individually by mail or email. If you meet the initial screening criteria, you will be invited to the DRI to complete the screening process by undergoing a battery of tests to determine your eligibility and whether you can be included as a participant on the active waiting list.
10. How much does it cost to participate in these trials?
After meeting the screening criteria and being selected for participation in the clinical trial, the DRI will cover all medical, diagnostic testing and drug costs related to the transplant procedure and medications. Patients will be responsible for travel and housing costs for their initial screening process.
11. If I meet the criteria to participate in the pilot trial and am placed on the waiting list, how do the DRI researchers select who on this list gets the transplant?
The selection of individuals from the active waiting list to receive islets is based on several factors including, but not limited to, donor-recipient tissue compatibility (commonly referred to as “matching”), time on the waiting list and obtaining a sufficient number of healthy islets from a donor organ to meet the metabolic demands of each potential recipient. This is based on a ratio of the recipient's body weight and the islet yield following the islet isolation process. The more candidates the DRI has on the active waiting list, the better the chance for a suitable “match.”
12. Will these clinical pilot trials be open to children?
No. Safety and effectiveness of this new approach will first be evaluated in adults. The risks associated with life-long exposure to anti-rejection drugs currently on the market cannot be justified for use in children or even in adults who are able to manage their diabetes with available tools, technology and education. The risk that these anti-rejection drugs pose, however, is justified in the case of individuals needing life-saving organ transplants or for patients with type 1 diabetes who are unable to detect their own dangerously-low blood sugars, those with hypoglycemic unawareness. In these cases, the risk-benefit ratio is justified.
13. What are the next steps after this pilot trial?
If we can demonstrate safety and achieve results comparable to previous successful islet trials using the liver, the DRI will seek FDA approval to incorporate other components into the BioHub platform that will help mimic the native pancreas. These components may include: oxygen promoters, which will provide the islets with critical levels of oxygen while blood vessels are formed; different types of helper cells that can promote blood vessel growth or perhaps decrease inflammation; local drug delivery approaches so that we can replace full-body anti-rejection drugs and their harsh side effects; and islets that are protectively coated or encapsulated to further shield them from injury. The goal of incorporating these components into the BioHub platform, alone or in combination, is to achieve long-term islet survival and optimal function, as well as to eliminate the need for systemic immunosuppression. In addition, DRI scientists and collaborators are developing and testing approaches for therapeutic options to reset the immune system and establish immune tolerance to accept the new, healthy islets, as well as prevent the recurrence of the autoimmune process that caused diabetes in the first place. If successful, life-long immunotherapy would not be needed.
Benno Schmidt reports on the DRI's clinical trial to test the omentum as a transplant site in the body for a DRI BioHub.
Watch DRItv and learn more >
The pilot clinical trial will test the omentum, an apron-like lining inside the abdomen, as a possible site for housing a DRI BioHub.
The biodegradable scaffold is made using a patient's own plasma together with thrombin, a clinical-grade enzyme. When combined, they create a "sticky gel" that holds the islets in place. The area of the omentum is then folded over and stitched, creating a protective "pouch" around the scaffold mixture.
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