Curing Diabetes

1. What is the investigational drug (CELZ-201) made of?
CELZ-201 is prepared from Perinatal Tissue Derived Cells (PTDCs). These are cells that are harvested immediately before or after birth from umbilical cord blood and tissue from a single human donor source. The Perinatal Tissue Derived Cells used in this trial will come from a single donor mother, at the time of childbirth, who experienced a normal, non-complicated pregnancy. These same PTDCs were found to be safe in subjects suffering from other diseases but have not yet been studied in patients with Type 1 diabetes.

2. Where will the trial take place?
The single trial site will be at The Diabetic Research Institute (DRI) in Miami, Florida.

3. What procedure will I have to undergo to receive CELZ-201?
This investigational drug will be administered as a single dose into the artery that supplies blood to the pancreas. Since the drug will be directly administered to your pancreas, the study doctor will insert a small catheter or tube in either your wrist or groin. Using an x-ray like machine, the doctor will confirm that the catheter is in the correct location prior to drug administration. After the infusion, you will stay overnight in the hospital for close observation and lab work. You will be released the following morning.

4. Has this procedure been done before in humans?
This particular procedure has never been done in the United States. The same Perinatal Tissue Derived Cell procedure has been used in other clinical trials but not in diabetes trials. There have also been studies using the same technique of administering medicine directly to the pancreas via an artery, but they were not administering the same investigational drug.

5. How will I know if I’m eligible for the trial?
The main criteria for participating in this trial include:
– Age 18 – 35 years
– Be newly diagnosed with Type 1 Diabetes (<180 days from first diagnosis)
– Have stable kidney and liver function

Additional inclusion and exclusion criteria will be reviewed during the screening process.

6. Will anti-rejection drugs be needed in this trial?
No anti-rejection or immunosuppression drugs will be required in this trial.

7. Will everyone who participates be given the same treatment?
Not every patient will receive the same treatment. The patients will be randomized to treatment and control groups at a 2:1 ratio. All patients will receive excellent diabetes care and education, but those randomized to the treatment group will also receive a single dose of CELZ-201.

8. How many patients will participate in this clinical trial?
Phase I/II trials are designed to be the first step in the clinical research process and will provide important data for larger studies. The FDA (Food and Drug Administration) has approved this pilot trial for eighteen (18) participants. 

9. How long is this trial?
If you are eligible to participate in this trial, you will need to return for follow-up every 3-6 months for two years. If you are selected for the treatment group and receive CELZ-201, you will (also) need to follow up every week for four weeks following the infusion procedure.

10. If I participated in a previous trial, would I be eligible for this trial?
This will depend on the type of trial you participated in. If you received another investigational drug during a previous trial, you may or may not qualify. Please call the DRI’s Clinical Team at 305-243-5321 to discuss your eligibility if you have previously participated in any other clinical trial.

11. After submitting the application, how soon will I be notified if I am eligible to participate in this clinical trial?
A member of the DRI’s Clinical Team will be notifying you of your status within two (2) weeks of your application submission. Each applicant will be contacted individually by phone, mail or email. Based on the information in your initial application expressing your interest in participating in this trial, a phone interview may be set up to conduct an initial screening to evaluate your eligibility. If you meet the initial screening criteria, you may be invited to the DRI to complete the screening process by undergoing multiple tests to determine your eligibility as a potential participant. 

12. How much does it cost to participate in this trial?
You will likely have expenses to participate in this trial that may or may not be covered by your insurance. After meeting the initial screening criteria and having been selected for participation in this clinical trial, you may have expenses associated with travel to and from the clinic/hospital where screening for this study will take place. Patients will be responsible for travel and housing costs for their initial screening process. Once enrolled, each patient may receive a $75 reimbursement for travel related expenses to help offset any costs for each visit to the clinical site. You and your insurance carrier will be responsible for paying for any laboratory studies that are conducted outside of the research protocol. The study will also pay for all costs related to the infusion procedure, including the overnight stay.

13. Will this clinical trial be open to children?
No, this trial is not currently open to children. The safety and effectiveness of this new investigational drug will first be evaluated in adults.

If you are interested in participating in this clinical trial or wish to receive additional information on the trials and/or eligibility criteria, please call 305-243-5321. You may also send an email to: islet@med.miami.edu.