CELZ-201

Can I Join the CELZ-201 Trial?

To be able to join the clinical trial, you must:

1. Be age 18—35 years old. You must be greater than 18 years of age to participate in the trial.
2. Have a new diagnosis of type 1 diabetes. You must be treated within 180 days of being diagnosed with type 1 diabetes by your physician or provider.
3. Have a negative pregnancy test if you are female. Participants can not participate in the trial if they are pregnant. Both male and female participants must agree to use contraception.
4. Have no other major medical problems. All participants must be healthy other than the new diagnosis of type 1 diabetes. The research team will let you know if you have any medical problems that wouldn’t allow you to participate in the trial.

How Long Will the Study Last?

During the screening period, to determine if you are eligible to receive the study treatment, you will come to the study center up to two times before receiving treatment.

After you have received the study treatment, you will be required to stay in the study for approximately 2 years.

Background

Type 1 diabetes is an autoimmune disease where the person’s immune system mistakenly attacks the beta cells in the pancreas. The beta cells are what make insulin in the human body. As the autoimmune attack progressively destroys the beta cells, the insulin production continues to slow. Without insulin, the body can’t move glucose from the bloodstream into cells and the patient will develop diabetes.

The purpose of this research study is to test the safety and feasibility of a single dose of CELZ-201, when it is given to people with new onset type 1 diabetes. This study involves an investigational drug.

The Study Drug: CELZ-201

CELZ-201 is prepared from Perinatal Tissue Derived Cells (PTDCs), all of which are obtained from a single human donor source. The batch of PTDCs we plan to use will be derived from a Master Cell Bank established from a single donor tissue collected at the time of delivery, from a mother with a normal, non-complicated pregnancy. The study drug will be administered as a one time infusion into the dorsal pancreatic artery.

Preliminary observations in subjects with other diseases suggest that these PTDCs may modulate immune responses and inflammation. Hence, the study team plans to investigate whether this preparation of PTDCs (CELZ-201) is safe, and yields benefits for patients with T1DM.

Study Design

There will be 18 patient included in this study.

• 12 patients will get the single infusion of CELZ-201 with standard of care medical management. They are in the treatment group.
• 6 patients will only receive only standard of care medical management. They are in the control group.

You can’t choose your group. A computer will choose your group for you and it is by chance, like flipping a coin. This is known as random assignment or randomization. You will have a one in two chances of receiving treatment with CELZ.

What to Expect?‍

Each study visit will include SOME or ALL of the following tests/procedures:

• Medical history. You will answer questions about your health, including medical history, allergies, illnesses, conditions, or surgeries.
• Physical examination. You will have an overall examination of your body. You will have your height and weight measured. Some visits may have shorter exams than others.
• Vital signs. Your blood pressure, temperature, heart rate, and breathing rate will be measured.
• Abdominal ultrasound. A probe is passed over your abdomen to look at your pancreas and other surrounding areas.
• 7-day blood glucose log. You will be asked to record your blood glucose levels 4 times/day, before breakfast, lunch, dinner, and at bedtime.
• Clarke survey. You will be asked to complete an 8 question survey about your awareness of low blood sugar.
• Laboratory blood draws. Blood will be taken from the vein for lab tests to check your health.
• Urine sample. You will be asked to give a sample of urine to test your kidney function and general health.
• Mixed-meal tolerance test. You must fast overnight for at least 10 hours and then will drink a liquid meal preparation. Your blood will be sampled at multiple times within 4 hours to assess C-peptide, insulin, glucose, glucagon, and amylin.
• Pregnancy test. You will be asked to give a urine sample to test for pregnancy.
• COVID-19 test. You will have a saliva or nasal swab test to determine if you are infected with the virus that causes COVID-19.

What to Expect if I am Selected for the Treatment Group?

If you are randomly selected to be a part of the treatment group and will receive the CELZ-201 drug, you will be required to have a catheter placed in your groin or wrist to access the dorsal pancreatic artery. The investigational drug will be infused under x-ray to make sure that it is delivered near your pancreas. Patients who are in the treatment group will also be required to have overnight observation in the hospital, and will be discharged the following morning.

You will also be asked to come to the research site at the University of Miami Diabetes Research Institute, where your study doctor or site staff will check your health, the effects of the Study Product, and to see if you can continue in the study.
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Visit 1: Day –30

• Approximately 30 days prior for screening and baseline visit

Visit 2: Day –1 and/or 0

• Randomization, hospitalization, and treatment
• Investigational CELZ-201 infusion for Group I (treatment arm)

Visit 3: Day 1

• Post operative care and follow up

Visits 4-7: Days 7, 14, 21, and 28

• One visit every week for close follow up and safety monitoring labs.

Visits 8-15: Months 3, 6, 9, 12, 15, 18, 21, and 24

• One visit every 3 months for safety monitoring and follow-up testing.
• In addition, at any time there may be unscheduled visits. A final or end of treatment visit will be done at month 24 or prior based on duration of your participation in the study.

Reimbursement of $75 per site visit will be paid to each selected trial participant.

What are the Risks of Participation in the Clinical Trial?

There are possible risks to the infusion procedure/administration of the investigational drug that include but are not limited to:

• Pain or discomfort at the catheter site
• Bleeding
• Infection/fever
• Blood clots
• Allergic reaction
• Variations in blood sugar
• Risk of tumor or cancer
• Injury to the pancreas or surrounding organs