Safety and Efficacy of CELZ-201 in Patients With Recent Onset Type 1 Diabetes

NCT ID: NCT05626712

Last Updated: 2025-10-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-07

Study Completion Date

2028-01-31

Brief Summary

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The brief purpose of this research study is to learn about the safety and efficacy of intra-arterial administration of CELZ-201 in patients with newly diagnosed Type 1 Diabetes Mellitus (T1D).

Detailed Description

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The proposed study is a Phase I/IIa randomized, controlled clinical trial to evaluate CELZ-201 therapy as an intervention for the treatment of recent onset Type 1 Diabetes. The objective is to determine the safety and efficacy of CELZ-201 administration, based on the timing and dose of CELZ-201 treatment. Subjects who meet eligibility criteria will be randomized to treatment or control groups, in a 2:1 ratio. Subjects in the Group I (Treatment Group, n=12) will receive standard of care for type 1 diabetes and CELZ-201 within 1 month from enrollment (within 1 year of diagnosis). Subjects in Group II (Control Arm, n=6) and will receive enhanced standard of care for type 1 diabetes.

Conditions

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Type 1 Diabetes Diabetes Mellitus, Type 1

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

The study is a two-arm, open label, randomized trial. Subjects who meet eligibility criteria will be randomized to treatment or control groups in a 2:1 ratio.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CELZ-201 Treatment Group

Participants in this group will receive a single dose of CELZ-201, in addition to standard of care of care for Type 1 Diabetes treatment.

Group Type EXPERIMENTAL

CELZ-201 Administration

Intervention Type BIOLOGICAL

Participants in this group will receive a single dose of CELZ-201, in addition to standard of care for Type 1 Diabetes treatment. Perinatal tissue derived cells will be administered at a dose of 1x10\^6 cells/kg via an intra-arterial infusion into the dorsal pancreatic artery.

Control Group

Participants in this group will receive standard of care for Type 1 Diabetes only.

Group Type PLACEBO_COMPARATOR

Control Group

Intervention Type OTHER

Enhanced standard of care for Type 1 Diabetes treatment only.

Interventions

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CELZ-201 Administration

Participants in this group will receive a single dose of CELZ-201, in addition to standard of care for Type 1 Diabetes treatment. Perinatal tissue derived cells will be administered at a dose of 1x10\^6 cells/kg via an intra-arterial infusion into the dorsal pancreatic artery.

Intervention Type BIOLOGICAL

Control Group

Enhanced standard of care for Type 1 Diabetes treatment only.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Subject must be able to understand and provide signed informed consent.
2. Males and females, 18-35 years of age.
3. Diagnosis of T1D within 1 year, with stimulated C-peptide peak level \>0.6 ng/mL as assessed by 4-hour MMTT at the time of Visit 0 (screening).
4. Diagnosed with T1D, according to ADA standard criteria, and confirmed by positivity to at least two islet autoantibodies, GAD65, IA-2, or ZnT8.
5. Mentally stable and able to comply with the procedures of the study protocol
6. Subjects must be willing to comply with "standard-of-care" diabetes management.
7. Subjects with eGFR \>80 ml/min/1.73m2
8. Female subjects of childbearing potential must have a negative pregnancy test upon study entry.
9. Female (and male) subjects with reproductive potential must agree to use two FDA approved methods of birth control for the entire duration of the study.

Potential subjects of childbearing potential should agree to use effective contraception for the entire 2-year period.
10. Adequate venous access to support study required blood draws.

Exclusion Criteria

1. Inability or unwillingness of a subject to give written informed consent or comply with study protocol.
2. BMI\>28 kg/m.
3. HbA1c \> 9%
4. Subjects with poorly controlled hypertension as defined by systolic blood pressure \>140 mmHg or diastolic blood pressure \>90 mmHg.
5. Subjects with any history of cardiac disease, including but not limited to myocardial infarction, uncompensated heart failure, fluid overload, as well as any clinically significant abnormality identified on prior cardiac stress test, angiogram evaluation, or echocardiogram.
6. Subjects with liver disease, portal hypertension, any coagulopathy (including history of Factor V deficiency) or long-term anti-coagulant therapy (except low-dose aspirin). Other hepatic conditions including hepatic anatomic abnormalities or variants that would place the individual at increased risk in the judgment of the investigator are also considered exclusionary.
7. Symptomatic cholecystolithiasis; acute or chronic pancreatitis; or current symptomatic peptic ulcer disease.
8. Subjects with uncontrolled thyroid disease: thyroid stimulating hormone \<0.3 mU/L or \>5 mU/L; free T4 \<5.0 ug/dL or \>11.0 ug/dL.
9. Any of the following laboratory findings: hemoglobin \<11.5 g/dL (females) or \<13.2 g/dL (males); leukocytes \<3,000/μL; neutrophils \<1,500/μL; lymphocytes \<800/μL; platelets \<100,000/μL; elevation in AST and ALT \>2 x ULN (upper limit of normal); LDL cholesterol \>160; Triglycerides \>3 x ULN; total bilirubin \>1.5 x ULN.
10. Screening laboratory evidence consistent with significant chronic active infection (i.e.., hepatitis B and C, tuberculosis, and HIV), and IGRA Tuberculosis (Tb) test during screening
11. Ongoing acute infections, e.g., acute respiratory tract, urinary tract, or gastrointestinal tract infections.
12. Subjects with eating disorders.
13. Ongoing or anticipated use of diabetes medications other than insulin.
14. Current or ongoing use of non-insulin pharmaceuticals that affect glycemic control within 7 days of screening.
15. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 6 weeks of randomization.
16. Patients who have participated in previous clinical studies, other than observational studies, will be excluded.
17. Concomitant therapy with immunosuppressive drugs, immunomodulators, or cytotoxic agents, or previous therapy less than 3 months from randomization.
18. History or diagnosis of malignancy with the exception of a history of localized basal or squamous cell carcinoma.
19. Any history of gastroparesis or other severe gastrointestinal disease.
20. Presence of an allograft.
21. Diagnosed or self-reported drug or alcohol abuse.
22. An individual who has a medical, psychological or social condition that, in the opinion of the Principal Investigator, would interfere with safe and proper completion of the trial.
23. Pregnancy or ongoing breastfeeding for women; unwillingness or inability of both females and males of childbearing age to use a reliable and effective form of contraception, for the entire 2-year duration of the study.
24. Inability to perform any of the assessments required for endpoint analysis.
25. Known history of serious allergic reactions, including anaphylaxis to CELZ-201 or its preparation components. Specifically, patients with a prior history of heparin induced thrombocytopenia or any other adverse reaction to heparin, will be excluded.
26. The investigator believes that participating in the trial is not in the best interest of the patient, or the investigator considers patient unsuitable for enrollment (such as unpredictable risks or subject compliance issues).
27. Positive for coronavirus disease (COVID)-19 by PCR or evidence of active infection per local institutional standards.
28. Allergy to iodine contrast or anesthesia
29. For female subjects: Pregnant, nursing, or planning to become pregnant during the course of entire duration of the study (approximately little over two years) or unwillingness to comply with contraceptive requirements.
30. For male subjects: Male subjects with a female partner who is planning to become pregnant with the male subject during the entire course of the study or unwillingness to comply with contraceptive requirements.
Minimum Eligible Age

18 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Creative Medical Technology Holdings Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Camillo Ricordi, MD

Role: PRINCIPAL_INVESTIGATOR

University of Miami, Diabetes Research Institute

Locations

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Diabetes Research Institute, University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Creative Medical Technology

Role: CONTACT

(702) 588-1890

Facility Contacts

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Camillo Ricordi, MD

Role: primary

(305) 243-5321

Other Identifiers

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CELZ-201-001

Identifier Type: -

Identifier Source: org_study_id

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