Islet Cell Transplant for Type 1 Diabetes

NCT ID: NCT01909245

Last Updated: 2025-11-10

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-10-16
• Study Completion Date: 2026-09-22

Brief Summary

City of Hope National Medical Center, located in Duarte, CA, is hosting a clinical study on islet cell transplantation, an experimental procedure being evaluated as a treatment for patients with type 1 diabetes. Islet cell transplantation involves taking insulin-producing cells from organ donors and transplanting them into the liver of a patient with diabetes. Once transplanted, the islets produce insulin, which can improve blood sugar control and eliminate the need to inject insulin or use an insulin pump.

Anti-thymocyte globulin (ATG) and alemtuzumab (Campath) are anti-rejection medications that work by decreasing a patient's T-cells. T-cells are special white blood cells that recognize and destroy unwanted things like infections but can also attack transplanted cells and organs. Reducing the number of T-cells at the time of transplant may protect islets and improve long-term transplant success. In previous research studies, islet transplantation has been successful in reducing low blood sugar episodes, improving overall blood sugar control, and in some cases, allowing patients with type 1 diabetes to stop taking insulin.

The purpose of this study is to determine if islet cell transplantation using ATG or alemtuzumab, along with additional medications to prevent the body from rejecting the transplanted cells, is a safe and effective treatment for type 1 diabetes. Study participants may receive up to three islet transplants and will be followed for five years to monitor blood sugar control, islet transplant function, and changes in quality of life.

Conditions

Type 1 Diabetes Mellitus

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Single Arm Study

Allogenic Human Islet Cell Transplant with immunosuppression

Group Type: EXPERIMENTAL

Allogenic Human Islet Cells

Intervention Type: BIOLOGICAL

Intraportal (into the liver) infusion of islet cells, with a maximum of three islet transplants.

Immunosuppressive Agents

Intervention Type: DRUG

Anti-rejection medications (to prevent the body from rejecting the islet cells) and other medications to guard against infection and support participant health and/or the health of the transplanted islets.

Gastrin 17

Intervention Type: DRUG

Gastrin-17 (or GAST-17) - a gut hormone injected under the skin for 30 days (optional treatment for islet dysfunction).

Interventions

Allogenic Human Islet Cells

Intraportal (into the liver) infusion of islet cells, with a maximum of three islet transplants.

Intervention Type: BIOLOGICAL

Immunosuppressive Agents

Anti-rejection medications (to prevent the body from rejecting the islet cells) and other medications to guard against infection and support participant health and/or the health of the transplanted islets.

Intervention Type: DRUG

Gastrin 17

Gastrin-17 (or GAST-17) - a gut hormone injected under the skin for 30 days (optional treatment for islet dysfunction).

Intervention Type: DRUG

Other Intervention Names

Islet transplant, islet transplantation; GAST-17

Eligibility Criteria

Inclusion Criteria

1. Age 18-68 years

2. Type 1 diabetes mellitus for at least 5 years

3. Ability and willingness to comply with post-transplant regimen, including taking anti-rejection medications, use of reliable contraception, frequent clinic visits, lab tests, careful recording of blood glucose values, insulin doses and medications, and completing detailed follow-up studies

4. Unstable blood sugar control characterized by:

Frequent hypoglycemia (blood glucose ≤ 54 mg/dl more than once per week) -AND/OR- Hypoglycemia unawareness (Clarke score of 4 or more) -AND/OR- One or more severe hypoglycemic episodes in 12 months preceding enrollment. -AND/OR- Erratic blood glucose levels that interfere with daily activities -AND/OR- One or more hospital visits for diabetic ketoacidosis in the 12 months preceding enrollment

5. One or two or previous islet transplants > 1 month prior to screening with continuing insulin requirements and/or HbA1c > 6.5%

6. Successful kidney transplant > 3 months prior to screening

7. Stable maintenance immunosuppression consisting of tacrolimus alone or in conjunction with sirolimus, mycophenolate mofetil, myfortic or azathoprine; or cyclosporine in conjunction with sirolimus, mycophenolate mofetil, or myfortic +/- ≤ 10 mg/day corticosteroids

8. No history of acute rejection related to kidney graft in last 12 months and low risk of rejection

9. Under continuing care of physician for kidney graft, who has provided letter in support of candidate's consideration for study participation

Exclusion Criteria

1. Body Mass Index (BMI) > 33

2. Insulin requirements > 1.2 units/kg/day

3. Known sensitization to both rATG -and- alemtuzumab

4. Significant kidney dysfunction

5. Significant liver/gall bladder disease

6. Significant cardiovascular disease

7. Active proliferative retinopathy

8. High blood pressure despite appropriate treatment

9. High cholesterol/triglycerides despite appropriate treatment

10. Anemia or other blood disorders that require medical treatment

11. WBC <3,000/ul

12. Increased risk of bleeding, other chronic hemostasis disorders, or treatment with chronic anticoagulant therapy

13. Recent unresolved acute infection (except for mild skin infection or nail fungal infection), or chronic infection

14. Epstein-Barr Virus (EBV) IgG negative

15. Any history of malignancy, except for completely resected squamous or basal cell carcinoma of the skin or in situ cancer of the cervix

16. Recent history of non-adherence to medical treatment, or inability to demonstrate capacity to comply with strict blood glycemic control and insulin therapy

17. Psychiatric illness that is untreated, or likely to interfere significantly with study compliance despite treatment

18. Previous organ/tissue transplant, except as noted above

19. Administration of live attenuated vaccines within 2 months of enrollment

20. Presence of a chronic disease that must be chronically treated with a contraindicated agent

21. Use of investigational agents within four weeks of enrollment

22. Active alcohol or substance abuse, including cigarette smoking

23. Pregnant women, women intending future pregnancy, women of reproductive potential who are unable or unwilling to follow effective contraceptive measures prior to study entry and for as long as they are on immunosuppression medication, and women presently breast feeding are excluded

24. Individuals without health insurance

25. History of gastric bypass

26. Any medical condition that in the opinion of the investigator will interfere with safe participation in the trial

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 68 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of California, Los Angeles

OTHER

Sponsor Role: collaborator

City of Hope Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Fouad Kandeel, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

City of Hope Medical Center

Locations

City of Hope Medical Center

Duarte, California, United States

Countries

United States

References

Aslamy A, Oh E, Ahn M, Moin ASM, Chang M, Duncan M, Hacker-Stratton J, El-Shahawy M, Kandeel F, DiMeglio LA, Thurmond DC. Exocytosis Protein DOC2B as a Biomarker of Type 1 Diabetes. J Clin Endocrinol Metab. 2018 May 1;103(5):1966-1976. doi: 10.1210/jc.2017-02492.

Reference Type: RESULT

PMID: 29506054 (View on PubMed)

Related Links

http://www.cityofhope.org/islets

City of Hope Islet Cell Transplantation Program

Other Identifiers

12446

Identifier Type: -

Identifier Source: org_study_id