Islet Cell Transplant for Type 1 Diabetes

NCT01909245 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 12446
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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

Keywords

Islet cells; hypoglycemia; hypoglycemia unawareness; islet after kidney transplantation; islet transplant; insulin independence; insulin dependence; labile diabetes

Outcome Measures

Primary Outcomes (3)

• Proportion of subjects who are insulin independent, hypoglycemia free, AND with hemoglobin A1c < or = 6.5% at 1 year post-transplant

  1 year post-transplant

• Proportion of subjects who are insulin independent, hypoglycemia free, AND with hemoglobin A1c < or = 6.5% at 2 years post-transplant

  2 years post-transplant

• Proportion of subjects who are insulin independent, hypoglycemia free, AND with hemoglobin A1c < or = 6.5% at 5 years post-transplant

  5 years post-transplant

Secondary Outcomes (1)

• Proportion of subjects who are free of severe hypoglycemic episodes AND have a hemoglobin A1c < or = 7.0%

  +75 days, +6 months, +12 months, and +2, +3, +4 and +5 years post islet transplant

Other Outcomes (16)

• Proportion of subjects experiencing reduction/elimination of hypoglycemic episodes

  +75 days, +6 months, +12 months, and +2, +3, +4 and +5 years post islet transplant

• Duration of insulin independence

  +75 days, +6 months, +12 months, and +2, +3, +4 and +5 years post islet transplant

• Proportion of subjects who maintain a positive c-peptide secretion response to glucose/glucagon stimulation

  +75 days, +6 months, +12 months, and +2, +3, +4 and +5 years post islet transplant

Study Arms (1)

Single Arm Study

EXPERIMENTAL

Allogenic Human Islet Cell Transplant with immunosuppression

Biological: Allogenic Human Islet Cells; Drug: Immunosuppressive Agents; Drug: Gastrin 17

Interventions

Allogenic Human Islet Cells BIOLOGICAL

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

Also known as: Islet transplant, islet transplantation

Single Arm Study

Immunosuppressive Agents 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.

Single Arm Study

Gastrin 17 DRUG

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

Also known as: GAST-17

Single Arm Study

Eligibility Criteria

• Age: 18 Years - 68 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18-68 years
• Type 1 diabetes mellitus for at least 5 years
• 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
• 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
• One or two or previous islet transplants \> 1 month prior to screening with continuing insulin requirements and/or HbA1c \> 6.5%
• Successful kidney transplant \> 3 months prior to screening
• 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
• No history of acute rejection related to kidney graft in last 12 months and low risk of rejection
• Under continuing care of physician for kidney graft, who has provided letter in support of candidate's consideration for study participation

You may not qualify if:

• Body Mass Index (BMI) \> 33
• Insulin requirements \> 1.2 units/kg/day
• Known sensitization to both rATG -and- alemtuzumab
• Significant kidney dysfunction
• Significant liver/gall bladder disease
• Significant cardiovascular disease
• Active proliferative retinopathy
• High blood pressure despite appropriate treatment
• High cholesterol/triglycerides despite appropriate treatment
• Anemia or other blood disorders that require medical treatment
• WBC \<3,000/ul
• Increased risk of bleeding, other chronic hemostasis disorders, or treatment with chronic anticoagulant therapy
• Recent unresolved acute infection (except for mild skin infection or nail fungal infection), or chronic infection
• Epstein-Barr Virus (EBV) IgG negative
• Any history of malignancy, except for completely resected squamous or basal cell carcinoma of the skin or in situ cancer of the cervix

Sponsors & Collaborators

• City of Hope Medical Center: lead
• University of California, Los Angeles: collaborator

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91010, United States

Location

Related Publications (1)

• 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.

  PMID: 29506054 RESULT

Related Links

• City of Hope Islet Cell Transplantation Program

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Hypoglycemia

Interventions

Islets of Langerhans Transplantation; Immunosuppressive Agents; gastrin 17

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Endocrine Surgical Procedures; Surgical Procedures, Operative; Transplantation; Immunologic Factors; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Study Officials

• Fouad Kandeel, MD, PhD

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 22, 2013
• First Posted: July 26, 2013
• Study Start: October 16, 2013
• Primary Completion (Estimated): September 22, 2026
• Study Completion (Estimated): September 22, 2026
• Last Updated: November 10, 2025

Record last verified: 2025-11

Locations

US (1)