A Phase 3 interventional study of Islets of Langerhans transplantation in Type 1 Diabetes Mellitus, sponsored by CellTrans Inc.. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-04-06.
Sponsored by CellTrans Inc. · Phase 3, Interventional, and Treatment
In an earlier Phase 1/2 clinical trial using the Edmonton Protocol of steroid free immunosuppression, investigators at University of Illinois at Chicago (UIC) demonstrated the safety of islet preparation, iset transplantation, and medical treatment at UIC. Therefore, the primary purpose of the present Phase 3 clinical trial is to demonstrate the safety and efficacy of allogeneic islet transplantation in improving glycemic control in Type 1 diabetic patients using the UIC protocol that was developed and proven effective during the Phase 1/2 clinical trial.
This study is a Phase 3 single center, uncontrolled trial in which 1-3 allogeneic pancreatic islet transplants are performed for each study subject. Follow-up evaluations after transplant continue for 52 weeks after the final islet transplantation. Thereafter, subjects may enroll for a 5-year follow-up study and an additional 5 year to 10 year follow-up study to evaluate the function of the islets and to measure and regulate immunosuppressive drug levels and side effects.
The safety of islet transplantation depends primarily on the incidence of serious and unexpected complications or adverse events and the ability of the cell isolation laboratory to produce uncontaminated islet cell preparations with minimal endotoxin content.
All study subjects are followed for safety for one year. An independent Data Monitoring Committee (DMC), composed of 3 members who have training in medicine and/or organ transplantation, will review eligibility and safety data within 2 weeks after each islet transplantation and every two months thereafter. An independent monitor, who is knowledgeable about Good Clinical Practice (GCP) guidelines and regulations, monitors the study for compliance with 21 CFR and according to ICH GCP Guidelines. Within the Clinical Research Center, representatives of the Scientific Advisory Committee and the Research Subject Advocacy Program monitor safety. These entities report to the UIC Institutional Review Board (IRB), which also reviews safety data annually and on occurrence of serious adverse events. The principal investigator also reports serious adverse events to the US Food and Drug Administration (FDA).
Success: Islet transplantation is considered a success when subjects do not use insulin, and they achieve a fasting glucose level not exceeding 140 mg/dL more than three times in a week, and not exceeding two-hour post-prandial values of 180 mg/dL more than four times in a week.
Partial Success: Subjects who have a reduction in insulin requirements but who do not achieve insulin independence and present with a reduction in HbA1c and number of hypoglycemic episodes are considered to have partial success of islet transplantation. Reduction in insulin-requirements are assessed by comparing the pre-transplant insulin requirement recorded over two consecutive days (expressed as insulin units per kg) with the requirement on the two consecutive days preceding the subsequent islet infusion, and the requirements on two consecutive days at six months and again on two consecutive days at one year after the final transplant.
Failure: Absence of measurable levels of C-peptide after transplantation is considered as failure of islet cell transplantation.
3,521 studies on the registry are indexed under Diabetes Mellitus, Type 1; 576 are open to participants now.
This study's enrollment of 21 is below the median of 40 across 2,648 interventional studies indexed under Diabetes Mellitus, Type 1.
Browse Diabetes Mellitus, Type 1 studies →CellTrans Inc. is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
All subjects will receive up to 3 transplantations of allogeneic human islets of Langerhans.
Biological: Islets of Langerhans transplantation
Each subject may receive 1-3 transplantations of allogeneic human islets of Langerhans and the following medications: Basiliximab 20 mg iv 2 hours before transplant and 20 mg iv 2 weeks post-transplant; Tacrolimus 1 mg p.o. bid adjusted to reach target trough levels of 3-6 ng/ml; Sirolimus 0.2 mg/kg loading dose, then 0.1 mg/kg p.o. daily adjusted to reach target trough levels of 10-15 ng/ml during the first 3 months post transplant and 7-10 ng/ml thereafter; Etanercept 50 mg iv 1 hour before transplant and 25 mg s.c. on days 3, 7,and 10 post-transplant; Exenatide 5-mcg s.c. bid for 1 week, then 10 mcg bid for 6 months after each transplant
Also known as: Islets of Langerhan (Islets), Basiliximab (Simulect®), Tacrolimus (Prograf®), Sirolimus (Rapamune®), Etanercept(Enbrel®), Exenatide (Byetta®)
Treatment Emergent Adverse Events
Safety endpoints: Incidence and severity of events related to islet infusion, immunosuppression, and islet preparations
Time frame: From first islet transplant through one year after last transplant (maximum 3 infusions possible), an average of 1 year
Number of Subjects Reaching the Efficacy Goal
A successful primary endpoint was defined as HbA1c ≤ 6.5% at the one-year follow-up visit and absence of severe hypoglycemic events (SHE) from Day 28 post-first transplant to 1 year after first and last transplant. The primary analysis was to estimate the true rate of the composite favorable outcome at 1 year following first and last transplant in patients in the ITT population.
Time frame: One year after islet transplant
Number of Patients Presenting With Insulin Independence at Day 365 Post First and Last Transplant
Number of patients presenting with insulin independence, including: Absence of exogenous insulin injection reported at Day 365. * Fasting capillary glucose level not exceeding 140 mg/dL (7.8 mmol/L) more than 3 times in a week (based on measuring capillary glucose levels a minimum of 7 times in a 7-day period) at Day 365 ± 28 days. * Fasting plasma glucose level ≤ 126 mg/dL (7.0 mmol/L) at Day 365 ± 28 days (if the fasting plasma glucose level is \> 126 mg/dL \[7.0 mmol/L\], it must have been confirmed in an additional 1 out of 2 measurements). * Two-hour post-prandial capillary glucose not exceeding 180 mg/dL (10.0 mmol/L) more than 1 out of every 7 times in a week (based on measuring capillary glucose levels a minimum of 7 times in a 7-day period) at Day 365 ± 28 days. * Evidence of endogenous insulin production defined as fasting or stimulated C-peptide levels ≥ 0.5 ng/mL (0.16 nmol/L) at Day 365 ± 28 days
Time frame: 1 year after islet infusion
Hypoglycemic Episodes by HYPO Score
Hypoglycemic episodes will be measured by the Ryan hypoglycemic (HYPO) Score derived from the number and severity of hypoglycemic episodes recorded throughout the follow-up phase from Day 28 to Day 365. (From Ryan et al., 2004) "A HYPO score was generated based on a combination of scores from the 4 weeks of readings and the patients' self-reported episodes over the previous year using the scoring system found in online appendix 2 (available at http://diabetes.diabetesjournals.org). The record sheets returned by the patients were analyzed for the number of episodes of glucose values recorded as \<2.5 mmol/l and between 2.5 and 2.9 mmol/l. Points were awarded if symptoms were absent or were neuroglycopenic rather than autonomic... Thus the more severe the problem with hypoglycemia, the higher the score." A Ryan score ranges from 0 (no event) to a cumulative sum of episode points of total events reported during the 4 weeks then multiplied by 13 to provide a 1-year value.
Time frame: One year after the last transplant
Reduction in Hypoglycemic Severity Measured by %Reduction in HYPO Score
%reduction in Ryan HYPO Score \[%(baseline score - 1-year post transplant score)/baseline\] at time of evaluation.
Time frame: One year after the first and last transplant
Potentially eligible patients with diabetes underwent a two-part screening phase to determine eligibility, followed by a waiting list period (as needed). The first subject consented on September 5, 2007. The last subject consented on November 4, 2014.
| Milestone | Treatment |
|---|---|
| Started | 21 |
| Completed | 19 |
| Not completed | 2 |
| Withdrew: Withdrawal by subject | 2 |
| Milestone | Treatment |
|---|---|
| Started | 19 |
| Completed | 5 |
| Not completed | 14 |
| Withdrew: In follow-up (ongoing) | 6 |
| Withdrew: Death | 1 |
| Withdrew: Did not consent to continue into 10-year follow up | 7 |
| Milestone | Treatment |
|---|---|
| Started | 5 |
| Completed | 0 |
| Not completed | 5 |
| Withdrew: In follow-up (ongoing) | 5 |
Safety endpoints: Incidence and severity of events related to islet infusion, immunosuppression, and islet preparations
| Participants | Treatment |
|---|---|
| TEAE | 21 |
| Serious TEAE | 11 |
| TEAEs rated as severe or beyond | 16 |
| TEAEs leading to death or discontinuation | 0 |
A successful primary endpoint was defined as HbA1c ≤ 6.5% at the one-year follow-up visit and absence of severe hypoglycemic events (SHE) from Day 28 post-first transplant to 1 year after first and last transplant. The primary analysis was to estimate the true rate of the composite favorable outcome at 1 year following first and last transplant in patients in the ITT population.
| Participants | Treatment |
|---|---|
| Success at 1 yr post first transplant | 8 |
| Success at 1 yr post last transplant | 11 |
Number of patients presenting with insulin independence, including: Absence of exogenous insulin injection reported at Day 365. * Fasting capillary glucose level not exceeding 140 mg/dL (7.8 mmol/L) more than 3 times in a week (based on measuring capillary glucose levels a minimum of 7 times in a 7-day period) at Day 365 ± 28 days. * Fasting plasma glucose level ≤ 126 mg/dL (7.0 mmol/L) at Day 365 ± 28 days (if the fasting plasma glucose level is \> 126 mg/dL \[7.0 mmol/L\], it must have been confirmed in an additional 1 out of 2 measurements). * Two-hour post-prandial capillary glucose not exceeding 180 mg/dL (10.0 mmol/L) more than 1 out of every 7 times in a week (based on measuring capillary glucose levels a minimum of 7 times in a 7-day period) at Day 365 ± 28 days. * Evidence of endogenous insulin production defined as fasting or stimulated C-peptide levels ≥ 0.5 ng/mL (0.16 nmol/L) at Day 365 ± 28 days
| Participants | Treatment |
|---|---|
| First Tx Day 365 Absence of exogenous insulin | 10 |
| Last Tx Day 365 Absence of exogenous insulin | 12 |
| First Tx Day 365 Fasting capillary glucose in a week not exceeding 140 mg/dL more than 3X | 7 |
| Last Tx Day 365 Fasting capillary glucose in a week not exceeding 140 mg/dL more than 3X | 4 |
| First Tx Day 365 Fasting plasma glucose ≤126 mg/dL | 10 |
| Last Tx Day 365 Fasting plasma glucose ≤126 mg/dL | 11 |
| First Tx Day 365 Post-prandial capillary glucose in a week: Not exceeding 180 mg/dL more than 1X/7X | 5 |
| Last Tx Day 365 Post-prandial capillary glucose in a week: Not exceeding 180 mg/dL more than 1X/7X | 3 |
| First Tx Day 365 C-peptide (fasting or stimulated) ≥0.5 ng/mL | 10 |
| Last Tx Day 365 C-peptide (fasting or stimulated) ≥0.5 ng/mL | 10 |
Hypoglycemic episodes will be measured by the Ryan hypoglycemic (HYPO) Score derived from the number and severity of hypoglycemic episodes recorded throughout the follow-up phase from Day 28 to Day 365. (From Ryan et al., 2004) "A HYPO score was generated based on a combination of scores from the 4 weeks of readings and the patients' self-reported episodes over the previous year using the scoring system found in online appendix 2 (available at http://diabetes.diabetesjournals.org). The record sheets returned by the patients were analyzed for the number of episodes of glucose values recorded as \<2.5 mmol/l and between 2.5 and 2.9 mmol/l. Points were awarded if symptoms were absent or were neuroglycopenic rather than autonomic... Thus the more severe the problem with hypoglycemia, the higher the score." A Ryan score ranges from 0 (no event) to a cumulative sum of episode points of total events reported during the 4 weeks then multiplied by 13 to provide a 1-year value.
| score on a scale | Treatment |
|---|---|
| Baseline (pre-Tx) | 266 (2 to 1638) |
| First Tx Day 365 | 40.6 (0 to 1234) |
| Last Tx Day 365 | 18.7 (0 to 1234) |
%reduction in Ryan HYPO Score \[%(baseline score - 1-year post transplant score)/baseline\] at time of evaluation.
| percentage of HYPO baseline | Treatment |
|---|---|
| % Reduction from baseline to first tx day 365 | 66.1 (-300 to 100) |
| % Reduction from baseline to last tx day 365 | 90.1 (-106 to 100) |
Collected over From first islet transplant through one year after last transplant (maximum 3 infusions possible), an average of 1 year.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment | 0/21 (0%) | 11/21 (52.4%) | 21/21 (100%) |
| Event | Treatment |
|---|---|
| PneumoniaInfections and infestations | 2/21 |
| Abdominal painGastrointestinal disorders | 1/21 |
| Intra-abdominal haemorrhageGastrointestinal disorders | 1/21 |
| Nausea/VomitingGastrointestinal disorders | 1/21 |
| AnaemiaBlood and lymphatic system disorders | 1/21 |
| PancytopeniaBlood and lymphatic system disorders | 1/21 |
| Myocardial ischaemiaCardiac disorders | 1/21 |
| HypoglycaemiaEndocrine disorders | 1/21 |
| AstheniaGeneral disorders | 1/21 |
| CholecystitisHepatobiliary disorders | 1/21 |
| Event | Treatment |
|---|---|
| VomitingGastrointestinal disorders | 17/21 |
| fatigueGeneral disorders | 16/21 |
| anemiaBlood and lymphatic system disorders | 15/21 |
| astheniaGeneral disorders | 14/21 |
| acneSkin and subcutaneous tissue disorders | 14/21 |
| Abnormal loss of weightMetabolism and nutrition disorders | 13/21 |
| diarrheaGastrointestinal disorders | 13/21 |
| HeadacheNervous system disorders | 12/21 |
| Transaminases increasedInvestigations | 10/21 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 10/21 |
| Age, Continuous(years) | Treatment |
|---|---|
| Median | 47.0 (21 to 67) |
| Sex: Female, Male(Participants) | Treatment |
|---|---|
| Female | 15 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | Treatment |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 20 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Treatment |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 20 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Treatment |
|---|---|
| United States | 21 |
| weight(kg) | Treatment |
|---|---|
| Median | 63.8 (52.5 to 83.4) |
| height(cm) | Treatment |
|---|---|
| Median | 165 (150.9 to 181.9) |
| BMI(kg/m^2) | Treatment |
|---|---|
| Mean | 23.4 ± 2 |
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