Trial Outcomes & Findings for Islet Transplantation in Type 1 Diabetics Using the Edmonton Protocol of Steroid Free Immunosuppression (NCT NCT00133809)

NCT ID: NCT00133809

Last Updated: 2016-07-18

Results Overview

Independence from insulin injections is measured by the actual use of insulin by the study participants.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 8 participants
• Primary outcome timeframe: one year after transplant
• Results posted on: 2016-07-18

Participant Flow

Ten participants fulfilled entry criteria and were listed for islet transplantation. Prior to receiving the transplant, one of the participants became ineligible while on the organ donor waiting list due to glucose control being stabilized with an insulin pump, and an additional participant was lost to follow up.

Participant milestones

Measure	Islet Transplant All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Islet Transplantation in Type 1 Diabetics Using the Edmonton Protocol of Steroid Free Immunosuppression

Baseline characteristics by cohort

Measure	Islet Transplant n=8 Participants 10 subjects were found eligible and were can be matched to an appropriate donor will receive/have received an islet transplant---1 INELIGIBLE WHILE ON WAITLIST. Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants

PRIMARY outcome

Timeframe: one year after transplant

Independence from insulin injections is measured by the actual use of insulin by the study participants.

Outcome measures

Measure	Islet Transplant n=8 Participants All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
The Number of Insulin-Independent Subjects at One Year Following Islet Cell Transplantation	5 participants

SECONDARY outcome

Timeframe: 1, 3, 6, 9,12,18, 24, 36, 48 and 60 months post-transplantation

Participants who did not need to take insulin at 1, 3, 6, 12, 18, 24, 36, 48 and 60 months following islet transplantation

Outcome measures

Measure	Islet Transplant n=8 Participants All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
Number of Insulin-independent Subjects Following Islet Transplantation 1 Month Post-Transplant	6 participants
Number of Insulin-independent Subjects Following Islet Transplantation 3 Months Post-Transplant	6 participants
Number of Insulin-independent Subjects Following Islet Transplantation 6 Months Post-Transplant	6 participants
Number of Insulin-independent Subjects Following Islet Transplantation 9 Months Post-Transplant	6 participants
Number of Insulin-independent Subjects Following Islet Transplantation 12 Months Post-Transplant	5 participants
Number of Insulin-independent Subjects Following Islet Transplantation 18 Months Post-Transplant	5 participants
Number of Insulin-independent Subjects Following Islet Transplantation 24 Months Post-Transplant	5 participants
Number of Insulin-independent Subjects Following Islet Transplantation 36 Months Post-Transplant	3 participants
Number of Insulin-independent Subjects Following Islet Transplantation 48 Months Post-Transplant	2 participants
Number of Insulin-independent Subjects Following Islet Transplantation 60 Months Post-Transplant	2 participants

SECONDARY outcome

Timeframe: 1, 3, 6, 9,12,18,24, 36, 48 and 60 months post-transplantation

HbA1C was assessed in subjects 1, 3, 6, 9,12,18,24, 36, 48 and 60 months after transplantation and the number of subjects with values ≤ 6.5% was recorded which indicated better control of blood glucose levels.

Outcome measures

Measure	Islet Transplant n=8 Participants All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
Number of Subjects With HbA1C ≤ 6.5% 1 Month Post-Transplant	6 participants
Number of Subjects With HbA1C ≤ 6.5% 3 Months Post-Transplant	7 participants
Number of Subjects With HbA1C ≤ 6.5% 6 Months Post-Transplant	8 participants
Number of Subjects With HbA1C ≤ 6.5% 9 Months Post-Transplant	8 participants
Number of Subjects With HbA1C ≤ 6.5% 12 Months Post-Transplant	8 participants
Number of Subjects With HbA1C ≤ 6.5% 18 Months Post-Transplant	8 participants
Number of Subjects With HbA1C ≤ 6.5% 24 Months Post-Transplant	7 participants
Number of Subjects With HbA1C ≤ 6.5% 36 Months Post-Transplant	6 participants
Number of Subjects With HbA1C ≤ 6.5% 48 Months Post-Transplant	5 participants
Number of Subjects With HbA1C ≤ 6.5% 60 Months Post-Transplant	4 participants

SECONDARY outcome

Timeframe: 1, 3, 6, 9,12,18, 24, 36, 48 and 60 months post-transplantation

Number of Participants With Endogenous Insulin Production Post-transplant, Assessed by Fasting C-peptide Levels at 1, 3, 6, 9,12,18, 24, 36, 48 and 60 months after islet cell transplantation

Outcome measures

Measure	Islet Transplant n=8 Participants All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 1 Month Post-Transplant	6 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 3 Months Post-Transplant	6 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 6 Months Post-Transplant	6 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 9 Months Post-Transplant	6 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 12 Months Post-Transplant	5 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 18 Months Post-Transplant	5 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 24 Months Post-Transplant	5 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 36 Months Post-Transplant	5 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 48 Months Post-Transplant	3 participants
The Number of Subjects Exhibiting Fasting C-peptide Levels ≥ 0.5 ng/mL 60 Months Post-Transplant	3 participants

Adverse Events

Islet Transplant

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	Islet Transplant n=8 participants at risk All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
Gastrointestinal disorders Hospitalization for diarrhea, nausea, vomiting, and abdominal pain	12.5% 1/8 • Number of events 1 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.
Infections and infestations Pneumonia	12.5% 1/8 • Number of events 1 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.

Other adverse events

Measure	Islet Transplant n=8 participants at risk All subjects who are found eligible and who can be matched to an appropriate donor will receive/have received an islet transplant Transplantation of Human Islets: Human islets, at least 9,000 islet equivalents per kilogram of body weight. Transplant involves surgical procedure
Blood and lymphatic system disorders Anemia	87.5% 7/8 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.
Gastrointestinal disorders Diarrhea	87.5% 7/8 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.
Hepatobiliary disorders Elevated Liver Enzymes	100.0% 8/8 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.
Blood and lymphatic system disorders Leukopenia	100.0% 8/8 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.
Skin and subcutaneous tissue disorders Mouth Ulcers	100.0% 8/8 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.
Gastrointestinal disorders Abdominal Hernia	62.5% 5/8 • Adverse event data were collected for as long as the study participants were willing to be followed for safety monitoring. The follow up time ranged from 3 years to 5 years and 9 months.

Additional Information

Nicole Turgeon MD

Emory University

Phone: 404-727-3257

Email: nturgeo@emory.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place