Trial Outcomes & Findings for Raptiva and Sirolimus in Islet Transplantation for Type 1 Diabetes (NCT NCT00672204)

Results Overview

Islet transplant recipients will be considered insulin-independent with full islet graft function if they are able to titrate off insulin therapy for at least 1 week and all of the following criteria are met: * HbA1c \< 7.0% or a ≥2.5% decrease from baseline; * fasting capillary glucose level should not exceed 140 mg/dL (7.8 mmol/L) more than three times in the past week (based on measuring capillary glucose levels a minimum of 7 times in a seven day period); * 2-hour post-prandial capillary glucose should not exceed 180 mg/dl (10.0 mmol/L) more than three times in the past week (based on measuring capillary glucose levels a minimum of 21 times in a seven day period); * fasting serum glucose level ≤126 mg/dL (7.0 mmol/L); if the fasting serum glucose level is \>126 mg/dL (7.0 mmol/L), it must be confirmed in an additional one out of two measurements; * evidence of endogenous insulin production defined as fasting or stimulated C-peptide levels ≥0.5 ng/mL (0.16 nmol/L).

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

23 participants

Primary outcome timeframe

1 year following the first islet transplant

Results posted on

2017-10-25

Participant Flow

Recruited from 2008 - 2009 at the University of Minnesota.

Raptiva was removed from market.

Participant milestones

Participant milestones
Measure	Allogeneic Islets of Langerhans Allogeneic islets of Langerhans anti-thymocyte globulin : 2.0 mg/kg on days -2, and -1 IV Raptiva : Treatment Day -1 pretransplant to Treatment Day 90 after tx.: 1.0 mg/kg/wk SQ; Treatment Day 91 to Treatment Day 365: 0.5 mg/kg/wk SQ; Allogeneic islets of Langerhans transplant : Up to 3 intraportal infusions of cadaveric pancreatic islets of Langerhans. Each infusion to contain at least 5,000 islet equivalents/kg body weight. Sirolimus : Initial dose 0.1 mg/kg PO on day -2, followed by 0.05 mg/kg daily, whole blood 24-hour trough adjusted to target 3-15 ng/ml as tolerated
Overall Study STARTED	23
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	20

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Raptiva and Sirolimus in Islet Transplantation for Type 1 Diabetes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Allogeneic Islets of Langerhans n=23 Participants Allogeneic islets of Langerhans anti-thymocyte globulin : 2.0 mg/kg on days -2, and -1 IV Raptiva : Treatment Day -1 pretransplant to Treatment Day 90 after tx.: 1.0 mg/kg/wk SQ; Treatment Day 91 to Treatment Day 365: 0.5 mg/kg/wk SQ; Allogeneic islets of Langerhans transplant : Up to 3 intraportal infusions of cadaveric pancreatic islets of Langerhans. Each infusion to contain at least 5,000 islet equivalents/kg body weight. Sirolimus : Initial dose 0.1 mg/kg PO on day -2, followed by 0.05 mg/kg daily, whole blood 24-hour trough adjusted to target 3-15 ng/ml as tolerated
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	23 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	43.1 years STANDARD_DEVIATION 9.4 • n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Region of Enrollment United States	23 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year following the first islet transplant

Population: Raptiva was removed from market after 3 subjects were transplanted

Islet transplant recipients will be considered insulin-independent with full islet graft function if they are able to titrate off insulin therapy for at least 1 week and all of the following criteria are met: * HbA1c \< 7.0% or a ≥2.5% decrease from baseline; * fasting capillary glucose level should not exceed 140 mg/dL (7.8 mmol/L) more than three times in the past week (based on measuring capillary glucose levels a minimum of 7 times in a seven day period); * 2-hour post-prandial capillary glucose should not exceed 180 mg/dl (10.0 mmol/L) more than three times in the past week (based on measuring capillary glucose levels a minimum of 21 times in a seven day period); * fasting serum glucose level ≤126 mg/dL (7.0 mmol/L); if the fasting serum glucose level is \>126 mg/dL (7.0 mmol/L), it must be confirmed in an additional one out of two measurements; * evidence of endogenous insulin production defined as fasting or stimulated C-peptide levels ≥0.5 ng/mL (0.16 nmol/L).

Outcome measures

Outcome measures
Measure	Allogeneic Islets of Langerhans n=3 Participants Allogeneic islets of Langerhans anti-thymocyte globulin : 2.0 mg/kg on days -2, and -1 IV Raptiva : Treatment Day -1 pretransplant to Treatment Day 90 after tx.: 1.0 mg/kg/wk SQ; Treatment Day 91 to Treatment Day 365: 0.5 mg/kg/wk SQ; Allogeneic islets of Langerhans transplant : Up to 3 intraportal infusions of cadaveric pancreatic islets of Langerhans. Each infusion to contain at least 5,000 islet equivalents/kg body weight. Sirolimus : Initial dose 0.1 mg/kg PO on day -2, followed by 0.05 mg/kg daily, whole blood 24-hour trough adjusted to target 3-15 ng/ml as tolerated
The Proportion of Insulin-independent Subjects With Full Islet Graft Function	3 participants

Adverse Events

Allogeneic Islets of Langerhans

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Bernhard J. Hering, M.D.

University of Minnesota

Phone: 612-626-5735

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place