Trial Outcomes & Findings for Pancreatic Islet Cell Transplantation (NCT NCT00214786)

Results Overview

To assess the number of patients who achieve insulin independence at 12-month after islet cell transplantation

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

4 participants

Primary outcome timeframe

12 months post transplant

Results posted on

2017-06-14

Participant Flow

Recruitment period took place between April 2003 thru November 2005. Consent took place in the transplant clinic at Baylor University Medical Center (BUMC).

Patients signed informed consent after a consultation visit with tyhe PI. Then they proceeded through the evaluation phase of the study. During this phase, some patients were excluded based off of clinical findings (i.e. lab reports or procedures that did not meet protocol standards).

Participant milestones

Participant milestones
Measure	Islet Cell Transplantation Patients who received islet cell transplantation. The recipients will be given islet cell preparation with more than 4000 Islet Equivalent (IE)/kg for multiple times up to 3 infusions.
Overall Study STARTED	4
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pancreatic Islet Cell Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Islet Cell n=4 Participants Patients with allogeneic islet cell transplantation
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	35.5 years STANDARD_DEVIATION 11.56143 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months post transplant

To assess the number of patients who achieve insulin independence at 12-month after islet cell transplantation

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Achievement of Insulin Independence at 12-month Post Transplant	1 participant

SECONDARY outcome

Timeframe: 12 months after transplantation

Number of patients who achieved absence of hypoglycemic unawareness

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Presence or Absence of Hypoglycemic Unawareness	3 participants

SECONDARY outcome

Timeframe: 12 months after transplantation

Blood glucose \<70 mg/dl, number of times reported per month

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Incidence of Hypoglycemic Episodes	7.3 episodes per month Standard Error 2.5

SECONDARY outcome

Timeframe: 12 months after transplantation

Percentage of insulin requirement at month 12 against that at baseline in the patients who did not achieve insulin independence. The percentage less than 100% indicates that subjects reduced insulin requirements 12 months after islet transplantation when compared with those at pre-transplant, while the parentage more than 100% represents that patients needed higher amount of exogenous insulin 12 months after islet transplantation.

Outcome measures

Outcome measures
Measure	Islet Cell n=3 Participants Allogeneic Islet Cell Transplantation
Change of Insulin Requirements in Patients Who Did Not Become Insulin Independent	34.3 Percent decrease compared to baseline Standard Error 10.5

SECONDARY outcome

Timeframe: At transplantation

The sum of Islet mass obtained after transport using the two-layer preservation method, remote site processing and islet culture. Islet mass as defined by Islet Equivalent per kilogram recipient body weight.

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Islet Cell Mass Obtained After Remote Site Processing	8717 Islet Equivalent per kilogram Standard Error 2243

SECONDARY outcome

Timeframe: 12 months after transplantation

Outcome measures

Outcome measures
Measure	Islet Cell n=1 Participants Allogeneic Islet Cell Transplantation
The Number of Islet Cell Infusions Needed to Achieve Insulin Independence	2 number of infusion Standard Error 0

SECONDARY outcome

Timeframe: 12 months after transplantation

Glomerular filtration rate measured by sodium iothalamate I-125 injection (GLOFIL)

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Renal Function	127.3 ml/min Standard Error 7.9

SECONDARY outcome

Timeframe: 12 months after transplantation

Number of participants who experienced serious adverse events related to immunosuppression regimen

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Morbidity Related to the Immunosuppression Regimen	2 participant

SECONDARY outcome

Timeframe: 12months after transplantation

Number of participants who experienced serious adverse events related to islet cell infusion

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
Morbidity Related to the Islet Cell Infusion	3 participant

SECONDARY outcome

Timeframe: 12 months after transplantation

Averaged score in subscales of 'physical functioning', 'Role limitations due to emotional problems', 'energy/fatigue', 'emotional well-being', 'social functioning', 'pain' and 'general health' in the RAND 36-item short form health survey (SF-36). Full scale range is 0-100 for all subscales with 100 as the best outcome and 0 as the worst outcome.

Outcome measures

Outcome measures
Measure	Islet Cell n=4 Participants Allogeneic Islet Cell Transplantation
The Quality of Life of the Recipients Measured With the RAND 36-item Short Form Health Survey	79.6 Scores on a scale Full Range 12.4 • Interval 57.8 to 88.8

Adverse Events

Islet Cell

Serious events: 4 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Islet Cell n=4 participants at risk Patients with allogeneic islet cell transplantation
Hepatobiliary disorders Elevated Liver Function Tests	100.0% 4/4 • Number of events 4 • 12 months after transplantation The number of adverse events which was observed within 12 months after transplantation
Gastrointestinal disorders Nausea and Vomiting	25.0% 1/4 • Number of events 1 • 12 months after transplantation The number of adverse events which was observed within 12 months after transplantation
Respiratory, thoracic and mediastinal disorders Pneumonia	25.0% 1/4 • Number of events 1 • 12 months after transplantation The number of adverse events which was observed within 12 months after transplantation
Gastrointestinal disorders Abdominal Pain	25.0% 1/4 • Number of events 1 • 12 months after transplantation The number of adverse events which was observed within 12 months after transplantation

Other adverse events

Other adverse events
Measure	Islet Cell n=4 participants at risk Patients with allogeneic islet cell transplantation
Skin and subcutaneous tissue disorders mouth ulcer	100.0% 4/4 • Number of events 4 • 12 months after transplantation The number of adverse events which was observed within 12 months after transplantation
Gastrointestinal disorders nausea and vomiting	100.0% 4/4 • Number of events 4 • 12 months after transplantation The number of adverse events which was observed within 12 months after transplantation

Additional Information

Dr. Marlon Levy

Baylor University Medical Center

Phone: 214-820-2050

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place