Trial Outcomes & Findings for Autologous Adult Stem Cells to Patients With Type 1 Diabetes and a Successful Renal Transplant (NCT NCT00788827)

Results Overview

Safety will be evaluated in terms of adverse events graded according to CTCTAE toxicity criteria and laboratory test results. All adverse events will also be graded for relationship to treatment and as expected and unexpected.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

7 participants

Primary outcome timeframe

14 days

Results posted on

2019-11-18

Participant Flow

Participant milestones

Participant milestones
Measure	Autologous CD34+ Stem Cells Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion
Overall Study STARTED	7
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Autologous CD34+ Stem Cells Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion
Overall Study Lack of Efficacy	2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm n=7 Participants Autologous CD34+ Stem Cells
Age, Categorical <=18 years	0 Participants n=7 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=7 Participants
Age, Categorical >=65 years	0 Participants n=7 Participants
Age, Continuous	54.6 years STANDARD_DEVIATION 4.2 • n=7 Participants
Sex: Female, Male Female	2 Participants n=7 Participants
Sex: Female, Male Male	5 Participants n=7 Participants
Region of Enrollment United Kingdom	7 participants n=7 Participants

PRIMARY outcome

Timeframe: 14 days

Safety will be evaluated in terms of adverse events graded according to CTCTAE toxicity criteria and laboratory test results. All adverse events will also be graded for relationship to treatment and as expected and unexpected.

Outcome measures

Outcome measures
Measure	Autologous Stem Cells n=7 Participants Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion	Post Infusion of Stem Cells After the infusion of stem cells
Number of Participants Who Experienced Adverse Events Haematoma at femoral catheter insertion	1 participants	—
Number of Participants Who Experienced Adverse Events Fatigue	7 participants	—

SECONDARY outcome

Timeframe: 12 weeks

Population: Patients who were infused with stem cells

Mean HbA1c laboratory measurements pre and post stem cell infusion

Outcome measures

Outcome measures
Measure	Autologous Stem Cells n=5 Participants Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion	Post Infusion of Stem Cells n=5 Participants After the infusion of stem cells
Hba1C Data of Pre and Post Stem Cell Infusion	7.2 percentage Standard Deviation 1.3	7.24 percentage Standard Deviation 1.2

SECONDARY outcome

Timeframe: 12 weeks

Population: Participants who received a stem cell infusion

Mean insulin requirement was calculated for each participant pre and post stem cell infusion

Outcome measures

Outcome measures
Measure	Autologous Stem Cells n=5 Participants Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion	Post Infusion of Stem Cells n=5 Participants After the infusion of stem cells
Insulin Level	59.4 iu/day Standard Deviation 25.7	54.06 iu/day Standard Deviation 18.2

SECONDARY outcome

Timeframe: 12 weeks

Population: Participants who received a stem cell infusion

Each participant had mean amylase data analysed to give a pre and post mean result

Outcome measures

Outcome measures
Measure	Autologous Stem Cells n=5 Participants Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion	Post Infusion of Stem Cells n=5 Participants After the infusion of stem cells
Amylase Level	48.49 units/L Standard Deviation 25.2	75.52 units/L Standard Deviation 34.2

SECONDARY outcome

Timeframe: 12 weeks

Population: Participants who received stem cell infusion

Each participant had serum creatinine analysis pre and post stem cell infusion to give mean result

Outcome measures

Outcome measures
Measure	Autologous Stem Cells n=5 Participants Up to 5 x 10 log 8 of autologous stem cells on a single occasion Autologous CD34+ stem cells: Up to 5 x 10 log 8 of autologous stem cells on a single occasion	Post Infusion of Stem Cells n=5 Participants After the infusion of stem cells
Serum Creatinine	128.22 umol/L Standard Deviation 11.9	118.64 umol/L Standard Deviation 15.5

Adverse Events

Single Arm

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Single Arm n=7 participants at risk Autologous stem cells
Nervous system disorders Fatigue	100.0% 7/7 • Number of events 7 • 12 weeks
Blood and lymphatic system disorders Haemotoma	14.3% 1/7 • Number of events 1 • 12 weeks

Additional Information

Professor Charles Pusey

Imperial College London

Phone: +44 3313 2308

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place