Umbilical Cord Blood Infusion to Treat Type 1 Diabetes

• ClinicalTrials.gov Identifier: NCT00305344
• Recruitment Status: Completed
• First Posted: March 21, 2006
• Results First Posted: July 10, 2012
• Last Update Posted: July 10, 2012
• Sponsor: University of Florida
• Collaborators: Juvenile Diabetes Research Foundation; National Institutes of Health (NIH)
• Information provided by (Responsible Party): University of Florida

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Type 1 Diabetes Mellitus
• Interventions: Procedure: Autologous Umbilical Cord Blood Transfusion; Biological: Cord blood
• Enrollment: 23

Participant Flow

Recruitment Details	23 total subjects were enrolled.
Pre-assignment Details	Patients with autologous cord blood in private storage facilities were eligible. No eligible patients were excluded.

Arm/Group Title	Cord Blood Recipient
Arm/Group Description	Umbilical Cord Recipient
Period Title: Overall Study
Started	23
Completed	23
Not Completed	0

Baseline Characteristics

Arm/Group Title		Cord Blood Recipient
Arm/Group Description		Umbilical Cord Recipient
Overall Number of Baseline Participants		23
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	23 participants
	<=18 years	23 100.0%
	Between 18 and 65 years	0 0.0%
	>=65 years	0 0.0%
Age Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	23 participants
		5.1 (1)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	23 participants
	Female	14 60.9%
	Male	9 39.1%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	23 participants
		23

Outcome Measures

1. Primary Outcome

Title	Children With T1D Underwent a Single Autologous UCB Transfusion
Description	All participants were monitored for 2 years. Baseline and post-infusion mixed meal tolerance tests were performed to determine whether autologous cord blood infusion preserved endogenous insulin production. The change in median area under the curve for C-peptide (measure of insuln production) from baseline to to 2 years during a 2 hour mixed meal tolerance test was used as the primary outcome measure and was reported in ng/ml/120 minutes
Time Frame	Baseline to Year 2

Outcome Measure Data

Analysis Population Description

All participants received their own autologous umbilical cord blood (UCB)

Arm/Group Title	Recipients Receiving Cord Blood
Arm/Group Description:	Children with type 1 diabetes who underwent an autologous cord blood infusion
Overall Number of Participants Analyzed	23
Median (Inter-Quartile Range); Unit of Measure: ng /ml /120 min
	-0.6; (-1.0 to -0.37)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Recipients Receiving Cord Blood
	Comments	Null hypothesis: there will be no difference between AUC C-peptide at baseline and 1 or 2 years post cord blood infusion
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	>0.05
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

Adverse Events

Time Frame	2 years
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Cord Blood Recipients
Arm/Group Description	Children with type 1 diabetes who underwent an autologous cord blood infusion
All-Cause Mortality
	Cord Blood Recipients
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	Cord Blood Recipients
	Affected / at Risk (%)
Total	0/23 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	1%
	Cord Blood Recipients
	Affected / at Risk (%)
Total	0/23 (0.00%)

Limitations and Caveats

Small sample size. No randomized control group

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Dr Michael Haller
• Organization: University of Florida
• Phone: 352.273.9264
• EMail: hallemj@peds.ufl.edu

Publications of Results:

Haller MJ, Wasserfall CH, Hulme MA, Cintron M, Brusko TM, McGrail KM, Sumrall TM, Wingard JR, Theriaque DW, Shuster JJ, Atkinson MA, Schatz DA. Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide. Diabetes Care. 2011 Dec;34(12):2567-9. doi: 10.2337/dc11-1406. Epub 2011 Oct 19.

Haller MJ, Wasserfall CH, McGrail KM, Cintron M, Brusko TM, Wingard JR, Kelly SS, Shuster JJ, Atkinson MA, Schatz DA. Autologous umbilical cord blood transfusion in very young children with type 1 diabetes. Diabetes Care. 2009 Nov;32(11):2041-6. doi: 10.2337/dc09-0967.

• Responsible Party: University of Florida
• ClinicalTrials.gov Identifier: NCT00305344
• Other Study ID Numbers: 1-2005-362; GCRC 593 ( Other Identifier: General Clinical Research Center ); UF IRB-01 125-2004 ( Other Identifier: UF Institutional Review Board )
• First Submitted: March 17, 2006
• First Posted: March 21, 2006
• Results First Submitted: March 2, 2012
• Results First Posted: July 10, 2012
• Last Update Posted: July 10, 2012