Clinical trial NCT01996228: Reversal of Type 1 Diabetes in Children by Stem Cell Educator Therapy
Official title: Reversal of Type 1 Diabetes in Children by Stem Cell Educator Therapy
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Type 1 Diabetes
Keywords: Type 1 diabetes
autoimmune
cord blood stem cell
stem cell educator
immune modulation
children
Control autoimmunity
stimulate the regeneration of islet beta cells
Lead sponsor: Throne Biotechnologies Inc.
Sponsor class: INDUSTRY
Collaborators: name: Second Xiangya Hospital of Central South University
First posted: 2013-11-27
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Type 1 diabetes (T1D) is an autoimmune disease that usually occurs in children and reduces their pancreatic islet beta cells and thereby limits insulin production. Millions of individuals worldwide have T1D, and the number of children with diagnosed or undiagnosed T1D is increasing annually. Insulin supplementation is not a cure. It does not halt the persistent autoimmune response, nor can it reliably prevent devastating complications such as neuronal and cardiovascular diseases, blindness, and kidney failure. A true cure has proven elusive despite intensive research pressure over the past 25 years. Notably, Dr.Zhao and his team have successfully developed a groundbreaking technology Stem Cell Educator therapy (Zhao Y, et al.BMC Medicine 2011, 2012). To date, clinical trials in adult patients have demonstrated the safety and efficacy of Stem Cell Educator therapy for the treatment of T1D and other autoimmune-associated diseases. Here, the investigators will evaluate the safety and efficacy of Stem Cell Educator therapy in children with type 1 diabetes.


Eligibility criteria: Inclusion Criteria:

* 1. T1D patients are screened for enrollment in the study if both clinical signs and laboratory tests meet the diagnosis standards of American Diabetes Association.

  2. Children from 3 through 18 years old and body weight \> 15 kg.

  3. Presence of at least one autoantibody to the pancreatic islet β cells (IA-2, GAD, ICA, ZnT8, or IAA).

  4. Written informed consent from the child and child's parents or legal representative.

Exclusion Criteria:

* 1. Any clinically significant diseases in liver, kidney, and heart.

  2. Additional exclusion criteria include no immunosuppressive medication, no viral diseases or diseases associated with immunodeficiency

  3. Significantly abnormal hematology results at screening.

  4. Presence of any infection diseases or inflammation conditions, including active skin infections, flu, fever, upper or lower respiratory track infections.

Primary outcomes:
measure: Autoimmune control
timeFrame: 90 days post treatment
Secondary outcomes:
measure: Metabolic control
timeFrame: 3-24 months post treatment
Other outcomes:
Locations:
city: Changsha
country: China
facility: The Second Xiangya Hospital
state: Hunan
Study officials:
affiliation: Tianhe Stem Cell Biotechnologies
name: Yong Zhao, MD,PhD
role: STUDY_CHAIR
affiliation: Second Xiangya Hospital of Central South University
name: Zhiguang Zhou, Md,PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Zhao Y, Jiang Z, Zhao T, Ye M, Hu C, Yin Z, Li H, Zhang Y, Diao Y, Li Y, Chen Y, Sun X, Fisk MB, Skidgel R, Holterman M, Prabhakar B, Mazzone T. Reversal of type 1 diabetes via islet beta cell regeneration following immune modulation by cord blood-derived multipotent stem cells. BMC Med. 2012 Jan 10;10:3. doi: 10.1186/1741-7015-10-3.
citation: Zhao Y, Jiang Z, Zhao T, Ye M, Hu C, Zhou H, Yin Z, Chen Y, Zhang Y, Wang S, Shen J, Thaker H, Jain S, Li Y, Diao Y, Chen Y, Sun X, Fisk MB, Li H. Targeting insulin resistance in type 2 diabetes via immune modulation of cord blood-derived multipotent stem cells (CB-SCs) in stem cell educator therapy: phase I/II clinical trial. BMC Med. 2013 Jul 9;11:160. doi: 10.1186/1741-7015-11-160.
citation: Zhao Y. Stem cell educator therapy and induction of immune balance. Curr Diab Rep. 2012 Oct;12(5):517-23. doi: 10.1007/s11892-012-0308-1.
citation: Zhao Y, Mazzone T. Human cord blood stem cells and the journey to a cure for type 1 diabetes. Autoimmun Rev. 2010 Dec;10(2):103-7. doi: 10.1016/j.autrev.2010.08.011. Epub 2010 Aug 20.