Type 1 DiabetesPhase 1/2NCT01996228
Reversal of Type 1 Diabetes in Children by Stem Cell Educator Therapy
A Phase 1/2 study testing Stem Cell Educator in people with Type 1 Diabetes, run by Throne Biotechnologies Inc. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 14 years
- Conditions the study is for
- Type 1 Diabetes
- What is being tested?
- Stem Cell Educator
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Nov 2013to Oct 2019
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The Second Xiangya Hospital
Changsha, Hunan, 410011
Xia Li, MD, PhD, info.T1D@tianhecell.com
About this study
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.
- Conditions
- Type 1 Diabetes
- Being tested
- Stem Cell Educator
- Keywords
- Type 1 diabetes, autoimmune, cord blood stem cell, stem cell educator, immune modulation, children, Control autoimmunity, stimulate the regeneration of islet beta cells
- Medical subject headings
- Diabetes Mellitus, Type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell EducatorDevice
Also called Procedure: Apharesis and Stem Cell Educator Therapy, Biological: Cord blood
| Study group | Role | What they receive |
|---|---|---|
| Cord Blood-derived multipotent stem cells | Experimental | Human cord blood-derived multipotent stem cells (CB-SC) display unique phenotypes, such as the expression of embryonic stem (ES) cell markers, multipotential of differentiations, very low immunogenecity, and immune modulations in patients. Receives: Device: Stem Cell Educator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Autoimmune control
Before treatment, test autoimmune-related markers as baseline; After treatment for 90 days, repeat testing autoimmune-related markers.
Measured: 90 days post treatment
Secondary outcomes (1)
Metabolic control
Before treatment, test for C-peptide levels and HbA1C as baseline; After treatment, test C-peptide levels and HbA1C on the 3rd month.
- Analysis of islet beta cell function
- Test for C-peptide levels on every 6 month;
- Full evaluation of islet beta cell function after two years.
Measured: 3-24 months post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2013 (estimated)
- Study startNov 2013
- Primary completionOct 2019 (estimated)
- Study completionOct 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Type 1 DiabetesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Throne Biotechnologies Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Second Xiangya Hospital of Central South University
- Responsible party
- Principal investigator, Yong Zhao, MD, PhD, Throne Biotechnologies Inc.
- Investigators
- Yong Zhao, MD,PhD, Study chair, Tianhe Stem Cell BiotechnologiesZhiguang Zhou, Md,PhD, Principal investigator, Second Xiangya Hospital of Central South University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01996228
- Sponsor's protocol ID
- 2013-0002
- Status last verified
- Feb 2019
- First submitted
- 21 Nov 2013
- Last updated
- 5 Feb 2019
- Results posted
- No
Publications
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.
PubMed 22233865Zhao 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.
PubMed 23837842Zhao 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.
PubMed 22833322Zhao 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.
PubMed 20728583
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reversal of Type 1 Diabetes in Children by Stem Cell Educator Therapy
- ClinicalTrials.gov ID
- NCT01996228
- Acronym / sponsor ID
- 2013-0002
- 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
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Throne Biotechnologies Inc.Industry (pharma, biotech, devices)
- Collaborators
- Second Xiangya Hospital of Central South University
- Study start
- Nov 2013
- Primary completion
- Oct 2019 (estimated)
- Study completion
- Oct 2019 (estimated)
- First posted
- 27 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cord Blood-derived multipotent stem cells | Experimental | Device: Stem Cell EducatorHuman cord blood-derived multipotent stem cells (CB-SC) display unique phenotypes, such as the expression of embryonic stem (ES) cell markers, multipotential of differentiations, very low immunogenecity, and immune modulations in patients. |
Who may qualify
- Ages
- 6 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Stem Cell EducatorAlso: Procedure: Apharesis and Stem Cell Educator Therapy, Biological: Cord blood | Device | Cord Blood-derived multipotent stem cells |
Primary outcome
Autoimmune control
Time frame: 90 days post treatment
Before treatment, test autoimmune-related markers as baseline; After treatment for 90 days, repeat testing autoimmune-related markers.
1 secondary outcome
Metabolic control
Time frame: 3-24 months post treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2019.
- Background: PubMed 22233865Zhao 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.
- Background: PubMed 23837842Zhao 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.
- Background: PubMed 22833322Zhao 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.
- Background: PubMed 20728583Zhao 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yong Zhao, MD,PhD study chair
Tianhe Stem Cell Biotechnologies
Zhiguang Zhou, Md,PhD principal investigator
Second Xiangya Hospital of Central South University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Stem Cell Educator in people with Type 1 Diabetes, run by Throne Biotechnologies Inc. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yong Zhao, MD, PhD (001 630 723 1968, zhao@tianhecell.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Autoimmune control (time frame: 90 days post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01996228, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2019.