Efficacy and Safety Study of Autologous Hematopoietic Stem Cell Transplantation to Treat New Onset Type 1 Diabetes
Prospective Study of Autologous Hematopoietic Stem Cell Transplantation to Treat New Onset Type 1 Diabetes
1 other identifier
interventional
50
1 country
1
Brief Summary
Type 1 diabetes is an autoimmune disease and results from T cell autoimmunity mediated destruction of the majority of insulin-producing pancreatic β-cells. Hence,the development of new therapies to control T cell autoimmunity, and to preserve the remaining β-cell function will be of great significance in managing patients with type 1 diabetes Autologous nonmyeloablative hematopoietic stem cell transplantation (AHST) has been tested for the treatment of patients with new onset of type 1 diabetes. This therapeutic strategy can result in exogenous insulin independence by destroying pathogenic memory T cells and preserving the remaining β-cell function. However, little is known about the efficacy of AHST in the dynamics of immunocompetent cell reconstitution and how the reconstituted immune system regulates β-cell specific antibody response. Furthermore, many Chinese patients at diagnosis of type 1 diabetes have progressed to develop diabetic ketoacidosis (DKA). Whether treatment with AHST could still achieve adequate glycemic control and preserve the β-cell function and what the factors are associated with the therapeutic efficacy have not been explored. This is a phase Ⅱ clinical trial in patients who have been diagnosed with type 1 diabetes within the previous 12 months.This study is to determine:
- The effects of autologous hematopoietic stem cell transplantation on the reconstitution of immune system
- β-cell preservation following stem cell transplantation
- The potential factors affecting efficacy of stem cell transplantation
- Whether this new therapy is safe.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2006
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2006
CompletedFirst Submitted
Initial submission to the registry
April 21, 2011
CompletedFirst Posted
Study publicly available on registry
April 26, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2015
CompletedNovember 1, 2016
October 1, 2016
6.5 years
April 21, 2011
October 31, 2016
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Changes in C-peptide levels during standard-meal tolerance test from baseline to different time points after transplantation
3 months, 6 months, 12 months, then yearly after transplantation, up to 10 years
Secondary Outcomes (5)
Changes in serum levels of HbA1c from baseline to different time points after transplantation
3 months, 6 months, 12 months, then yearly after transplantation, up to 10 years
Temporal changes of exogenous insulin requirement from baseline to different time points after transplantation
3 months, 6 months, 12 months, then yearly after transplantation, up to 10 years
Dynamic changes in islet antibody status from baseline to different time points after transplantation
3 months, 6 months, 12 months, then yearly after transplantation, up to 10 years
Dynamic changes in lymphocyte immunophenotyping and cytokine profiles from baseline to different time points after transplantation
3 months, 6 months, 12 months, then yearly after transplantation, up to 10 years
mortality and dysfunction of other endocrine glands
up to 10 years
Study Arms (1)
stem cell transplantation
EXPERIMENTALInterventions
Hematopoietic stem cells were mobilized with cyclophosphamide (CY, 2.0 g/m2) and granulocyte colonystimulating factor (10 μg/kg per day) and then collected from peripheral blood by leukapheresis and cryopreserved. The cells were infused after conditioning with CY (200 mg/kg) and rabbit antithymocyte globulin (4.5 mg/kg).
Eligibility Criteria
You may qualify if:
- diagnosis of diabetes according to the guidelines of the World Health Organization 1999
- the duration of diabetes is no more than 12 months
- positive for for glutamic acid decarboxylase antibody (GADA), protein tyrosine phosphatase antibody (IA-2A), islet cell antibody (ICA) and/or insulin autoantibody (IAAs)
You may not qualify if:
- pregnancy
- mental disorders
- blood diseases
- the presence of any other severe diseases that could potentially influence the transplantation outcomes
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
at Division of Endocrinology, the Affiliated Drum Tower Hospital of Nanjing University
Nanjing, Jiangsu, 210008, China
Related Publications (1)
Li L, Shen S, Ouyang J, Hu Y, Hu L, Cui W, Zhang N, Zhuge YZ, Chen B, Xu J, Zhu D. Autologous hematopoietic stem cell transplantation modulates immunocompetent cells and improves beta-cell function in Chinese patients with new onset of type 1 diabetes. J Clin Endocrinol Metab. 2012 May;97(5):1729-36. doi: 10.1210/jc.2011-2188. Epub 2012 Mar 14.
PMID: 22419704DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Dalong Zhu, MD,PhD
the Affiliated Drum Tower Hospital of Nanjing University
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Chief Physician
Study Record Dates
First Submitted
April 21, 2011
First Posted
April 26, 2011
Study Start
June 1, 2006
Primary Completion
December 1, 2012
Study Completion
December 1, 2015
Last Updated
November 1, 2016
Record last verified: 2016-10