ClinicalTrials.gov
ClinicalTrials.gov Menu

Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00807651
Recruitment Status : Unknown
Verified June 2016 by Guang Ning, Shanghai Jiao Tong University School of Medicine.
Recruitment status was:  Active, not recruiting
First Posted : December 12, 2008
Last Update Posted : June 7, 2016
Sponsor:
Information provided by (Responsible Party):
Guang Ning, Shanghai Jiao Tong University School of Medicine

Brief Summary:
This is a phase II trial in individuals who have been diagnosed with type 1 diabetes within the previous 6 months. The study is evaluating whether stem cell transplantation is safe when chemotherapy and immunotherapy are used in combination and if it has immune resetting effect that may halt the immune attack to pancreas islets and thus preserve the body's own insulin production.

Condition or disease Intervention/treatment Phase
Type 1 Diabetes Mellitus Procedure: AHSCT Drug: Insulin therapy Phase 2

Detailed Description:

Type 1 diabetes is an autoimmune disease in which the immune system mistakenly attacks the insulin-producing beta cells in the pancreas. Generally, at the time someone is diagnosed with type 1 diabetes, not all of a person's beta cells have been destroyed. It's important to preserving the remaining precious beta cells so as to stop the diabetes progression.

The exact mechanism of action of Autologous Hematopoietic Stem Cell Transplantation(AHST) in autoimmune disorders is not fully understood. Preliminary data supported post-AHST immune resetting included an increase in thymus-derived naive T cells, decreased central-memory T cells, increased output of recent thymic emigrants, and recovery of a diverse but distinct T-cell receptor repertoire following AHST. In the patients of type 1 diabetes, decreasing titer of anti-GAD antibody may bring improvement of beta-cell function after intensive immunosuppression. Furthermore, there may exit the possibility of regeneration of beta cells from surviving beta cells or from pancreatic or bone marrow stem cells.

Patients recently diagnosed (less than 6 months) with type 1 diabetes mellitus proved by positive antibody against glutamic acid decarboxylase will be included in this study. Hematopoietic stem cells will be mobilized with cyclophosphamide (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 injected intravenously after conditioning with cyclophosphamide (200 mg/kg) and rabbit antithymocyte globulin (4.5 mg/kg). This procedure is performed in isolated rooms at the Bone Marrow Transplantation Unit of Shanghai Ruijin Hospital affiliated to Shanghai Jiao-Tong University School of Medicine. Patients will be discharged from the hospital 1 month after transplantation and continue the follow-ups for 3 years. Patients fitting the inclusion criteria but not agreeing to perform the transplantation are the control group and they will be followed in parallel with transplanted patients.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy Study of Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes-a Phase II Study
Study Start Date : February 2008
Estimated Primary Completion Date : June 2016
Estimated Study Completion Date : December 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Active Comparator: insulin therapy
The participants not accepted written informed consent will receive insulin therapy
Drug: Insulin therapy
All study participants not accepted written informed consent will received insulin therapy with consistent or multiply subcutaneous injection.
Other Name: insulin injection therapy

Experimental: AHSCT
The participants accepted written informed consent will receive the therapy of autologous hematopoietic stem cell transplantation(AHSCT)
Procedure: AHSCT
All study participants given written informed consent will perform autologous hematopoietic stem cell transplantation(AHSCT) and be treated with immunosuppression.
Other Name: hematopoietic stem cell




Primary Outcome Measures :
  1. Exogenous insulin dose [ Time Frame: 1 month, 3 months, 6 months, 12 months, 24 months, 36 months ]

Secondary Outcome Measures :
  1. Anti-GAD titres [ Time Frame: 1 month, 3 months, 6 months, 12 months, 24 months, 36 months ]
  2. C-peptide level [ Time Frame: 1 month, 3 months, 6 months, 12 months, 24 months, 36 months ]
  3. HbA1c level [ Time Frame: 3 months, 6 months, 12 months, 24 months, 36 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   14 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Type 1 diabetes mellitus diagnosed by clinical/metabolic parameters and positive anti-GAD antibody
  • Less than 6 months from diagnosis

Exclusion Criteria:

  • Previous diabetic ketoacidosis
  • Pregnancy
  • Severe psychiatric disorder
  • Severe organic impairment (renal, hepatic, cardiac, pulmonary)
  • Active infectious disease
  • Previous or present neoplastic disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00807651


Locations
China, Shanghai
Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai, China, 200025
Sponsors and Collaborators
Shanghai Jiao Tong University School of Medicine
Investigators
Principal Investigator: Guang Ning, MD. PhD. Shanghai Jiao Tong University School of Medicine

Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Guang Ning, Director, Shanghai Jiao Tong University School of Medicine
ClinicalTrials.gov Identifier: NCT00807651     History of Changes
Other Study ID Numbers: CCEMD006
First Posted: December 12, 2008    Key Record Dates
Last Update Posted: June 7, 2016
Last Verified: June 2016

Keywords provided by Guang Ning, Shanghai Jiao Tong University School of Medicine:
Diabetes Mellitus
Stem cells
Autologous stem cell transplantation
Autoimmune diseases

Additional relevant MeSH terms:
Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases
Insulin, Globin Zinc
Insulin
Hypoglycemic Agents
Physiological Effects of Drugs