Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes
Recruitment status was Recruiting
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Type 1 Diabetes Mellitus |
Procedure: Immunosuppression and autologous stem cell transplantation |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: 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 |
- Exogenous insulin dose [ Time Frame: 1 month, 3 months, 6 months, 12 months, 24 months, 36 months ] [ Designated as safety issue: No ]
- Anti-GAD titres [ Time Frame: 1 month, 3 months, 6 months, 12 months, 24 months, 36 months ] [ Designated as safety issue: No ]
- C-peptide level [ Time Frame: 1 month, 3 months, 6 months, 12 months, 24 months, 36 months ] [ Designated as safety issue: No ]
- HbA1c level [ Time Frame: 3 months, 6 months, 12 months, 24 months, 36 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 30 |
| Study Start Date: | February 2008 |
| Estimated Study Completion Date: | February 2013 |
| Estimated Primary Completion Date: | January 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Active Comparator: insulin therapy
The participants not accepted written informed consent will receive insulin therapy
|
Procedure: Immunosuppression and autologous stem cell transplantation
All study participants given written informed consent will perform autologous hematopoietic stem cell transplantation.
Other Name: hematopoietic stem cell
|
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.
Eligibility| Ages Eligible for Study: | 14 Years to 35 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations| Contact: Guang Ning, MD.PhD. | 86-21-64370045 ext 665340 | guangning@medmail.com.cn |
| Contact: WeiQiong Gu, MD. | 86-21-64370045 ext 665391 | weiqionggu@hotmail.com |
| China, Shanghai | |
| Shanghai Jiao Tong University School of Medicine | Recruiting |
| Shanghai, Shanghai, China, 200025 | |
| Contact: Guang Ning, MD.PhD. 86-21-64370045 ext 665340 guangning@medmail.com.cn | |
| Principal Investigator: Guang Ning, MD.PhD. | |
| Principal Investigator: | Guang Ning, MD. PhD. | Shanghai Jiao Tong University School of Medicine |
More Information
Publications:
Additional 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 |
| Study First Received: | December 11, 2008 |
| Last Updated: | March 7, 2012 |
| Health Authority: | China: Ethics Committee |
Keywords provided by 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 |
ClinicalTrials.gov processed this record on May 21, 2013