NCT00315133

Brief Summary

The study evaluates the effect of inactivation of the immune system with chemotherapy and immunotherapy and infusion of bone marrow stem cells in early onset type 1 diabetes mellitus. We hypothesize that reprograming the immune system will stop immune aggression to the insulin producing cells allowing their regeneration and thus decreasing or eliminating the need of exogenous insulin.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Dec 2003

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

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

December 1, 2003

Completed
2.4 years until next milestone

First Submitted

Initial submission to the registry

April 13, 2006

Completed
4 days until next milestone

First Posted

Study publicly available on registry

April 17, 2006

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
Last Updated

January 18, 2017

Status Verified

January 1, 2017

Enrollment Period

7 years

First QC Date

April 13, 2006

Last Update Submit

January 13, 2017

Conditions

Type 1 Diabetes Mellitus

Keywords

Diabetes mellitusStem cellsAutologous stem cell transplantationAutoimmune diseases

Outcome Measures

Primary Outcomes (1)

  • C-peptide levels

    Stimulated C-peptide levels will be measured.

    Every 6 months

Secondary Outcomes (6)

  • Transplant-related toxicity

    Every 6 months or when reported

  • Anti-GAD titres

    Every 6 months

  • Exogenous insulin dose

    Every 6 months

  • Hemoglobin A1C

    Every 6 months

  • Immunologic reconstitution parameters

    Yearly

  • +1 more secondary outcomes

Study Arms (1)

Transplant arm

EXPERIMENTAL

This is a single arm study. The intervention is immunosuppression and autologous stem cell transplantation.

Procedure: Immunosuppression and autologous stem cell transplantation

Interventions

Immunosuppression and autologous stem cell transplantationPROCEDURE

Immunosuppression and autologous stem cell transplantation: Mobilization of hematopoietic stem cells (HSC) with cyclophosphamide (2 g/m2) and granulocyte-colony stimulating factor (G-CSF, 10 ug/kg/d), followed by collection and cryopreservation of unselected HSC and conditioning with cyclophosphamide (200 mg/kg) plus rabbit anti-thymocyte globulin (ATG 4.5 mg/kg).

Transplant arm

Eligibility Criteria

Age12 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Type 1 diabetes mellitus diagnosed by clinical/metabolic parameters and positive anti-GAD antibodies
  • Less than 12 weeks from diagnosis

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo

Ribeirão Preto, Ribeirão Preto, 14048-900, Brazil

Location

Related Publications (3)

  • Burt RK, Oyama Y, Traynor A, Kenyon NS. Hematopoietic stem cell therapy for type 1 diabetes: induction of tolerance and islet cell neogenesis. Autoimmun Rev. 2002 May;1(3):133-8. doi: 10.1016/s1568-9972(02)00033-2.

    PMID: 12849006BACKGROUND

  • Voltarelli JC, Couri CE, Stracieri AB, Oliveira MC, Moraes DA, Pieroni F, Coutinho M, Malmegrim KC, Foss-Freitas MC, Simoes BP, Foss MC, Squiers E, Burt RK. Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA. 2007 Apr 11;297(14):1568-76. doi: 10.1001/jama.297.14.1568.

    PMID: 17426276RESULT

  • Couri CE, Oliveira MC, Stracieri AB, Moraes DA, Pieroni F, Barros GM, Madeira MI, Malmegrim KC, Foss-Freitas MC, Simoes BP, Martinez EZ, Foss MC, Burt RK, Voltarelli JC. C-peptide levels and insulin independence following autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA. 2009 Apr 15;301(15):1573-9. doi: 10.1001/jama.2009.470.

    PMID: 19366777RESULT

MeSH Terms

Conditions

Diabetes Mellitus, Type 1Diabetes MellitusAutoimmune Diseases

Interventions

Immunosuppression Therapy

Condition Hierarchy (Ancestors)

Glucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

ImmunotherapyImmunomodulationBiological TherapyTherapeuticsImmunologic TechniquesInvestigative Techniques

Study Officials

  • Julio C Voltarelli, MD PhD

    University Hospital, School of Medicine of Ribeirão Preto, Brazil

    PRINCIPAL INVESTIGATOR

  • Maria C Oliveira, MD PhD

    University Hospital, Ribeirão Preto Medical School, Brazil

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Julio C. Voltarelli

Study Record Dates

First Submitted

April 13, 2006

First Posted

April 17, 2006

Study Start

December 1, 2003

Primary Completion

December 1, 2010

Study Completion

December 1, 2014

Last Updated

January 18, 2017

Record last verified: 2017-01

Locations

BR(1)