Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy

This study is not yet open for participant recruitment.
Verified October 2013 by DiaVacs, Inc.
Sponsor:
Information provided by (Responsible Party):
DiaVacs, Inc.
ClinicalTrials.gov Identifier:
NCT01947569
First received: September 18, 2013
Last updated: October 22, 2013
Last verified: October 2013
  Purpose

Phase IB will evaluate the safety of autologous, ex vivo-engineered, co-stimulation impaired dendtritic cells to maintain and improve functional residual beta cell mass in new onset Type I Diabetes Mellitus (T1DM) patients. Efficacy measures will be collected and summarized.

Phase IIA will evaluate the safety and efficacy of 3 randomized treatment groups in new onset T1DM patients to assess if the antisense DNA-treated co-stimulation-impaired immunoregulatory dendritic cells (iDC) will safely preserve and/or increase B-cell mass resulting in improvement and/or normalization of blood glucose levels and glycated hemoglobin A1c.


Condition Intervention Phase
Treatment of Type I Diabetes Mellitus.
Biological: Biological
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: Autologous Co-stimulation-impaired Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy: A Sequential Open Label Phase IB Safety Assessment/ Randomized, Double-blind Phase IIA Efficacy Trial to Maintain and Improve Functional Beta Cell Mass in New Onset Disease T1DM Patients

Resource links provided by NLM:


Further study details as provided by DiaVacs, Inc.:

Primary Outcome Measures:
  • The incidence of treatment-emergent adverse events. [ Time Frame: Month 12 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • 2-hour area under the curve (AUC) average of C-peptide at 12 months after completion of administration of assigned therapy (Protocol Month 15). [ Time Frame: 12 Months ] [ Designated as safety issue: No ]

Estimated Enrollment: 90
Study Start Date: October 2013
Estimated Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: iDC recipients
iDC cells modified by in vitro engineering.
Biological: Biological
Active Comparator: Control DC recipients
DC cells which have not been modified.
Biological: Biological
Placebo Comparator: Placebo recipients
Saline injections.
Biological: Biological

  Eligibility

Ages Eligible for Study:   12 Years to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:(both open label phase IB safety and Phase IIA study):

  1. Patients with new onset T1DM (>18 years of age for the phase IB and then >16 (first 10 subjects), >12 years of age for the second 10 subjects, > 8 years for the next 10 subjects and, finally, >8 years of age for the remainder of the phase IIA patients) within 6 months of diabetes mellitus diagnosis.
  2. Evidence of decreased β-cell function as measured by C-peptide and blood glucose levels consistent with impaired glucose tolerance.
  3. Evidence of at least one high-risk HLA haplotype.
  4. Evidence of at least one diabetes-related autoantibody (e.g. IA-2, GAD, ZnT8) ,
  5. Adequate immune competence as assessed by immunoreactivity to alloantigens in mixed leukocyte culture and reactivity to viral antigens (CEF Pool Assay) in vitro.
  6. Normal hematologic, liver and kidney function.
  7. Female participants of childbearing potential in this study must agree to use an effective form of birth control during study participation. Reliable and effective forms of birth control include: true abstinence, intrauterine device (IUD), hormonal-based contraception, double-barrier contraception [condom or occlusive cap (diaphragm or cervical cap) with spermicide], or surgical sterilization (vasectomy for male partner, tubal ligation or hysterectomy). Sexually active male participants must agree to use an effective form of birth control such as condoms.

Exclusion Criteria:(both open label phase IB safety and Phase IIA study):

  1. Enrollment or history of enrollment in a drug, or biologic therapy study sponsored by TrialNet.
  2. A significant history or current evidence of cardiac disease, uncontrolled hypertension, serious arrhythmias.
  3. Evidence of active infection requiring antibiotic therapy.
  4. History of other concurrent significant medical diseases.
  5. Pregnant or lactating women.
  6. Patients requiring chronic systemic corticosteroids.
  7. Any other immune disorder including but not limited to other autoimmune diseases, HIV, HBV, HCV, HPV, HSV positivity.
  8. Impaired renal function with a creatinine level > 1.5.
  9. Administration of the following therapies while patients are undergoing treatment on this protocol: i) radiation therapy; ii) chemotherapy; iii) corticosteroids (except when administered in life-threatening circumstances); iv) other particle or cell-based therapies; v) other biologic therapies; vi) other therapies aimed at modulating the immune system; vii) other endocrine-related therapies, hormone replacement (other than thyroxine and contraceptive), glucoregulation.
  10. A hemaglobinopathy known to interfere with the ability to accurately determine HbA1c.
  11. No prior radiation therapy, immunotherapy, or chemotherapy.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01947569

Contacts
Contact: Orville G. Kolterman, MD 7242085707 okolterman@alumnistandford.edu
Contact: Peter Gregoire, MBA 7242085707 pgregoire@futurelifesourcesllc.org

Locations
United States, Pennsylvania
UPMC Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15213
Principal Investigator: Mary Korytokowski, MD         
Sponsors and Collaborators
DiaVacs, Inc.
  More Information

No publications provided

Responsible Party: DiaVacs, Inc.
ClinicalTrials.gov Identifier: NCT01947569     History of Changes
Other Study ID Numbers: DV-0100-100
Study First Received: September 18, 2013
Last Updated: October 22, 2013
Health Authority: United States: Food and Drug Administration

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 April 16, 2014