Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy

NCT01947569 | Unknown Phase 1 DMC

• 1 other identifier: DV-0100-100
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Treatment of Type I Diabetes Mellitus.

Outcome Measures

Primary Outcomes (1)

• The incidence of treatment-emergent adverse events.

  Month 12

Secondary Outcomes (1)

• 2-hour area under the curve (AUC) average of C-peptide at 12 months after completion of administration of assigned therapy (Protocol Month 15).

  12 Months

Study Arms (3)

iDC recipients

EXPERIMENTAL

iDC cells modified by in vitro engineering.

Biological: Biological

Control DC recipients

ACTIVE COMPARATOR

DC cells which have not been modified.

Biological: Biological

Placebo recipients

PLACEBO COMPARATOR

Saline injections.

Biological: Biological

Interventions

Biological BIOLOGICAL

Control DC recipients; Placebo recipients; iDC recipients

Eligibility Criteria

• Age: 12 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• Evidence of decreased β-cell function as measured by C-peptide and blood glucose levels consistent with impaired glucose tolerance.
• Evidence of at least one high-risk HLA haplotype.
• Evidence of at least one diabetes-related autoantibody (e.g. IA-2, GAD, ZnT8) ,
• Adequate immune competence as assessed by immunoreactivity to alloantigens in mixed leukocyte culture and reactivity to viral antigens (CEF Pool Assay) in vitro.
• Normal hematologic, liver and kidney function.
• 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.

You may not qualify if:

• Enrollment or history of enrollment in a drug, or biologic therapy study sponsored by TrialNet.
• A significant history or current evidence of cardiac disease, uncontrolled hypertension, serious arrhythmias.
• Evidence of active infection requiring antibiotic therapy.
• History of other concurrent significant medical diseases.
• Pregnant or lactating women.
• Patients requiring chronic systemic corticosteroids.
• Any other immune disorder including but not limited to other autoimmune diseases, HIV, HBV, HCV, HPV, HSV positivity.
• Impaired renal function with a creatinine level \> 1.5.
• 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.
• A hemaglobinopathy known to interfere with the ability to accurately determine HbA1c.
• No prior radiation therapy, immunotherapy, or chemotherapy.

Sponsors & Collaborators

• DiaVacs, Inc.: lead

Study Sites (1)

UPMC

Pittsburgh, Pennsylvania, 15213, United States

Location

MeSH Terms

Interventions

Biological Products

Intervention Hierarchy (Ancestors)

Complex Mixtures

Central Study Contacts

Orville G. Kolterman, MD

CONTACT

7242085707; okolterman@alumnistandford.edu

Peter Gregoire, MBA

CONTACT

7242085707; pgregoire@futurelifesourcesllc.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2013
• First Posted: September 20, 2013
• Study Start: October 1, 2013
• Primary Completion: November 1, 2014
• Last Updated: October 23, 2013

Record last verified: 2013-10

Locations

US (1)