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Treatment of Type I Diabetes Mellitus.Phase 1/2NCT01947569

Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy

A Phase 1/2 study testing Biological in people with Treatment of Type I Diabetes Mellitus., run by DiaVacs, Inc. at 1 site in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 12 to 35 years
Conditions the study is for
Treatment of Type I Diabetes Mellitus.
What is being tested?
Biological
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
90 participants
Where
1 siteUnited States
Study dates
Oct 2013to Nov 2014
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 35 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

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.

Trial locations and contacts

1 site in 1 country.

Orville G. Kolterman, MD contact

7242085707

okolterman@alumnistandford.edu

United States1 site
  • UPMC

    Pittsburgh, Pennsylvania, 15213

    Mary Korytokowski, MD

Other trials in United States

About this study

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

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.

Being tested
Biological

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BiologicalBiological

Study groups in NCT01947569
Study groupRoleWhat they receive
iDC recipientsExperimentaliDC cells modified by in vitro engineering.
Receives: Biological: Biological
Control DC recipientsActive comparatorDC cells which have not been modified.
Receives: Biological: Biological
Placebo recipientsPlacebo comparatorSaline injections.
Receives: Biological: Biological

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The incidence of treatment-emergent adverse events.

    Measured: Month 12

Secondary outcomes (1)
  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).

    Measured: 12 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Sep 2013 (estimated)
  2. Study startOct 2013
  3. Primary completionNov 2014 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
90 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01947569
Sponsor's protocol ID
DV-0100-100
Status last verified
Oct 2013
First submitted
18 Sep 2013
Last updated
23 Oct 2013
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Biological in people with Treatment of Type I Diabetes Mellitus., run by DiaVacs, Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 23 Oct 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Orville G. Kolterman, MD (7242085707, okolterman@alumnistandford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The incidence of treatment-emergent adverse events. (time frame: Month 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2013.

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