TArgeting Type 1 Diabetes Using POLyamines (TADPOL) (TADPOL)

April 15, 2026 updated by: Emily K. Sims

TArgeting Type 1 Diabetes Using POLyamines (TADPOL): A Randomized, Double-Masked, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Difluoromethylornithine (DFMO) to Preserve Insulin Production in Type 1 Diabetes

The goal of this clinical trial is to test a drug known as DFMO in people with Type 1 Diabetes (T1D). The main question[s] it aims to answer are:

  • Does it reduce stress on the cells that make insulin?
  • Does it preserve what is left of the body's insulin production? Participants will take either DFMO or a placebo (looks like DFMO but has no active ingredients) two times a day for about 6 months. Participants will have 6 in person visits and 1 phone visit over a period of 12 months. Visits will include blood draws urine collection and other tests.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This study will be a multicenter, double-blind, placebo-controlled, 2:1 random assigned, phase II clinical trial for individuals with recent onset type 1 diabetes. The investigators are conducting a double masked placebo-controlled intention to treat study enrolling persons with new onset T1D with documented continued residual C-peptide production. Within 45 days of screening and a run-in period during which eligibility will be determined and glycemic control optimized, subjects will have a 6-month double-masked treatment period with either DFMO or placebo. After a 6-month wash-out period the durability of effect will be assessed. Subjects will be randomly assigned either 1000mg/m2/day oral DFMO or placebo treatment at a 2:1 ratio.

Study Type

Interventional

Enrollment (Estimated)

81

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Maria L Spall, BSN
  • Phone Number: 317-278-7034
  • Email: malnicho@iu.edu

Study Contact Backup

  • Name: Operations Manager
  • Phone Number: 317-278-8879
  • Email: tadpol@iu.edu

Study Locations

  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Barbara Davis Center
        • Contact:
        • Principal Investigator:
          • Kimber Simmons, MD
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University of Chicago
        • Principal Investigator:
          • Siri Greeley, MD
        • Contact:
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • IU Health Riley Hospital for Children
        • Contact:
        • Principal Investigator:
          • Emily K Sims, MD, MS
        • Sub-Investigator:
          • Linda A DiMeglio, MD, MPH
        • Contact:
    • Kansas
      • Kansas City, Kansas, United States, 64108
        • Recruiting
        • Children's Mercy Hospital
        • Contact:
        • Principal Investigator:
          • Cintya Schweisberger, DO
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Recruiting
        • MHealth Fairview Masonic Children's Hospital and Specialty Clinics
        • Contact:
        • Principal Investigator:
          • Brandon Nathan, MD
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin
        • Principal Investigator:
          • Susanne Cabrera, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 40 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Males and females 4- ≥40 years of age with a clinical diagnosis of T1D
  2. T1D clinical diagnosis with insulin start date no more than 100 days prior to the time of randomization
  3. Random non-fasting C-peptide level of >0.2 pmol/mL (equivalent to >0.6ng/ml) at screening.
  4. Positive for any one of the following diabetes-related autoantibodies (IAA, GAA, IA-2, or ZnT8)
  5. Treatment naïve of any immunomodulatory agent
  6. Normal hearing at screening, defined as acceptable results of pure-tone audiometry (<20 decibel [dB] baseline thresholds for all frequencies tested

Exclusion Criteria:

  1. Presence of severe, active disease that interferes with dietary intake or requires the use of chronic medication, with the exception of well-controlled hypothyroidism and mild asthma not requiring oral steroids. Presence of any psychiatric disorder that will affect ability to participate in study.
  2. Diabetes other than T1D
  3. Chronic illness known to affect glucose metabolism (e.g. Cushing syndrome, polycystic ovarian disorder, cystic fibrosis) or taking medications that affect glucose metabolism (e.g. steroids, metformin)
  4. Inability to swallow pills
  5. Psychiatric impairment or current use of anti-psychotic medication
  6. Any condition that, in the investigator's opinion, may compromise study participation or may confound the interpretation of the study results.
  7. Neutropenia (< 1,500 neutrophils/μL)
  8. Leukopenia (< 3,000 leukocytes /μL)
  9. Lymphopenia ( < 800 lymphocytes/μL)
  10. Thrombocytopenia (<100,000 platelets/μL)

  11. Clinically significant anemia or Hemoglobin as defined below:

    In Adults: Hgb <12.0g/dL in females and <13.0g/dL in males In Children: 12- <18: <11.4 g/dL in females and <12.4 g/dL in males In Children: 4- <12: Hgb <11.2 g/dL

  12. Impaired renal function (assessed by history and BUN/Creatinine, DFMO is renally excreted)
  13. Allergy to milk or soy (components of Boost® drink used for mixed meal tolerance testing)
  14. Female participants of child-bearing age with reproductive potential, must not be pregnant and agree to use 2 effective forms of birth control or be abstinent during the study period (see below). Male participants (including men who have had vasectomies) whose partners are pregnant or may be pregnant should use condoms while on study drug, until 2 weeks after discontinuation of drug, while the partner is pregnant.
  15. Active seizure disorder, defined as requiring chronic medication at the time of study or having had a seizure within the past 12 months at the time of screening
  16. Enrollment into another intervention trial.
  17. Use of an automated insulin delivery system, including hybrid closed loop or fully closed loop insulin pumps) that do not allow for manual suspension or temporary modification of insulin delivery for bolus dosing.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Treatment Arm
Difluoromethylornithine (DFMO) pill ,1000mg/m2/day, for 6 months
Drug: DFMO
DFMO orally twice a day
Other Names:
  • Difluoromethylornithine
Placebo Comparator: Placebo Arm
Placebo pill taken twice a day orally for 6 months
Drug: Placebo
Placebo orally twice a day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical efficacy of 1000 mg/m2/day of oral DFMO after 6 months of treatment
Time Frame: 6 month
Primary endpoint defining clinical efficacy will be based on mixed-meal stimulated C-peptide area under the curve (AUC; in arbitrary units) in the treatment group compared to placebo after 6 months of DFMO treatment
6 month
Number of participants with treatment-related adverse events as assessed by CTCAE v5
Time Frame: through study completion, an average of one year
A summary of serious and non-serious adverse events (AEs) will be reported.
through study completion, an average of one year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical efficacy of 1000 mg/m2/day of oral DFMO after 3 months of treatment, 9 months after treatment (or 3 months after treatment end), and 12 months after treatment (or 6 months after treatment end).
Time Frame: through study completion, an average of one year
Secondary endpoints will be based on mixed meal stimulated C-peptide AUC (arbitrary units) at 3 months after treatment, 9 months after treatment, and 12 months after treatment.
through study completion, an average of one year
Decrease in urinary polyamides after 6 months of DFMO treatment.
Time Frame: up to 24 weeks after treatment
Decrease in urinary putrescine (in umol/g Cr) from baseline after 6 months of DFMO treatment, measured using high performance liquid chromatography.
up to 24 weeks after treatment
Biomarkers of β cell stress at 3, 6, 9, and 12 months after treatment.
Time Frame: through study completion, an average of one year
Fasting and stimulated proinsulin/c-peptide ratios (%) will be measured using immunoassays and reported at baseline, 3 months after treatment, 6 months after treatment, 9 months after treatment, and 12 months after treatment.
through study completion, an average of one year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Emily K. Sims

Collaborators

Juvenile Diabetes Research Foundation
Cancer Prevention Pharmaceuticals, Inc.

Investigators

  • Study Chair: Emily K Sims, MD,MS, Indiana University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 14, 2023

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

May 1, 2028

Study Registration Dates

First Submitted

October 12, 2022

First Submitted That Met QC Criteria

October 21, 2022

First Posted (Actual)

October 26, 2022

Study Record Updates

Last Update Posted (Actual)

April 20, 2026

Last Update Submitted That Met QC Criteria

April 15, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 4-SRA-2022-1205-M-B

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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