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

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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. We 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.


Child to Adult


Participants must meet all of the following criteria: 1. Males and females 6-&#8804;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 naive of any immunomodulatory agent 6. Normal hearing at screening, defined as acceptable results of pure-tone audiometry (<20 decibel [dB] baseline thresholds for frequencies 250, 500, 1000, and 2000 HzEXCLUSION CRITERIA Participants must NOT meet any of the following 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) 16 Protocol Version 3.0 February 20,2023 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. Hematologic abnormalities at screening (anemia, leukopenia (particularly neutropenia), or thrombocytopenia) 8. Impaired renal function (assessed by history and BUN/Creatinine, DFMO is renally excreted) 9. Allergy to milk or soy (components of Boost? drink used for mixed meal tolerance testing) 10. 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. 11. 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 12. Enrollment into another intervention trial.


Barbara Davis Center
University of Colorado Hospital

Principal Investigator
Photograph of Kimber Simmons

Kimber Simmons

Study ID

Protocol Number: 23-0398

More information available at NCT05594563

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