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
Emily K. Sims
Summary
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.
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…
Eligibility
- Age range
- 4–40 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugDFMO
DFMO orally twice a day
- DrugPlacebo
Placebo orally twice a day
Locations (7)
- Barbara Davis CenterAurora, Colorado
- University of ChicagoChicago, Illinois
- IU Health Riley Hospital for ChildrenIndianapolis, Indiana
- Children's Mercy HospitalKansas City, Kansas
- University of MichiganAnn Arbor, Michigan
- MHealth Fairview Masonic Children's Hospital and Specialty ClinicsMinneapolis, Minnesota