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.
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
81
Barbara Davis Center
Aurora, Colorado, United States
RECRUITINGUniversity of Chicago
Chicago, Illinois, United States
RECRUITINGIU Health Riley Hospital for Children
Indianapolis, Indiana, United States
Clinical efficacy of 1000 mg/m2/day of oral DFMO after 6 months of treatment
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
Time frame: 6 month
Number of participants with treatment-related adverse events as assessed by CTCAE v5
A summary of serious and non-serious adverse events (AEs) will be reported.
Time frame: through study completion, an average of one year
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).
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.
Time frame: through study completion, an average of one year
Decrease in urinary polyamides after 6 months of DFMO treatment.
Decrease in urinary putrescine (in umol/g Cr) from baseline after 6 months of DFMO treatment, measured using high performance liquid chromatography.
Time frame: up to 24 weeks after treatment
Biomarkers of β cell stress at 3, 6, 9, and 12 months after treatment.
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.
Time frame: through study completion, an average of one year
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Children's Mercy Hospital
Kansas City, Kansas, United States
RECRUITINGUniversity of Michigan
Ann Arbor, Michigan, United States
RECRUITINGMHealth Fairview Masonic Children's Hospital and Specialty Clinics
Minneapolis, Minnesota, United States
RECRUITINGMedical College of Wisconsin
Milwaukee, Wisconsin, United States
RECRUITING