Can a simple pill save Insulin-Making cells in type 1 diabetes?
NCT ID NCT05594563
First seen Jun 25, 2026 · Last updated Aug 21, 2026 · Updated 3 times
Summary
This phase 2 trial tests whether the drug DFMO can protect the body's remaining insulin-producing cells in people recently diagnosed with type 1 diabetes. About 81 participants will take either DFMO or a placebo twice daily for six months. Researchers will measure insulin production and check for side effects over a year.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Difluoromethylornithine (DFMO)
- What this could lead to
- If it works, this could point toward a way to help people with new-onset type 1 diabetes keep making some of their own insulin for longer.
- What could go wrong
- This is a small, early-phase trial with only 81 people. The drug may not work better than placebo, and side effects like hearing changes are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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74 people
The number who actually took part.
- Started
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Mar 2023
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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4 to 40 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Barbara Davis Center
Aurora, Colorado, 80045, United States
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Children's Mercy Hospital
Kansas City, Kansas, 64108, United States
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IU Health Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
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MHealth Fairview Masonic Children's Hospital and Specialty Clinics
Minneapolis, Minnesota, 55454, United States
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Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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University of Chicago
Chicago, Illinois, 60637, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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