DFMO in Children With Type 1 Diabetes

• Status: Completed
• Trial end date: 2020-01-06
• Target enrollment: 0
• Participant gender: All

Summary

This study is a multicenter, double-blind, placebo-controlled, 2:1 randomly assigned, phase 1 clinical trial for individuals with type 1 diabetes. It is a blinded dose-ranging study enrolling patients with new onset type 1 diabetes with documented continued residual C-peptide production. After a 4 week screening and run-in period during which eligibility will be determined and glycemic control optimized, subjects will have a 3-month double-masked treatment period with either DFMO or placebo. After a 3 month wash-out period the durability of effect will be assessed. Subjects will be randomly assigned (6 to DFMO; 3 to placebo in each cohort) to 1 of 4 sequential dose cohorts.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Indiana University
• Treatments: Eflornithine

Criteria

Inclusion Criteria:

1. Males and females 12-40 years of age with a clinical diagnosis of T1D within 2 - 8 months after diagnosis at the time of visit 2.

2. Random non-fasting C-peptide level of >0.2 pmol/mL at visit 1.

3. Positive for any one of the following diabetes-related autoantibodies (mIAA, GADA, IA-2A, or ZnT8A)

4. Treatment naïve of any immunomodulatory agent

5. Normal hearing at screening, defined as acceptable results of pure-tone audiometry (<20 decibel [dB] baseline thresholds for frequencies 250, 500, 1000, and 2000 Hz

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. 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. Female participants of child-bearing age must not be pregnant and agree to use an effective form of birth control or be abstinent during the study period.

10. BMI >95% for age and sex