DFMO in Children With Type 1 Diabetes


Sponsored by Indiana University 

Focus Areas: Beta Cell Therapies

What is This Study?

In this phase 1 study, alpha difluoromethylornithine (DFMO), a medication most commonly used to treat sleeping sickness is being given to participants aged 12-40 who have been diagnosed with type 1 diabetes within the past two to eight months. DFMO has been shown to preserve beta cell production and delay diabetes onset in mice.

Why do We Care?

This is an entirely new type of medication for the preservation of beta cells. As a Phase 1 study, the primary outcome is to determine what a safe level of DFMO is up to 750 mg per day. The secondary outcomes will be to measure the biomarkers of beta cell stress, as well as measures of insulin production/glycemia. If this drug is proven safe at higher doses, it will be an exciting and novel way approach to slowing and preventing the development of type 1 diabetes (T1D).

When Might we See Results?

Results for this study are expected in late 2020.

Further Reading

Stay tuned for in-depth coverage from Beyond Type 1 on the results of this study. To learn more about this trial click here

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WRITTEN BY BT1 Editorial Team, POSTED 03/23/20, UPDATED 07/25/23

This piece was authored collaboratively by the Beyond Type 1 Editorial Team.