Fenofibrate (also spelled Phenofibrate) is commonly prescribed for high cholesterol and high triglycerides. In 2017, it was the 70th most commonly prescribed medication in the United States with more than eleven million prescriptions. However, it is not approved for use in children under 18. It is also sometimes prescribed for diabetic retinopathy (eye damage from long term diabetes), and (off label) for gout.
However, it also showed some promise in NOD mice (an animal model for T1D), and so one person took it "off label" when they were diagnosed with T1D as a 19 year old. They did not need to inject insulin for years after that, a huge result. I blogged on this:
https://cureresearch4type1diabetes.blogspot.com/2020/06/strong-results-from-single-case-use-of.html
with an update:
https://cureresearch4type1diabetes.blogspot.com/2021/03/possible-cures-for-type-1-in-news-march.html
The Study
The good news is that this study started in Sept-2022 and is scheduled to finish in July-2024. However, the US registry lists them as still recruiting. If this is true, completion will be delayed. The European registry does not differentiate between recruiting and active, not recruiting.
The study includes about 100 children (10 to 17 years old) divided into two groups: one gets 160 mg Fenofibrate and one gets placebo. This is a once per day pill given for a year. The study is randomized and blinded. The primary result is C-peptide and secondary results include more C-peptide numbers, insulin usage, adverse events, A1c, and several immunology measures.
European Clinical Trial Record: https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-003916-28/PL
US Clinical Trial Record: https://clinicaltrials.gov/study/NCT05909800
Discussion
This is exactly the kind of study I want to see for Fenofibrate, and I'm very excited to see its results.
There are two trials of Fenofibrate in people with T1D which are currently running. The first is designed to prevent kidney function loss rather than cure/prevent/delay T1D itself and therefore I have not been following it. It is NCT04929379. They are enrolling 40 adults with T1D for at least 8 years and have diabetic kidney disease. It started in 2022 and they hope to finish in 2025. No intermediate results have been published, and I don't expect any. But the bigger issue is that all their end points involve kidney function. They are not measuring C-peptide, A1c, or insulin use at all. So even if the medicine had a big impact, they would not see it.
The second is designed to prevent rather than cure/prevent/delay T1D itself, so I have not been following it, either. It is NCT01320345. They are enrolling 450 (!) adults with T1D and have eye problems. It started in 2016 and they hope to finish in 2025. No intermediate results have been published, and I don't expect any. But the bigger issue is that all their end points involve eye function. They are not measuring C-peptide, A1c, or insulin use at all. So even if the medicine had a big impact, they would not see it.
Personal note: I try not to get excited about potential T1D cures. I think treating all potential cures dispassionately is better for me and better for this blog. (Plus, my child was diagnosed over 22 years ago, so getting excited would not have panned out.) But I am human, and I do sometimes get excited.
I am optimistic about Fenofibrite potential to prevent T1D in the honeymoon phase. I can't explain exactly why this honeymoon treatment and no other. Maybe it is because it really seems to have worked for one person, for many years, and (as yet) there is no clinical trial results to dampen my enthusiasm.
