A new clinical trial is exploring whether two different immune therapies, used alone or in combination, can delay or prevent the onset of T1D in people who are at high risk, but have not yet been diagnosed with T1D. The study will test a cell-based therapy using a patient's own cells (regulatory T cells) and and a drug called rituximab.
It involves drawing a person’s blood, isolating a specific type of immune cell, multiplying these cells in a laboratory, and then infusing them back into the same person. These regulatory T cells are the body’s natural “peacekeepers,” responsible for preventing the immune system from attacking its own tissues. Rituximab is a well-established prescription medication that works by targeting and removing a different type of immune cell, known as a B cell. B cells play a role in the autoimmune attack that causes T1D.
The regulatory T cells will be administered through an infusion, while rituximab will be given as an injection. The trial will involve multiple infusions and injections over a period of time. This is not a new treatment; both regulatory T cells and rituximab have been used in other diseases. However, combining them to prevent type 1 diabetes is novel.
Rituximab has been approved by the FDA for many years to treat other autoimmune conditions, such as rheumatoid arthritis, as well as certain types of cancer. Its safety profile is well understood from its use in these other conditions. Treg therapy is still experimental and not approved for general use. However, it has been studied in human clinical trials for more than a decade for T1D and other autoimmune diseases. To date, these studies have generally shown it to be safe.
The Study
This study is a randomized, controlled trial, participants will be randomly assigned to different treatment groups. The trial will include approximately 150 children aged 6 to 16 who already have two autoantibodies associated with type 1 diabetes and therefore are at very high risk of developing type 1 diabetes. They will be followed for five years.
There will be four groups in the trial, which cover all the combinations of treatment and placebo:
1. Group I will receive both regulatory T cells and Rituximab.
2. Group II will receive regulatory T cells and a placebo.
3. Group III will receive Rituximab and a placebo.
4. Group IV will receive two placebos, one simulating Rituximab, the other the cell infusion.
The primary endpoints of the study are the number of days from the start of the treatment to the first signs of high blood sugar levels and the number of adverse events reported.
The secondary endpoints include C-peptide levels, daily insulin dose, and the number of participants in remission.
Families interested in the trial can contact the study team for more information:
Marta Druch, Clinical Trial Director Email: m.druch@poltreg.com Phone: +48 731 471 845
Grzegorz Orlik, Medical Director Email: g.orlik@poltreg.com Phone: +48 790 680 020
The trial is being conducted at eight locations across Poland:
- Bialystok, Poland (Recruiting)
- Gdansk, Poland (Not yet recruiting)
- Katowice, Poland (Not yet recruiting)
- Lodz, Poland (Not yet recruiting)
- Lublin, Poland (Not yet recruiting)
- Opole, Poland (Not yet recruiting)
- Rzeszów, Poland (Not yet recruiting)
- Wroclaw, Poland (Not yet recruiting)
More details, including full eligibility criteria, are available on the clinical trial registry: https://clinicaltrials.gov/study/NCT06688331
This trial is funded by PolTREG S.A., a company specializing in cellular therapies.
Discussion
One thing that I like about this study is that they are testing two different treatments, but all the possibilities (both treatments, one treatment, the other treatment, and no treatments). This means it should be easy to see if either treatment is helpful and if the treatments help each other be more effective than either alone.
Running a clinical trial on children ages 6 to 16 is also good, since that is the age that most people with T1D are diagnosed.
The long duration of the trial, five years, means we will be waiting a long time for results. This is unfortunate, but if the goal is prevention, I do not see any other way to run the clinical trial.
Related Trials
Study NCT00279305, a Phase 2 study which completed in 2009 with 87 honeymooner T1D participants aged 8-45, investigated Rituximab. The study found that rituximab preserved C-peptide generation as compared to placebo.
Study NCT01280682, completed in 2018 in China. It enrolled 120 honeymooner T1D patients aged 8-70. This trial showed that rituximab treatment slowed the decline in C-peptide compared to insulin-only treatment and resulted in lower HbA1c levels at six months.
Study NCT03929601 is an ongoing Phase 2 study with 74 honeymooner T1D participants aged 8-45. This study compares sequential Rituximab and Abatacept therapy to Rituximab alone, aiming to improve C-peptide response at 24 months. Secondary endpoints include safety, insulin use, and HbA1c. It is expected to finish in March 2029.
Previous blogging: https://cureresearch4type1diabetes.blogspot.com/2024/08/combo-of-rituximab-and-abatacept-starts.html
Study NCT07041268, an ongoing Phase 2 trial recruiting an estimated 116 honeymooner T1D adolescents (12-17 years old) in Russia, is examining repeated Rituximab. The primary endpoint is the change in mean C-peptide levels, with secondary goals including insulin dose and HbA1c. This study anticipates completion by March 2028.
More Information
For more information about this clinical trial, you can visit the following links:
- FDA's Clinical Trials Registry: https://clinicaltrials.gov/study/NCT06688331
- EU Clinical Trials Registry: https://euclinicaltrials.eu/search-for-clinical-trials/?lang=en&EUCT=2023-505226-33-00
- Company Website: https://www.poltreg.com
- Press Release Announcing the Start of This Clinical Trial: https://poltreg.com/poltreg-doses-first-patient-in-phase-ii-trial-for-pre-symptomatic-type-1-diabetes-with-ptg-007/
- Web Page Created by the Researchers to Explain the Study to Participants: https://www.poltreg.com/study-explanation
- Wikipedia Page for Rituximab: https://en.wikipedia.org/wiki/Rituximab
- DiaTribe Article on This Treatment: https://diatribe.org/rituximab-and-regulatory-t-cells-for-type-1-diabetes
- JDCA Article on This Treatment: https://www.jdca.org/rituximab-and-regulatory-t-cells-for-type-1-diabetes
Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official BreakthroughT1D or JDCA news, views, policies or opinions. I sometimes use generative AI ("chatbots") to generate draft blogs, parts of blogs, or drafter alternate wordings for these blogs. I always review every part of every published blog to ensure that it is saying what I want, in the tone that I want, truthfully, and accurately. My kid has type-1 diabetes and has participated in clinical trials, which might be discussed here. I am obese and right on the border of T2D and therefore may be taking drugs for those conditions. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog!
