OPT101 is a small peptide (a type of protein) designed to target CD40, which plays a central role in the inflammatory autoimmune response that damages the insulin-producing beta cells in people with T1D. The drug is given by subcutaneous injection, meaning it is injected just under the skin, like insulin.
The researchers hypothesize that by regulating inflammation related to CD40 immune cells (called Th40 cells), they can protect or restore the function of pancreatic beta cells, which would improve a person’s ability to produce their own insulin.
The Study
This is a Phase 2, randomized, placebo-controlled trial testing the safety and early efficacy of OPT101. The study is not a single, uniform experiment, but is divided into three parts (A, B, and C).
Part A is an open-label phase where 18 adults will receive escalating doses of OPT101 (1.0, 1.5, or 2.0 mg/kg) three times a week for two weeks to evaluate tolerance and how the drug is processed by the body. Parts B and C will be the core, placebo-controlled portions. In these parts, participants will be randomized to receive either the highest tolerated dose from Part A or a placebo for 48 weeks . The study plans to enroll a total of 72 participants across all parts. In each group, twice as many people will get the treatment as get the placebo.
Everyone enrolled will be between 18 and 50 years old, but the time since diagnosis varies by part. Part A allows diagnosis within the last 20 years, Part B is for people diagnosed between 1 and 10 years prior, and Part C is for those diagnosed within the last year. So parts A and B include both honeymooners and people with established T1D, while part C only includes honeymooners.
The primary objective of the study is to measure the "Change in C-peptide" from baseline to the end of the 48-week treatment period. C-peptide is a direct and reliable indicator of a person's own insulin production. The secondary endpoints are designed to gather more detailed information and include measuring various pharmacokinetic parameters (i.e. to understand how the drug behaves in the body).
The clinical trial officially began recruiting patients in April 2026, with a primary completion date estimated for May 2026 and a final study completion date in August 2028.
For people interested in learning more or potentially participating, the global contact for the study is Lisa Boswell, who can be reached by phone at 303-503-9954 or via email at lb@op-t.com.
The study is being conducted at several locations across the United States. The recruiting sites include the University of Alabama at Birmingham, the Diablo Clinical Research Center in California, the Barbara Davis Center at the University of Colorado Anschutz Medical Campus, the University of Chicago Medical Center, and the Rainier Clinical Research Center in Washington.
The trial is sponsored by Op-T LLC, the company developing OPT101. Support for the research into OPT101's mechanism of action has also been provided by a grant from the National Institutes of Health (NIH).
Discussion
The previous Phase 1 trial, presented at the ENDO 2025 meeting of the Endocrine Society, reported an increase in C-peptide in T1D subjects who had undetectable levels of it at the start of the study. This is a good result, and motivated this phase-II study. The results also indicated that the drug was effective in reducing inflammatory biomarkers like Th40, IFNγ, and TNFα.
The current trial introduces several changes based on the Phase 1 study. The most significant change is the route of administration, moving from an intravenous infusion to a subcutaneous injection. Intravenous infusions requires medical supervision (often by a nurse), while subcutaneous injection is the way insulin is injected (so anyone can do it).
Phase 1 studies are more focused on testing safety, and while this one generated preliminary data on efficacy, the current Phase 2 trial is designed to provide a more definitive answer on whether the drug works. This larger, longer, and placebo-controlled study will be critical to understanding the true potential of OPT101.
More Information
- ClinicalTrials.gov listing: https://clinicaltrials.gov/study/NCT06964087
- Previous Phase 1b study on ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT05428943
- Sponsor Company: Op-T LLC
- NIH Grant Information: https://taggs.hhs.gov/Detail/AwardDetail?arg_AwardNum=R01DK131196&arg_ProgOfficeCode=119
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 JDRF or JDCA news, views, policies or opinions. My kid has type-1 diabetes and has participated in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog!
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