OPT101 for Type 1 Diabetes

Overseen By CEO

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Op-T LLC

Must not be taking: Immuno-modulatory, Anticoagulants

Disqualifiers: Malignancy, Immune deficiency, Chronic infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety of a new treatment, OPT101, for people with Type 1 Diabetes. Participants will receive subcutaneous injections of OPT101 at varying doses or a placebo over a year to assess how their bodies handle the treatment. The trial is divided into parts, initially testing different doses, followed by a longer-term study using the best dose. Individuals who have had Type 1 Diabetes for up to 20 years and are willing to wear a continuous glucose monitor might be suitable for this trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in diabetes care.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are currently receiving an immuno-modulatory treatment, you would not be eligible to participate.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that OPT101 is safe and well-tolerated. In one study, participants taking OPT101 had lower levels of certain inflammation markers and experienced no serious side effects. Another report found that patients with both Type 1A and Type 1B diabetes tolerated OPT101 well, with no significant negative effects. While these results are encouraging, further research is underway to confirm OPT101's long-term safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Type 1 Diabetes?

Unlike the standard insulin therapies for Type 1 diabetes, OPT101 is unique because it potentially modifies the autoimmune response. Most treatments focus on managing blood sugar levels, but OPT101 aims to preserve the function of insulin-producing cells by targeting the underlying immune system dysfunction. This new approach could not only improve blood sugar control but also slow down the progression of the disease, offering a more comprehensive solution for people with Type 1 diabetes. Researchers are excited about OPT101 because it represents a promising shift towards addressing the root cause of the condition rather than just its symptoms.

What evidence suggests that OPT101 might be an effective treatment for Type 1 Diabetes?

Research has shown that OPT101 might help treat Type 1 Diabetes. Studies found that patients taking 2.8 mg/kg of OPT101 experienced a significant decrease in inflammation markers, such as Th40, IFNγ (a protein related to the immune system), and TNF-alpha (a protein involved in inflammation). Another study confirmed that OPT101 was safe and well-tolerated, also noting a reduction in certain inflammation markers. In this trial, participants will receive different doses of OPT101 or a matching placebo to further evaluate its potential in reducing inflammation, which is important in managing Type 1 Diabetes. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with Type 1 Diabetes who can consent to the study, are willing to wear a glucose monitor, and have stable health. Women must not be pregnant and agree to use birth control; men should also use contraception if their partners aren't. For certain parts of the trial, a specific immune cell level is required.

Inclusion Criteria

I agree to use effective birth control during the trial.

I am willing and able to consent to participate in this trial.

I am a woman who can have children and have tested negative for pregnancy.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part A: Tolerance and Pharmacokinetics

Open label exploration of tolerance and pharmacokinetics of subcutaneously administered OPT101 at increasing doses for two weeks

2 weeks

3 visits per week

Part B: Treatment

Testing the highest tolerated dose over one year in subjects with C-peptide 0.2 ng/ml

48 weeks

Part C: Optional Extension

Optional enrollment of additional subjects treated for one year with the highest tolerated dose

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• OPT101
• OPT101 Placebo to Match (PTM)

Trial Overview The study tests OPT101's safety through weekly injections over two weeks and compares its long-term effects (one year) against a placebo. Participants will receive either OPT101 or a matching placebo at one of three dosage levels.

How Is the Trial Designed?

Group I: Part C - OPT101 and Placebo to Match Experimental Treatment 2 Interventions

The highest tolerated dose in Part A will be dosed. 27 subjects (18 investigational product, 9 placebo)

Group II: Part B - OPT101 and Placebo to Match Experimental Treatment 2 Interventions

The highest tolerated dose from Part A will be tested over 48 weeks. 27 subjects (18 investigational product: 9 placebo)

Group III: OPT101, 1.0mg/kg, 1.5 mg/kg and 2.0 mg/kg Experimental Treatment 1 Intervention

Part A. 6 subjects will receive 1.0mg/kg OPT101 6 subjects will receive 1.5mg/kg OPT101 6 subjects will receive 2.0mg/kg OPT101

Who Is Running the Clinical Trial?

Op-T LLC

Lead Sponsor

Trials

3

Recruited

60+

Citations

1. medcentral.com medcentral.com/endocrinology/diabetes/type-1/encouraging-early-results-for-novel-type-1-diabetes-therapy

Encouraging Early Results for Novel Type 1 Diabetes ... Those receiving 2.8 mg/kg of OPT101, demonstrated a significant decrease in Th40, IFNγ, and tumor-necrosis-factor (TNF)-alpha (TNFα) by the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06964087

Pharmacokinetic and Early Efficacy of OPT101 in Patients ... This study will examine the safety of three times weekly SC injections of OPT101 at each of three dose levels over two weeks as well as one year of ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12546139/

MON-562 A New Drug, Opt101, Reduced Inflammatory ... Results: OPT101 proved safe and well tolerated. Using Th40 level criteria, 12 subjects identified as Type 1A and 6 as Type 1B. Residual C- ...

4. diabetesjournals.org diabetesjournals.org/diabetes/article/74/Supplement_1/2131-LB/160369/2131-LB-A-New-Drug-That-Targets-CD40-Induced-Two

2131-LB: A New Drug That Targets CD40 Induced Two Long ... T1D duration was 2 to 19 years (avg.12.8 years). In a double-blind study, 6 T1D subjects were randomly assigned to 1.1 mg/kg cohort or 2.8 mg/kg ...

5. thejdca.org thejdca.org/publications/report-library/archived-reports/2025-reports/potential-t1d-practical-cures-in-human-trials-today.html

Potential T1D Practical Cures in Human Trials Today Of 653 T1D active clinical trials, 2% qualify as potential Practical Cures. · 12 Practical Cure projects are being tested in 13 clinical trials.

6. taggs.hhs.gov taggs.hhs.gov/Detail/AwardDetail?arg_AwardNum=R01DK131196&arg_ProgOfficeCode=119

Award Information | HHS TAGGS Discovering the Mechanism of Action of a peptide drug, OPT101, that targets CD40 mediated inflammation in type 1 diabetes. Award Number: R01DK131196 ...

7. breakthrought1d.org breakthrought1d.org/pacificnorthwest/2022/12/21/t1d-clinical-trial-highlights-rainier-clinical-research-center-in-renton-wa/

Rainier Clinical Research Center in Renton WA OPT101, the new trial medication, is aimed at reducing the level of these t-cells. This is a Phase 1B trial, with lab testing producing ...