OPF-310 for Type 1 Diabetes

Overseen By Yuka Ishizaki

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Otsuka Pharmaceutical Factory, Inc.

Must be taking: Insulin

Must not be taking: Anticoagulants, Corticosteroids, Acetaminophen, Hydroxycarbamide

Disqualifiers: Insulin resistance, HIV, Hepatitis, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

13 Participants Needed

Trial Summary

What is the purpose of this trial?

This study is First In Human study for Encapsulated Porcine Islet Cells for Xenotransplantation (OPF-310). The purpose of this study to assess the safety, tolerability, and efficacy of OPF-310 transplantation and to define the recommended Phase 2 dose (RP2D) in adult subjects with unstable Type 1 Diabetes Mellitus (T1DM) and a level 3 (severe) hypoglycemic episode at least three times within the 1 year prior to enrollment despite treatment with a closed loop system (CLS) for at least 6 months.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does exclude those on certain treatments like warfarin, corticosteroids, and non-insulin hypoglycemic medications. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the treatment OPF-310 for Type 1 Diabetes?

Research shows that a combination of low-dose antithymocyte globulin (ATG) and pegylated granulocyte colony-stimulating factor (G-CSF) can help preserve beta-cell function in people with Type 1 Diabetes, which is important for managing the disease. This suggests that similar treatments might be effective in maintaining insulin production in patients. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults aged 35-65 with unstable Type 1 Diabetes Mellitus, who've had severe low blood sugar episodes despite using advanced glucose monitoring and insulin pumps. Participants must have a history of diabetes for at least 5 years, an HbA1c level between 7.5% and 9%, very low or undetectable C-peptide levels, and agree to lifetime follow-up.

Inclusion Criteria

I can sign and understand the consent form.

I am using birth control as required by local laws for clinical study participants.

I agree to lifetime follow-up after my transplant.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive OPF-310 transplantation to assess safety, tolerability, and efficacy

52 weeks

Regular visits for monitoring and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Treatment Details

Interventions

• OPF-310

Trial Overview The study tests the safety and effectiveness of OPF-310, encapsulated pig-derived pancreatic cells transplanted into humans with Type 1 Diabetes to improve blood sugar control. It aims to determine the proper dose for Phase 2 trials in patients with severe hypoglycemia history.

Participant Groups

Group I: OPF-310 Experimental Treatment 1 Intervention

13 patients will be transplanted OPF-310.

Who Is Running the Clinical Trial?

Otsuka Pharmaceutical Factory, Inc.

Lead Sponsor

Trials

2

Recruited

100+

References

1. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of faster aspart in insulin pumps in children and adolescents with type 1 diabetes mellitus: A single-center study with real-world data. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Anti-thymocyte globulin/G-CSF treatment preserves β cell function in patients with established type 1 diabetes. [2022]

3. Denmark pubmed.ncbi.nlm.nih.gov

Prevention of diabetes: effect of mycophenolate mofetil and anti-CD25 on onset of diabetes in the DRBB rat. [2016]

4. United States pubmed.ncbi.nlm.nih.gov

Antithymocyte Globulin Plus G-CSF Combination Therapy Leads to Sustained Immunomodulatory and Metabolic Effects in a Subset of Responders With Established Type 1 Diabetes. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Potential role of non-insulin adjunct therapy in Type 1 diabetes. [2022]