NCT06964087

Pharmacokinetic and Early Efficacy of OPT101 in Patients With Type 1 Diabetes Mellitus

A 48-week Phase 2 Multicenter Controlled Pharmacokinetic and Pilot Efficacy Trial of Subcutaneous OPT101 in Patients With Recent Onset Type 1 Diabetes Mellitus

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 72 (estimated)
• Sponsor: Op-T LLC · Industry
• Sex: All
• Age: 18 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

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 treatment with SC OPT101 or placebo to match at a single dose level.

Detailed description

This multipart study begins with an open label exploration of tolerance and pharmacokinetics of subcutaneously (SC) administered OPT101, given at increasing doses of 1.0, 1.5, or 2.0 mg/kg three times per week for two weeks to separate sequential cohorts of six adult patients (n=18 total) diagnosed with type 1 diabetes mellitus within 20 years of screening. In this Part A, serum C-peptide levels will not be a consideration. In Part B, the highest tolerated dose will then be tested over one year in a total of n=27 subjects having C-peptide 0.2 ng/ml, with 12 diagnosed within 1 to 5 years of screening, and 15 diagnosed within \>5 to 10 years. Randomization to treatment or control will be in a 2:1 ratio. An optional Part C will enroll an additional n=27 subjects having C-peptide 0.2 ng/ml who are 1 year from diagnosis with type 1 diabetes. Subjects will be randomized to treatment or control in a 2:1 ratio and will be treated for one year.

Conditions

• Type I Diabetes

Interventions

Type	Name	Description
DRUG	OPT101	Subcutaneous injection.
OTHER	OPT101 Placebo to Match (PTM)	5% Dextrose (w/v)

Timeline

• Start date: 2026-04-10
• Primary completion: 2026-05-31
• Completion: 2028-08-19
• First posted: 2025-05-09
• Last updated: 2026-04-06

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study