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

NCT06964087 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: OPT101-200-10
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Type I Diabetes

Outcome Measures

Primary Outcomes (1)

• Change in C-peptide (ng/mL)

  Change from baseline to End of Study in mixed-meal stimulated C-peptide (ng/mL). The area under the time-C-Peptide curve (AUC) is calculated, and the weighted mean C-peptide is used to convert the measurement back to nanomoles per liter.

  48-week

Secondary Outcomes (5)

• PK Parameter - Maximum Concentration (Cmax)

  48-week

• PK Parameter - Time to reach maximum concentration (Tmax)

  48-week

• PK Parameter - terminal half-life t1/2

  48-week

• PK Parameter - Area under the curve

  48-week

• Propotion of subjects with change in C-peptide greater than or equal to 0.2 ng/mL

  48-week

Study Arms (3)

OPT101, 1.0mg/kg, 1.5 mg/kg and 2.0 mg/kg

EXPERIMENTAL

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

Drug: OPT101

Part B - OPT101 and Placebo to Match

EXPERIMENTAL

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

Drug: OPT101; Other: OPT101 Placebo to Match (PTM)

Part C - OPT101 and Placebo to Match

EXPERIMENTAL

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

Drug: OPT101; Other: OPT101 Placebo to Match (PTM)

Interventions

OPT101 DRUG

Subcutaneous injection.

OPT101, 1.0mg/kg, 1.5 mg/kg and 2.0 mg/kg; Part B - OPT101 and Placebo to Match; Part C - OPT101 and Placebo to Match

OPT101 Placebo to Match (PTM) OTHER

5% Dextrose (w/v)

Part B - OPT101 and Placebo to Match; Part C - OPT101 and Placebo to Match

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• \. Able and willing and able to give informed consent for the trial (separate consent must be obtained for Parts B or C).
• \. Willing to wear a continuous glucose monitor for the duration of the trial (e.g., Freestyle Libre 3).
• \. Male or female aged ≥18 to 50 years on the day of signing informed consent. 4. Diagnosis of T1DM within the last 20 years for Part A, within 1 to 10 years \[N=15 at \>5 to 10, N=12 at 1 to 5 yrs\] for Part B, within less than or equal to 1 year for Part C.
• \. For Parts B and C only, T-cell phenotype Th40 level greater than or equal to 35% of CD3+ leukocytes (performed at the OPT lab).
• \. Is medically stable based on physical examination, medical history, laboratory results, and vital signs performed at screening.
• \. Women of childbearing potential (WOCBP) must have a negative highly sensitive serum test (beta- human chorionic gonadotropin) at screening and a negative urine pregnancy test at the Visit 1 Day 1 prior to receiving the investigational product.
• \. WOCBP must agree to use one of the following methods of birth control for the duration of the clinical trial: Systemic hormonal contraceptive (oral, injected, transdermal), intrauterine device, double barrier (e.g., cervical cap or diaphragm with condom or spermicide). Men with female partners must agree to use double barrier contraception, unless their partner is using systemic hormonal contraceptives or has an intrauterine device.

You may not qualify if:

• \. Current malignancy or history of malignancy other than basal cell carcinoma or squamous cell carcinoma in situ.
• \. Has an immune deficiency syndrome (for example, severe combined immunodeficiency syndrome, T-cell deficiency syndromes, B-cell deficiency syndromes, or chronic granulomatous disease), or bone marrow or organ transplantation, or a disease associated with lymphopenia.
• \. Has chronic kidney disease of Stage 2 or higher with eGFR of \<90 mL/min/1.73m2.
• \. Is currently receiving an immuno-modulatory treatment. 5. Patients with a history of venous and arterial thromboembolic events including, but not limited to, the following:
• Deep venous thrombosis, pulmonary embolism, myocardial infarction, stroke, transient ischemic attack, or arterial insufficiency causing digital gangrene.
• Patients with recent immobilization or recent surgery.
• Patients with a history of abnormal prothrombotic laboratories such as congenital or inherited deficiency of antithrombin III, protein C, protein S, or confirmed diagnosis of antiphospholipid syndrome.
• \. Has an active infections, is prone to infections or has chronic, recurrent or opportunistic infectious disease, including but not limited to, Epstein-Barr virus, cytomegalovirus, chronic renal infection, chronic chest infection, sinusitis, recurrent urinary tract infection, Pneumocystis carinii pneumonia, aspergillosis, latent or active granulomatous infection, histoplasmosis, or coccidioidomycosis or an open, draining, or infected non-healing skin wound or ulcer.
• \. Has recent or active hepatitis A infection, current/chronic hepatitis B and hepatitis C infection, or HIV infection. Participants with immunity to hepatitis B from previous infection, defined as negative HBsAg, positive anti-HBc, and positive hepatitis B surface antibody \[anti-HBs\] or vaccination \[defined as negative HBsAg, negative anti-HBc, and positive anti-HBs\] are eligible to participate.
• \. Has a history of latent or active tuberculosis. 9. Has received a live attenuated vaccine within the last 60 days including patients who plan to receive live attenuated vaccines during the study or within 60 days after the final dose of study treatment.
• \. Patients with the following should be excluded:
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• Abnormal coagulation test at screening: prothrombin time (PT; \>14 sec), activated partial thromboplastin time (aPTT; \>32 sec) or fibrinogen level (\<190 or \>450 mg/dL).
• Abnormal liver function tests (except in the case of known Gilbert's syndrome):
• i. AST or ALT ≥3x ULN and total bilirubin ≥2x ULN ii. AST or ALT ≥5x ULN iii. Abnormal platelet counts (\<150 or \> 450 x10 to the third/uL) iv. Abnormal white blood cell counts (\< 3.0 or \>11.0 x10 to the third/uL ) v. Abnormal eGFR (\< 90 mL/min) vi. Abnormal Factor VIII (\<50% or \>150% of normal) vii. Abnormal D-Dimer (\> 500 ng/mL of fibrinogen equivalent units (FEU)) 11. Patients planning to undergo elective procedures or surgeries at any time after signing the ICF through the follow-up visit.

Sponsors & Collaborators

• Op-T LLC: lead

Study Sites (1)

Rainier Clinical Research Center

Renton, Washington, 98057, United States

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 14, 2025
• First Posted: May 9, 2025
• Study Start: May 27, 2025
• Primary Completion (Estimated): May 19, 2026
• Study Completion (Estimated): August 19, 2028
• Last Updated: September 16, 2025

Record last verified: 2025-09

Locations

US (1)