Safety and Efficacy of CBX129801 in Patients With Type 1 Diabetes

Study Overview
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Brief Summary
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The purpose of the study is to determine the beneficial effects of CBX129801 (PEGylated synthetic human C-peptide) following weekly subcutaneous administration for 12 months in type 1 diabetes mellitus patients (T1DM) with mild to moderate diabetic peripheral neuropathy (DPN).

Official Title
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A Phase 2b, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of CBX129801 (Ersatta™), Long-Acting Synthetic C-Peptide, in Type 1 Diabetes Mellitus Subjects With Mild to Moderate Diabetic Peripheral Neuropathy

Conditions
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Diabetic Peripheral Neuropathy

Intervention / Treatment
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* Drug: CBX129801


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Give informed consent; 18-65 years old; Have type 1 diabetes mellitus for a minimum of 5 years, with a stable diabetic regimen (for at least 3 months); Have clinical signs of diabetic peripheral neuropathy at screening; Have abnormal sural nerve conduction observed bilaterally during screening; Be C-peptide deficient; Be in good general health (besides having type 1 diabetes mellitus); Practice effective contraception during and for at least 12 weeks after study participation; Have a body mass index (BMI) ≥18.0 and <35.0 kg/m2. Key Exclusion Criteria: Any significant cardiovascular, hematological, lymphatic, immunologic, urologic, dermatologic, psychiatric, renal, hepatic, pulmonary, endocrine (except for diabetes mellitus), central nervous, gastrointestinal, or other major disease; Unstable or inadequate glucose control; Any clinically significant laboratory value at screening; Occurrence of a severe, unexplainable hypoglycemic event (defined as requiring the assistance of another individual) within 6 months of Day 0, or recurrent episodes of non-severe hypoglycemia (≥3 per week on average) that are deemed clinically significant by the Investigator; Have had an islet cell, kidney, and/or pancreas transplant; If female, is pregnant or lactating; History of alcohol or substance abuse within 2 years; Positive screen for hepatitis B, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody; Initiation of treatment or change of dose of medication that could affect peripheral nerve function within 60 days; Previous treatment with CBX129801 or unmodified C-peptide; Receipt of an investigational product or therapeutic device, or participation in a drug research study, within a period of 30 days; Chronic use of oral steroids or use of Ampyra (dalfampridine) within 60 days.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CBX129801 High Dose<br>Solution for injection, 2.4 mg, weekly for 52 weeks | Drug: CBX129801<br> <br> |
| Experimental: CBX129801 Low Dose<br>Solution for injection, 0.8 mg, weekly for 52 weeks | Drug: CBX129801<br> <br> |
| Placebo Comparator: Placebo<br>Solution for injection, vehicle with no active, weekly for 52 weeks | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Bilateral change in sensory nerve conduction velocity |  | Predose and 12 months post dose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Vibration perception threshold |  | Predose and 6 and 12 months post dose | 
| Clinical composite score |  | Predose and 6 and 12 months post dose | 
| Pain Intensity due to DPN |  | Predose and 12 months post dose | 
| Sexual function questionnaires |  | Predose and 6 and 12 months post dose | 
| Quality of life questionnaire |  | Predose and 12 months post dose |