Safety and Efficacy of CBX129801 in Patients With Type 1 Diabetes

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

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).

Study Overview

• Status: Completed
• Conditions: Diabetic Peripheral Neuropathy
• Intervention / Treatment: Drug: CBX129801

• Study Type: Interventional
• Enrollment (Actual): 250
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
  • Quebec
    • Laval, Quebec, Canada, H7T 2P5
• Sweden
  • Göteborg, Sweden, 413 45
  • Linköping, Sweden, 581 85
  • Malmo, Sweden, 20502
  • Stockholm, Sweden, 171 76
  • Umea, Sweden, 90185
• United States
  • California
    • Escondido, California, United States, 92026
    • Fresno, California, United States, 93720
    • Irvine, California, United States, 92618
    • Los Angeles, California, United States, 90036
    • San Diego, California, United States, 92161
    • San Francisco, California, United States, 94110
    • Torrance, California, United States, 90502
    • Tustin, California, United States, 92780
    • Walnut Creek, California, United States, 94598
  • Georgia
    • Atlanta, Georgia, United States, 30318
    • Decatur, Georgia, United States, 30033
  • Idaho
    • Idaho Falls, Idaho, United States, 83404
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
  • Montana
    • Butte, Montana, United States, 59701
  • Nebraska
    • Omaha, Nebraska, United States, 68131
  • Nevada
    • Las Vegas, Nevada, United States, 89102
  • New York
    • New York, New York, United States, 10032
  • Texas
    • Dallas, Texas, United States, 75230
    • Houston, Texas, United States, 77030
    • Houston, Texas, United States, 77074
    • San Antonio, Texas, United States, 78229

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 63 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Cebix Incorporated
• Investigators: Dennis Kim, MD, Chief Medical Officer

Publications and helpful links

General Publications

• Wahren J, Foyt H, Daniels M, Arezzo JC. Long-Acting C-Peptide and Neuropathy in Type 1 Diabetes: A 12-Month Clinical Trial. Diabetes Care. 2016 Apr;39(4):596-602. doi: 10.2337/dc15-2068. Epub 2016 Feb 16.

Study record dates

Study Major Dates

• Study Start: October 1, 2012
• Primary Completion (Actual): November 1, 2014
• Study Completion (Actual): January 1, 2015

Study Registration Dates

• First Submitted: September 5, 2012
• First Submitted That Met QC Criteria: September 5, 2012
• First Posted (Estimate): September 7, 2012

Study Record Updates

• Last Update Posted (Estimate): January 30, 2015
• Last Update Submitted That Met QC Criteria: January 28, 2015
• Last Verified: January 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Endocrine System Diseases; Diabetes Complications; Diabetes Mellitus; Neuromuscular Diseases; Peripheral Nervous System Diseases; Diabetic Neuropathies
• Other Study ID Numbers: CBX129801-DN-201