Clinical Trial Assessing the GNbAC1 in Patients With Onset of Type 1 Diabetes Within 4 Years (RAINBOW-T1D)

Randomised, Double-Blind, Placebo-Controlled Study to Investigate GNbAC1 in Patients With Onset of Type 1 Diabetes Within 4 Years

The monoclonal antibody GNbAC1 targets the envelope protein (Env) of the human endogenous Multiple Sclerosis associated RetroVirus (MSRV), which could play a critical role in different autoimmune disorders, notably type 1 diabetes (T1D).

This study is a multicentre study evaluating for the first time the safety and efficacy of GNbAC1 in T1D subjects for a first bouble-blind period of 20 weeks followed by an optional open-label period of 24 weeks. The primary objective of the study is to assess the safety and tolerability of six consecutive 4-weekly doses of GNbAC1 in subjects with T1D. Secondary objectives are to determine the pharmacodynamic response to GNbAC1 on biomarkers of T1D.

Study Overview

• Status: Completed
• Conditions: Diabetes Mellitus Type 1
• Intervention / Treatment: Drug: GNbAC1; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 64
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Macquarie University, New South Wales, Australia
      • Macquarie University Hospital
    • Merewether, New South Wales, Australia
      • AIM Centre
    • St Leonards, New South Wales, Australia
      • Northern Sydney Local Health District - Royal North Shore Hospital
  • Queensland
    • Ipswich, Queensland, Australia
      • Ipswich Research Centre
    • South Brisbane, Queensland, Australia
      • Mater Misericordiae Ltd and Mater Medical Research Institute Limited
    • Southport, Queensland, Australia
      • Gold Coast Hospital and Health Service
  • South Australia
    • Adelaide, South Australia, Australia
      • Southern Adelaide Local Health Network - Repatriation General Hospital
    • Elizabeth Vale, South Australia, Australia
      • Northern Adelaide Local Health Network Incorporated, operating as Lyell McEwin Hospital
  • Victoria
    • Box Hill, Victoria, Australia
      • Eastern Health
    • Fitzroy, Victoria, Australia
      • St Vincent's Hospital (Melbourne) Limited
    • Geelong, Victoria, Australia
      • Barwon Health - University of Geelong
    • Heidelberg, Victoria, Australia
      • Heidelberg Repatriation Hospital
  • Western Australia
    • Nedlands, Western Australia, Australia
      • Keogh Institute of Medical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 51 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Main Inclusion Criteria:

• Subjects diagnosed with type 1 diabetes in the 4 years prior to signed informed consent;
• Peak stimulated C-peptide of ≥0.2 nmol/L during a mixed meal tolerance test performed during the Screening period;
• 18 to 55 years of age (both inclusive);
• Body weight >40 to ≤100 kg;
• Subjects positive for at least one diabetes-associated autoantibody (insulin, glutaminic-acid-decarboxylase-65 [GAD-65], tyrosine phosphatase-related antigen 2 [IA-2], ZnT8 or islet-cell antibody [ICA]).

Main Exclusion Criteria:

• Subjects with type 2 diabetes;
• Pregnant and nursing women.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Monthly IV repeated dose	Drug: Placebo; Monthly IV repeated dose
Experimental: GNbAC1; Monthly IV repeated dose	Drug: GNbAC1; Monthly IV repeated dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability of GNbAC1 in patients with recent onset type 1 diabetes: Serious Adverse Events (SAE) and Adverse Events (AE)	Serious Adverse Events (SAE) and Adverse Events (AE)	Week 1 to 24/48

Collaborators and Investigators

• Sponsor: GeNeuro Australia PTY Ltd
• Collaborators: Southern Star Research Pty Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): June 14, 2017
• Primary Completion (Actual): September 1, 2018
• Study Completion (Actual): May 7, 2019

Study Registration Dates

• First Submitted: June 1, 2017
• First Submitted That Met QC Criteria: June 6, 2017
• First Posted (Actual): June 7, 2017

Study Record Updates

• Last Update Posted (Actual): October 20, 2020
• Last Update Submitted That Met QC Criteria: October 19, 2020
• Last Verified: May 1, 2019

More Information

Terms related to this study

• Keywords: Monoclonal antibody; T1D; Diabetes Mellitus, Type 1; GNbAC1; Temelimab; Multiple Sclerosis associated retrovirus MSRV; Human Endogenous Retrovirus Type W (HERV-W)
• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: GNC-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No