Study of IMCY-0098 in Patients With Recent Onset Type 1 Diabetes

Study Overview
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Brief Summary
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This clinical study will evaluate the safety of an innovative approach expected to be disease-modifying by stopping the auto-immune-mediated destruction of islet β-cells in the pancreas. Three doses of the investigational product will be tested in successive cohorts. Although safety is the first objective of this study, we will gather efficacy data and perform a set of immunological tests to further understand the mechanism of action of this new approach in young adults with recent onset type 1 diabetes.

Official Title
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A Phase I Placebo-controlled, Double-blind, Dose Escalation Clinical Trial to Evaluate the Safety and Immune Responses of Imcyse's IMCY-0098 in Patients With Recent Onset Type 1 Diabetes

Conditions
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Type 1 Diabetes Mellitus

Intervention / Treatment
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* Drug: IMCY-0098
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female 18 to 30 years of age Initial diagnosis of Type 1 diabetes according to ADA/WHO criteria within the past 6 months Insulin requirement, as determined by the investigator Presence of at least one autoantibody (GAD65, IA-2, or ZnT8) Fasting C-peptide at screening >0.2 nmol/L and/or stimulated C-peptide ≥ 0,4 nmol/L. HLADR3-positive and/or HLADR4-positive Willingness to undergo the insulin treatment prescribed by the physician Body mass index (BMI) between 17-28 kg/m2 at screening Fully informed written consent obtained Males with reproductive potential should use barrier method of contraception (condom) from screening up to 90 days after last treatment with investigational product. Women of childbearing potential should use an highly effective contraception method from screening and for the whole duration of the study. Exclusion Criteria: Ongoing or planned pregnancy during the whole duration of the study or lactation Presence of significant medical conditions in particular chronic liver condition, chronic hematological disease, renal dysfunction of grade 2 or more according to the World Health Organization (WHO) Toxicity Scale . Has any current signs or symptoms of infection at entry or within 2 weeks of entry or has received intravenous antibiotics within 2 months prior to the first planned administration of the study product Has received any live, attenuated vaccine within 3 months prior to the first planned administration of the study product (i.e. oral poliomyelitis vaccine, measles-mumps-rubella vaccine, yellow fever vaccine, Japanese encephalitis vaccine, dengue vaccine, rotavirus vaccine, varicella vaccine, live-attenuated zoster vaccine, Bacillus Calmette-Guérin [BCG] vaccine, oral typhoid vaccine) History of, or current malignancy (except excised basal cell skin cancer) Clinical evidence of a diabetes-related complication that could interfere with patient's participation/completion of study Primary or secondary immune deficiency disorders Human Immunodeficiency virus (HIV), chronic hepatitis B virus (HBV) or hepatitis C virus (HCV) infection Presence at screening of abnormal laboratory values grade 2 or more according to the World Health Organization (WHO) Toxicity Scale Anti-diabetic treatments other than insulin in the week prior to first study drug administration Ongoing treatment with immunosuppressive agents or treatment within the past year with the exception of topical or intra nasal corticosteroids. Treatment with immunotherapy within the past 3 months Treatment with an investigational drug within the past 3 months Patients with a known hypersensitivity to any component of the drug product should be excluded from the study Patients under treatment with statins at the time of screening.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 30 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: Sequential Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1, low dose<br>4 SC injections of IMCY-0098 or Placebo | Drug: IMCY-0098<br>* Small synthetic peptide for SC admin. Solvent: alum hydroxide<br>* Other names: Imotope;Other: Placebo<br>* Solvent: alum hydroxide<br>|
| Experimental: Cohort 2, medium dose<br>4 SC injections of IMCY-0098 or Placebo | Drug: IMCY-0098<br>* Small synthetic peptide for SC admin. Solvent: alum hydroxide<br>* Other names: Imotope;Other: Placebo<br>* Solvent: alum hydroxide<br>|
| Experimental: Cohort 3, high dose<br>4 SC injections of IMCY-0098 or Placebo | Drug: IMCY-0098<br>* Small synthetic peptide for SC admin. Solvent: alum hydroxide<br>* Other names: Imotope;Other: Placebo<br>* Solvent: alum hydroxide<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of all adverse events reported for subjects | Safety assessed through measurement and comparison of any reactions or hypersensitivity to IMCY-0098 injection vs placebo. Number of adverse events will also be compared between groups with the addition of safety monitoring blood tests | up to 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of residual beta cell function and markers of metabolic control | Measured by a change in stimulated C-peptide production, daily insulin usage, glycated haemoglobin levels and glucose levels and excursions from baseline and between groups | up to 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diabetes Mellitus type 1, Autoimmune disease, Immunotherapy, Diabetes treatment, Residual beta cell function, Adult patients