Type 1 Diabetes Mellitus
Conditions
Brief summary
This study is the follow-up of study IMCY-T1D 001 (EudraCT: 2016-003514-27, NCT03272269) in which patients with recent onset T1D have been treated with IMCY-0098 or placebo. At the end of the primary 6 month study, patients will be proposed to enter this follow-up study to evaluate up to 12 months (V3 - Week 48) the safety, the immune responses and the clinical parameters. The study involves a follow-up of 6 months after the end of the initial participation to the IMCY-T1D-001 study. Subjects will undergo visits at 24 weeks, 36 weeks and 48 weeks post first study product administration in study IMCY-T1D-001. For each patient, the study comprises a total of 3 visits occurring over a period of approximately 24 weeks (from study entry). The patients will undergo planned assessments and procedures as outlined in the table of study procedures.
Detailed description
In this Long-Term Follow-Up (LTFU) study, the below objectives will be assessed 36 and 48 weeks after the first injection of IMCY-0098 in the study IMCY-T1D-001, in patients treated with IMCY-0098 at three doses or placebo: Primary Objective The primary objective of this study is to assess the long-term safety. Secondary Objective The secondary objective of this study is to evaluate the clinical response to IMCY-0098 by assessing disease activity. Exploratory Objectives * To evaluate the proinsulin-specific cytolytic CD4+ T cells induced by IMCY-0098 * To evaluate the impact of IMCY-0098 on autoreactive T-cell responses specific for autoantigens expressed by islet β-cells (proinsulin, GAD65, IGRP) on the longer-term. * To evaluate the impact of IMCY-0098 on autoantibodies against GAD65, IA 2, ZnT8 and insulin * Transcriptomic analysis on mRNA extracted from samples collected for Immunogenicity
Interventions
DRUGIMCY-0098 or placebo
Long-term follow-up
Sponsors
Imcyse SA
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Treatment or Placebo
Intervention model description
Long-term follow-up (LTFU), no study treatment administered.
Eligibility
Sex/Gender
ALL
Age
18 Years to 31 Years
Healthy volunteers
No
Inclusion criteria
* All patients who were treated with IMCY-0098 or placebo in the IMCY-T1D-001 clinical trial who are willing to participate to this long-term follow-up study.
Exclusion criteria
* Ongoing pregnancy or lactation * History of or current malignancy (except excised basal cell skin cancer) * Primary or secondary immune deficiency disorders * Human Immunodeficiency virus (HIV) infection. * Ongoing treatment with immunosuppressive agents with the exception of topical or intra nasal corticosteroids. * Treatment with an investigational drug within the past 3 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse Events | Throughout the study period (24weeks) |
| Serious Adverse Events | Throughout the study period (24 weeks) |
Countries
Belgium, Denmark, France, Germany, Lithuania, Sweden, United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Cohort 1 Patients having received 50 μg IMP (2x subcutaneous \[s.c.\] injections of 25 μg \[100 μL each\]) + 3x 25 μg IMP (each as 2x s.c. injections of 12.5 μg \[50 μL each\]) in study 2016-003514-27 | 4 |
| Cohort 2 Patients having received 150 μg IMP (2x s.c. injections of 75 μg \[300 μL each\]) + 3x 75 μg IMP (each as 2x s.c injections of 12.5 μg \[150 μL each\]) in study 2016-003514-27 | 7 |
| Cohort 3 Patients having received 450 μg IMP (2x s.c. injections of 225 μg \[900 μL each\]) + 3x 225 μg IMP (each as 2x s.c injections of 112.5 μg \[450 μL each\]) in study 2016-003514-27 | 12 |
| Placebo Patients having received Placebo in study 2016-003514-27 | 7 |
| Total | 30 |
Baseline characteristics
| Characteristic | Cohort 1 | Cohort 2 | Cohort 3 | Placebo | Total |
|---|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 4 Participants | 7 Participants | 12 Participants | 7 Participants | 30 Participants |
| Age, Continuous | 25.0 years STANDARD_DEVIATION 2.7 | 26.1 years STANDARD_DEVIATION 4.3 | 25.1 years STANDARD_DEVIATION 4.8 | 26.0 years STANDARD_DEVIATION 3.9 | 25.5 years STANDARD_DEVIATION 4.1 |
| Race and Ethnicity Not Collected | — | — | — | — | 0 Participants |
| Sex: Female, Male Gender categorical Female | 1 Participants | 2 Participants | 3 Participants | 3 Participants | 9 Participants |
| Sex: Female, Male Gender categorical Male | 3 Participants | 5 Participants | 9 Participants | 4 Participants | 21 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 7 | 0 / 12 | 0 / 7 |
| other Total, other adverse events | 2 / 4 | 3 / 7 | 6 / 12 | 2 / 7 |
| serious Total, serious adverse events | 0 / 4 | 0 / 7 | 0 / 12 | 0 / 7 |
Outcome results
Primary
Adverse Events
Time frame: Throughout the study period (24weeks)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Adverse Events | 5 Number of AE |
| Cohort 2 | Adverse Events | 6 Number of AE |
| Cohort 3 | Adverse Events | 10 Number of AE |
| Placebo | Adverse Events | 4 Number of AE |
Primary
Serious Adverse Events
Time frame: Throughout the study period (24 weeks)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Serious Adverse Events | 0 Number of SAE |
| Cohort 2 | Serious Adverse Events | 0 Number of SAE |
| Cohort 3 | Serious Adverse Events | 0 Number of SAE |
| Placebo | Serious Adverse Events | 0 Number of SAE |