E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
SIMPONI® to Arrest β-cell Loss in Type 1 Diabetes |
|
E.1.1.1 | Medical condition in easily understood language |
SIMPONI® to Arrest β-cell Loss in Type 1 Diabetes |
|
E.1.1.2 | Therapeutic area | Body processes [G] - Immune system processes [G12] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10067584 |
E.1.2 | Term | Type 1 diabetes mellitus |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine if golimumab can preserve β-cell function in children and young adults with newly diagnosed T1D. |
|
E.2.2 | Secondary objectives of the trial |
-To evaluate the impact of golimumab on measures of diabetes control in this subject population. -To evaluate the off-therapy durability of golimumab on measures of diabetes control in this subject population. -To determine the safety and tolerability of golimumab in children and young adults with T1D. -To evaluate the PK and immunogenicity of golimumab in this specific subject population with T1D.
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female 6 through 21 years of age 2. Be positive for at least 1 of the following diabetes-related autoantibodies obtained at study screening: - Glutamic acid decarboxylase (GAD-65) - IA-2 - ZnT8 - ICA; or - Insulin (if obtained within 10 days of the onset of exogenous insulin therapy) 3. Have a peak stimulated C-peptide level ≥0.2 pmol/mL following a 4-hour MMTT obtained at study screening. 4. Be medically stable on the basis of physical examination, medical history, and vital signs performed at screening. If there are abnormalities, they must be consistent with the underlying illness in the study population. 5. Females of childbearing potential must have a negative serum B-human chorionic gonadotropin [B-hCG]) test at screening and a negative urine pregnancy test at the Week 0 visit. |
|
E.4 | Principal exclusion criteria |
1. Has a history of significant renal, vascular, pulmonary, gastrointestinal, neurologic, hematologic, rheumatologic, or psychiatric disease. 2. Has significant cardiovascular disease, including history of myocardial infarction, congestive heart failure, angina, abnormal electrocardiogram or abnormal stress test. 3. Has a disease associated with lymphopenia, malignancy, bone marrow or organ transplantation, lymphoproliferative disorder, immune deficiency syndrome (eg, severe combined immunodeficiency syndrome, T-cell deficiency syndromes, B-cell deficiency syndromes, or chronic granulomatous disease). 4. Has another autoimmune disease (eg, RA, pJIA, PsA, AS, MS, systemic lupus erythematosus [SLE], celiac disease [clinically symptomatic and antibody positive, ie, tissue transglutaminase IgA]). excluding clinically stable autoimmune thyroiditis whether treated or untreated. 5. Has nervous system disorder including but not limited to Guillain Barre Syndrome, multiple sclerosis (MS), or progressive multifocal leukoencephalopathy (PML). |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The Mixed-meal Tolerance Test (MMTT) - stimulated 4-hour C-peptide area under the concentration-time curve (AUC) at Week 52. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
-Change from baseline in insulin use in units per kilogram body weight per day over time. -Change from baseline in HbA1c over time. -Hypoglycemic event rates (defined as blood glucose levels (BG) of ≤70, 55, and 35 mg/dL or clinical sequelae in the absence of a BG reading) through Week 52, after Week 52 through Week 104, and the entire study. -MMTT-stimulated 4-hour C-peptide AUC over time. Proportion of subjects with treatmentemergent adverse events (AEs) and severe AEs through Week 52 and 104. -Proportion of subjects with severe infections through Week 52 and 104. -Proportion of subjects with study agent injection site reactions through Week 52. -Summary of serum golimumab concentrations and the PK profile after induction and maintenance dosing. -Incidence and titers of antibodies to golimumab. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 4 |