Otelixizumab iis being developed for the treatment of patients with autoimmune T1DM with residual beta cell function (RBCF), for the purpose of preserving RBCF in this patient population. Currently there are no approved treatments for this indication. n the USA, the approved treatments for T1DM are insulin or insulin analogues. MedDRA version: 12.1 Level: LLT Classification code 10012608 Term: Diabetes mellitus insulin-dependent
Conditions
Interventions
Product Name: Otelixizumab
Product Code: TRX4
Pharmaceutical Form: Solution for infusion
Pharmaceutical form of the placebo: Intravenous infusion
Route of administration of the placebo: Intravenous us
Sponsors
TolerX, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects will be enrolled only if they meet all of the following criteria. 1. Previously enrolled in DEFEND-1. 2. Completed the Month 24 visit of DEFEND 1; or, if the Month 24 visit was skipped, at least 24 months have elapsed since the first dose of study drug in DEFEND 1. 3. No condition that, in the investigator’s judgment, is likely to cause the subject to be unable to understand the information in the assent form or Informed Consent Document (ICD) or interfere with the subject’s ability to provide assent or informed consent. Such conditions would include, but are not limited to, psychoses or mental retardation with an IQ below 65. 4. Informed Consent Document signed by the subject if the subject is legally an adult. If the subject is legally a minor, ICD signed by the subject’s parent, both parents, or guardian and assent form signed by the subject, in accordance with the regulatory and legal requirements of the participating country. 5. No condition or situation that, in the investigator’s judgment, is likely to cause the subject to be unable or unwilling to participate in study procedures or to complete all scheduled assessments. In Germany, persons who are confined to an institution by official or judicial order are not eligible. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: See above.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to demonstrate that subjects who received an 8 day series of otelixizumab infusions in DEFEND 1 have greater preservation of endogenous insulin secretion than subjects who received placebo, as assessed by area under the concentration-time curve (AUC) for mixed meal-stimulated C peptide, at 48 months after study drug administration.;Secondary Objective: The secondary Objectives are: 1. To compare endogenous insulin secretion, as assessed by mixed meal-stimulated C peptide AUC, at 36 months after study drug administration. 2. To compare the proportion of responders, defined as subjects with glycated hemoglobin (HbA1c) = 6.5% and mean daily insulin use < 0.5 IU/kg/day, between the otelixizumab and placebo groups. 3. To assess exogenous insulin use for the otelixizumab and placebo groups and for selected subpopulations. 4. To assess glycemic control, as measured by HbA1c and subject-reported hypoglycemic events, for the otelixizumab and placebo groups and for selected subpopulations. 5. To assess the long-term safety of the 8-day series of otelixizumab infusions administered in DEFEND 1. ;Primary end point(s): The primary efficacy endpoint is change from DEFEND-1 baseline in 2-hour mixed meal-stimulated C peptide AUC at Month 48. This AUC will be normalized for time interval by dividing it by 120 minutes (the number of minutes over which it is determined), and will be adjusted by inclusion of baseline C peptide AUC as a covariate in the analysis. | — |
Countries
Denmark, Finland, Germany, Spain, Sweden, United Kingdom
Outcome results
None listed