Number of Subjects in Study Archive: 48 participants (estimated enrollment)
Study Design: Interventional
Conditions: Diabetes Mellitus, Diabetes Mellitus, Type 1
Division: DEM
Duration: November 2020 - present
# Recruitment Centers: 16
Treatment: NNC0361-0041
Available Genotype Data: No
Image Summary: No
Transplant Type: None
Does it have dialysis patients: No
Access to biospecimens for TOPPLE T1D: A Multiple Ascending Dose Trial Investigating Safety, Tolerability, and Pharmacokinetics of NNC0361-0041 Administered Subcutaneously to Patients with Type 1 Diabetes Mellitus (TN27) is currently only available via collaboration. Please contact the parent study to ask about ancillary study opportunities.
Clinical Trials URL:
https://clinicaltrials.gov/ct2/show/NCT04279613
This trial is a phase-1, placebo-controlled, double-blinded within cohorts, randomized, multiple ascending dose trial with a sequential trial design to investigate the safety and tolerability of ascending subcutaneous weekly doses of NNC0361-0041 plasmid in patients with type 1 diabetes (T1D). This treatment delivers DNA into cells, where it can communicate with the immune system. Earlier lab studies suggest this treatment might retrain the immune system to stop its attack on insulin-producing beta cells. This study will determine whether NNC0361-0041 therapy is safe, and if it has an effect on the immune cells involved in T1D.
A total of 48 patients with T1D are planned to be studied in four cohorts of 12 patients (9 on active and 3 on placebo treatment). Within each cohort, sentinel enrollment will occur and safety assessment will occur before remaining participants are enrolled. The treatment period will be 12 weeks with once weekly dosing leading to 12 doses in total. Dose escalation will occur after a data safety review. An MMTT to assess insulin secretion will be completed at baseline, 1, 3, 6, and 12 months. The follow-up period will be 1 week after the last dose, as well as 4, 6, and 12 months after the first dose.
The main objective of this study is to evaluate the safety and tolerability of ascending subcutaneous weekly doses of NNC0361-0041 plasmid, a novel treatment that transfers DNA into cells to communicate with the immune system to stop its attack on insulin-producing beta cells, in patients with T1D. Participants will be monitored for adverse events and to test for safety and dosing.
The primary outcome is the number of adverse events recorded from the time of first dosing and until completion of follow-up. The secondary outcome is the change in the area under the plasma C-peptide concentration-time curve from baseline to 3 months.
Inclusion Criteria:
Exclusion Criteria:
This study is ongoing.