Adults 12 to 21, any sex, with Type 1 Diabetes Mellitus. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) During the On-treatment PeriodPrimary· Adverse events were reported from first dose of study treatment to 98 days after last dose, up to a maximum duration of approximately 65 weeks.
Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs led to study treatment discontinuation, and SAEs. On-treatment period is defined as from date of first administration of study treatment to 98 days after date of last administration of study treatment (including start and stop date).
Adverse Events
Group
Value
95% CI
CFZ533
28
Placebo
13
Serious Adverse Events
Group
Value
95% CI
CFZ533
4
Placebo
1
AEs leading to discontinuation of study treatment
Group
Value
95% CI
CFZ533
4
Placebo
0
Normalized Stimulated C-peptide Area Under the Curve (AUC) at Week 52Primary· At Week 52, 10 min prior to ingestion, at start of ingestion, and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal.
The mixed meal tolerance test (MMTT) has appropriate sensitivity to detect residual insulin secretion and beta cell function. In the MMTT, following an 8-10 hour overnight fast, a weight-based liquid meal provided as 6 mL/kg (maximum 360 mL) of mixed meal, ingested over 5 min with timed blood samples for glucose and C peptide determination obtained 10 min prior to ingestion (t = -10), at baseline (t = 0), and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal. The time collections for post load samples are based on the start time of the mixed meal.
Stimulated C-peptide AUC by
Group
Value
95% CI
CFZ533
0.42
0.36 – 0.49
Placebo
0.36
0.30 – 0.44
Maximum Plasma Concentration (Cmax) of CFZ533 After Intravenous (IV) AdministrationSecondary· Day 1: Pre-dose and 90 minutes after the start of the IV infusion (duration of the infusion is 30 minutes).
Cmax is defined as the maximum (peak) observed concentration following a dose. Free CFZ533 plasma concentrations were determined using a validated target-based sandwich enzyme-linked immunosorbent assay (ELISA) method.
Ctrough is the observed plasma concentration that is just prior to the beginning of, or at the end of a dosing interval. Free CFZ533 plasma concentrations were determined using a validated target-based sandwich ELISA method.
Day 1
Group
Value
95% CI
CFZ533
0.00
± 0.00
Week 4
Group
Value
95% CI
CFZ533
182
± 40.9
Week 8
Group
Value
95% CI
CFZ533
171
± 45.8
Week 12
Group
Value
95% CI
CFZ533
169
± 43.1
Week 16
Group
Value
95% CI
CFZ533
193
± 55.2
Week 20
Group
Value
95% CI
CFZ533
184
± 68.1
Week 24
Group
Value
95% CI
CFZ533
176
± 65.2
Week 28
Group
Value
95% CI
CFZ533
186
± 61.4
Time to Reach Maximum Plasma Concentration (Tmax) of CFZ533 After IV AdministrationSecondary· Day 1: Pre-dose and 90 minutes after the start of the IV infusion (duration of the infusion is 30 minutes).
Tmax is the time to reach maximum (peak) drug concentration after single-dose administration (time). Free CFZ533 plasma concentrations were determined using a validated target-based sandwich ELISA method. Theoretical sampling time points were used to report Tmax.
Group
Value
95% CI
CFZ533
1.5
1.5 – 1.5
Number of Participants With Full or Partial RemissionSecondary· Week 52
Full remission is defined by HbA1c ≤ 6.5% (48 mmol/mol) and no exogenous insulin use at Week 52.
Partial remission 1 is defined by Insulin Dose Adjusted HbA1c (IDAA1c) ≤ 9.0 at Week 52.
Partial remission 2 is defined by HbA1c \< 7.0% (53 mmol/mol) and total daily insulin dose \<0.5 units per kg per day at Week 52.
Two different criteria for partial remission were considered, and patients were assessed separately according to each criterion.
Full remission*
Group
Value
95% CI
CFZ533
0
Placebo
0
Partial remission 1
Group
Value
95% CI
CFZ533
20
Placebo
10
Partial remission 2
Group
Value
95% CI
CFZ533
15
Placebo
6
Normalized Stimulated C-peptide Area Under the Curve (AUC) at Week 72Secondary· At Week 72, 10 min prior to ingestion, at start of ingestion, and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal.
The mixed meal tolerance test (MMTT) has appropriate sensitivity to detect residual insulin secretion and beta cell function. In the MMTT, following an 8-10 hour overnight fast, a weight-based liquid meal provided as 6 mL/kg (maximum 360 mL) of mixed meal, ingested over 5 min with timed blood samples for glucose and C peptide determination obtained 10 min prior to ingestion (t = -10), at baseline (t = 0), and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal. The time collections for post load samples are based on the start time of the mixed meal.
Stimulated C-peptide AUC by
Group
Value
95% CI
CFZ533
0.40
0.34 – 0.47
Placebo
0.32
0.26 – 0.39
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 72 weeks..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The study was a Phase 2, multicounty, multicenter, non-confirmatory, investigator- and subject masked, randomized, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of CFZ533 on preservation of residual pancreatic β-cell function in new onset T1DM in pediatric and young adult subjects.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03781414 — Study of Efficacy, Safety, Tolerability, Pharmacokinetic (PK) and Pharmacodynamic (PD) of an Anti-CD40 Monoclonal Antibo
· Phase 2
· terminated
NCT03905525 — Study of Safety and Efficacy of Multiple Doses of CFZ533 in Two Distinct Populations of Patients With Sjogren's Syndrome
· Phase 2
· completed
NCT03827798 — Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa
· Phase 2
· active not recruiting
NCT03656562 — Study the Efficacy and Safety of VAY736 and CFZ533 in SLE Patients
· Phase 2
· completed
NCT03610516 — Safety, Pharmacokinetics and Preliminary Efficacy Study of CFZ533 in Patients With Lupus Nephritis.
· Phase 2
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 8 April 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04129528.