Clinical trial NCT02803918: A Study on Safety, Pharmacokinetics and Pharmacodynamics of Lixisenatide in Pediatric Patients With Type 2 Diabetes Mellitus (T2DM)
Official title: Randomized, Double-blind, Placebo-controlled, Dose Escalation, Study on Safety, Pharmacokinetics and Pharmacodynamics of Lixisenatide in Pediatric Patients With Type 2 Diabetes Mellitus Not Adequately Controlled With Metformin and/or Basal Insulin
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Type 2 Diabetes Mellitus
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2016-06-17
Enrollment: 23
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Primary Objective:

To demonstrate safety of 14-day repeated lixisenatide doses with 3 ascending doses as compared to placebo in pediatric patients with T2DM.

Secondary Objectives:

* To evaluate plasma concentrations of lixisenatide after repeated doses (3 ascending doses) and pharmacokinetic parameters of repeated lixisenatide doses in pediatric patients with T2DM.
* To evaluate the change from baseline in fasting and post-prandial plasma glucose concentrations during a standardized meal test after 3 ascending repeated doses of lixisenatide in comparison to placebo.

Detailed description: The total study duration will be up to 10 weeks that includes a 6-week treatment period with dose escalation every 2 weeks.

Eligibility criteria: Inclusion criteria :

* Male or female patients aged ≥10 and \<18 years old (at least 4 patients below 16 years old).
* Body mass index (BMI) \>85th percentile for age and gender; BMI ≤50 kg/m2.
* Male and female patients with documented T2DM insufficiently controlled with metformin at a stable dose and regimen for 8 weeks prior to randomization and/or basal insulin at stable dose (± 20%) and regimen for 8 weeks prior to randomization. The exact individual metformin dose will be selected according to local regulation and to the investigator's medical judgment.
* Glycated hemoglobin (HbA1c) \>6.5% and ≤11% at screening.

Exclusion criteria:

* If female, ongoing pregnancy (defined as positive serum pregnancy test), breast-feeding.
* Sexually active postmenarchal female patient who does not agree to use an adequate and highly effective method of contraception throughout the study duration and according to local regulation (ie, hormonal contraception, condom, etc.).
* Diabetes other than T2DM.
* Fasting plasma glucose \>250 mg/dL (\>13.9 mmol/L) at screening.
* Use of other oral or injectable antidiabetic or hypoglycemic agents other than metformin and basal insulin (eg, alpha glucosidase inhibitor, glucagon-like peptide (GLP-1) receptor agonist, dipeptidyl peptidase-IV (DPP-IV) inhibitors, short-acting insulin etc.) within 1 month prior to the screening visit.
* History of unexplained pancreatitis, chronic pancreatitis, pancreatectomy, stomach/gastric surgery, inflammatory bowel disease.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Primary outcomes:
measure: Number of patients with adverse events (AEs)
timeFrame: Up to 10 weeks
measure: Number of patients with treatment-emergent adverse events (TEAEs)
timeFrame: Up to 10 weeks
measure: Number of patients with anti-lixisenatide antibodies
timeFrame: Up to 10 weeks
Secondary outcomes:
measure: Assessment of pharmacokinetic (PK) parameters: lixisenatide plasma concentration
timeFrame: Day 14, Day 28 and Day 42
measure: Assessment of PK parameters: maximum concentration (Cmax)
timeFrame: Day 42
measure: Assessment of PK parameters: time to reach Cmax (Tmax)
timeFrame: Day 42
measure: Assessment of PK parameters: area under up to last concentration (AUClast)
timeFrame: Day 42
measure: Assessment of PK parameters: area under curve (AUC)
timeFrame: Day 42
measure: Assessment of pharmacodynamic parameter: plasma glucose AUC-0-4.5 hours
timeFrame: Day 14, Day 28 and Day 42
Other outcomes:
Locations:
city: Baton Rouge
country: United States
facility: Investigational Site Number 840002
state: Louisiana
city: New Orleans
country: United States
facility: Investigational Site Number 840006
state: Louisiana
city: Philadelphia
country: United States
facility: Investigational Site Number 840009
state: Pennsylvania
city: Lufkin
country: United States
facility: Investigational Site Number 840007
state: Texas
city: Phoenix
country: Mauritius
facility: Investigational Site Number 480001
city: Monterrey
country: Mexico
facility: Investigational Site Number 484001
city: Puebla
country: Mexico
facility: Investigational Site Number 484002
city: Cape Town
country: South Africa
facility: Investigational Site Number 710001
city: Barcelona
country: Spain
facility: Investigational Site Number 724002
city: Ankara
country: Turkey
facility: Investigational Site Number 792002
city: Izmir
country: Turkey
facility: Investigational Site Number 792001
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References:
citation: Barrientos-Perez M, Hsia DS, Sloan L, Nell H, Mungur O, Hovsepian L, Schmider W, Spranger R, Yang N, Niemoeller E. A study on pharmacokinetics, pharmacodynamics and safety of lixisenatide in children and adolescents with type 2 diabetes. Pediatr Diabetes. 2022 Sep;23(6):641-648. doi: 10.1111/pedi.13343. Epub 2022 Apr 24.