Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Saxagliptin as Monotherapy in Pediatric Patients With Type 2 Diabetes

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01204775
• Date of registration: 16/09/2010
• Prospective Registration: Yes
• Primary sponsor: AstraZeneca
• Public title: Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Saxagliptin as Monotherapy in Pediatric Patients With Type 2 Diabetes
• Scientific title: A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Saxagliptin (BMS-477118) as Monotherapy in Pediatric Patients With Type 2 Diabetes
• Date of first enrolment: June 2011
• Target sample size: 26
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01204775
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Canada, India, Israel, Italy, Russian Federation, South Africa, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: Bristol-Myers Squibb
• Affiliation: Bristol-Myers Squibb

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male and female patients eligible if 10 years of age, up to 17 years and 32 weeks of
age at the time of randomization, diagnosed as having type 2 diabetes prior to study
enrollment.

- HbA1c =7.0% and =10.5%

- Body weight = 30 kg.

- BMI > 85th percentile

Age and Reproductive Status a) Women of childbearing potential (WOCBP) and men must be
using an acceptable method of contraception to avoid pregnancy throughout the study and for
up to 4 weeks after the last dose of investigational product in such a manner that the risk
of pregnancy is minimized. The decision for appropriate methods to prevent pregnancy should
be determined by discussions between the investigator and the study subject.

b) WOCBP must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or
equivalent units of HCG) within 72 hours prior to the start of investigational product. c)
Women must not be breastfeeding. d) Sexually active fertile men must use effective birth
control if their partners are WOCBP.

Exclusion Criteria:

1. Target Disease Exceptions

a) Current use of the following medications for the treatment of diabetes, or use
within the specified timeframe prior to screening for the study: i) Six months:
insulin. ii) Four months: thiazolidinediones. iii) Two months: any other antidiabetic
treatment iv) Any previous use of DPP4-inhibitor and/or incretin mimetics b) Current
use of prescription or non-prescription weight loss drugs and their use within 3
months of screening.

2. Medical History and Concurrent Diseases

a) Significant co-morbidity that, in the opinion of the investigators would preclude
participation in the study (eg, current treatment for cancer). b) Previous diagnosis
of monogenic etiology of type 2 diabetes such as MODY (maturity onset of diabetes in
youth) or secondary diabetes (steroid use, Cushing's disease, acromegaly). c)
Significant cardiovascular history. d) History of hemoglobinopathies (sickle cell
anemia or thalassemias, sideroblastic anemia). e) History of unstable or rapidly
progressive renal disease. f) History of alcohol or drug abuse. g) Psychiatric or
cognitive disorder that will, in the opinion of investigators, limit the patient's
ability to comply with the study medications and monitoring. h) Administration of any
other study drug or participation in a clinical research trial within 30 days of
planned enrollment to this study (or a longer period if dictated by local regulatory
authorities). i) Any condition, which in the investigator's opinion may render the
subject unable to complete the study or may pose significant risk to the subject. j)
Immunocompromised individuals such as subjects that have undergone organ
transplantation or subjects diagnosed with human immunodeficiency virus. k) Subjects
on a commercial weight loss program with ongoing weight loss, or on an intensive
exercise program.

3. Physical and Laboratory Test Findings a) Fasting plasma glucose (FPG) > 255 mg/dL
(14.2 mmol/L) at screening will exclude the patient. b) Diabetic ketoacidosis (DKA)
within 6 months of study entr1) Target Disease Exceptions a) Current use of the
following medications : i) Six months: insulin. ii) Four months: thiazolidinediones.
iii) Two months: any other antidiabetic treatment iv) Any previous use of
DPP4-inhibitor and/or incretin mimetics b) Current use of prescription or
non-prescription weight loss drugs and their use within 3 months of screening.

2) Medical History and Concurrent Diseases

a) Significant co-morbidity that, in the opinion of the investigators would preclude
participation in the study (eg, current treatment for cancer). b) Previous diagnosis of
monogenic etiology of type 2 diabetes such as MODY (maturity onset of diabetes in youth) or
secondary diabetes (steroid use, Cushing's disease, acromegaly). c) Significant
cardiovascular history. d) History of hemoglobinopathies (sickle cell anemia or
thalassemias, sideroblastic anemia). e) History of unstable or rapidly progressive renal
disease. f) History of alcohol or drug abuse. g) Psychiatric or cognitive disorder that
will, in the opinion of investigators, limit the patient's ability to comply with the study
medications and monitoring. h) Administration of any other study drug or participation in a
clinical research trial within 30 days of planned enrollment to this study (or a longer
period if dictated by local regulatory authorities). i) Any condition, which in the
investigator's opinion may render the subject unable to complete the study or may pose
significant risk to the subject. j) Immunocompromised individuals such as subjects that
have undergone organ transplantation or subjects diagnosed with human immunodeficiency
virus. k) Subjects on a commercial weight loss program with ongoing weight loss, or on an
intensive exercise program. 3) Physical and Laboratory Test Findings

1. Fasting plasma glucose (FPG) > 255 mg/dL (14.2 mmol/L) at screening

2. Diabetic ketoacidosis (DKA) within 6 months of study entry (DKA can occur as a
presenting sign of type 2 diabetes in youth).

3. Abnormal renal function, which is defined as an abnormal creatinine clearance rate as
determined by the Schwartz Formula

Exclusion from study participation will apply to calculated glomerular filtration rate < 80
mL/min/1.73m2 (1.33 mL/s). d) Presence of one or more of the following: antibodies to
glutamic acid decarboxylase (GAD), islet cell autoantibody (ICA), protein tyrosine
phosphatase-like protein antibodies (IA-2). e) Active liver disease and/or significant
abnormal liver function defined as Aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) > 2 times upper limits of normal, and/or serum total bilirubin > 2.0
mg/dL. f) History of positive serologic evidence of current infectious liver disease
including anti-HAV (IgM), HbsAg, or anti-HCV. Patients who may have isolated positive anti
HBs may be included. g) Anemia of any etiology defined as hemoglobin = 10.7 g/dL (107 g/L)
for females and = 11.3 g/dL (113 g/L) for males. h) An abnormal TSH value at screening will
be further evaluated by free T4. Subjects with an abnormal T4 will be excluded. i)
Creatinine kinase (CK) = 3 X ULN. j) Clinically significant (CS) abnormalities in any
pre-randomization laboratory analyses or ECG that, in the investigator's opinion, would
preclude randomization. 4) Allergies and Adverse Drug Reaction

1. Subjects who have contraindications to therapy as outlined in the Saxagliptin
Investigator Brochure or local metformin package insert.

2. Subjects with known contraindications to DPP-IV therapy. 5) Prohibited Therapies
and/or M


Age minimum: 10 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Type 2 Diabetes

Intervention(s)

Drug: Placebo (Metformin)

Drug: Saxagliptin

Drug: Placebo (Saxagliptin)

Drug: Metformin IR

Drug: Metformin (Active Rescue)

Primary Outcome(s)

Mean Change in HbA1c From Baseline to Week 16 [Time Frame: 16 week short term treatment period]

Secondary ID(s)

2010-020360-38

CV181-058

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 18/04/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01204775