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Clinical Trial Results:
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.

Summary
EudraCT number	2010-020360-38
Trial protocol	BE Outside EU/EEA IT
Global end of trial date	22 Apr 2016

Results information
Results version number	v1(current)
This version publication date	23 Mar 2017
First version publication date	23 Mar 2017
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

CV181058

ISRCTN number

US NCT number

WHO universal trial number (UTN)

Sponsor organisation name

AstraZeneca AB, S-151 85 Södertälje, Sweden

Sponsor organisation address

S-151 85 Södertälje, Södertälje, Sweden,

Public contact

Eva Johnsson, Clinical Science Lead, GLOBAL_MEDICINES_DEV, AstraZeneca AB, Eva.Johnsson@astrazeneca.com

Scientific contact

Eva Johnsson, Clinical Science Lead, GLOBAL_MEDICINES_DEV, AstraZeneca AB, +46 31 7762484 762 484, Eva.Johnsson@astrazeneca.com

Is trial part of an agreed paediatric investigation plan (PIP)

Yes

EMA paediatric investigation plan number(s)

EMEA-000200-PIP01-08

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

14 Nov 2016

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

22 Apr 2016

Was the trial ended prematurely?

Yes

Main objective of the trial

To assess the safety and tolerability of saxagliptin monotherapy in pediatric subjects aged 10 to < 18 years when administered for up to 16 weeks of short-term therapy and 52 weeks of total therapy.

Protection of trial subjects

This study was conducted in accordance with Good Clinical Practice (GCP), as defined by the International Conference on Harmonization (ICH) and in accordance with the ethical principles underlying European Union Directive 2001/20/EC and the United States Code of Federal Regulations, Title 21, Part 50 (21CFR50). The study was conducted in compliance with the protocol. The protocol and the amendment and the subject informed consent and assent forms was received Institutional Review Board/Independent Ethics Committee (IRB/IEC) approval/favorable opinion prior to initiation of the study. Study personnel involved conducted this study was qualified by education, training, and experience to perform their respective tasks.

Background therapy

Evidence for comparator

Actual start date of recruitment

22 Apr 2011

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

European Union: 1

Country: Number of subjects enrolled

Taiwan: 1

Country: Number of subjects enrolled

United States: 6

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

This is a multicenter, 52-week, randomized, prospective, double-blind, parallel-group study. Approximately 136 subjects not currently on pharmacologic therapy for diabetes with HbA1c ≥ 7.0% to ≤ 10.5% will be randomized 1:1 to receive oral blinded saxagliptin or blinded placebo.

Screening details

After obtaining informed consent, study procedures will be performed to confirm eligibility.Glucose levels confirmed during the screening process. Screening tests to exclude type 1 diabetes will be performed.

Period 1 title

Overall Study (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Investigator, Monitor, Carer, Subject, Data analyst, Assessor

Are arms mutually exclusive

Yes

Saxagliptin

Arm description

Saxagliptin 2.5 mg or 5 mg according to body weight

Arm type

Experimental

Investigational medicinal product name

saxagliptin

Investigational medicinal product code

Other name

Pharmaceutical forms

Film-coated tablet

Routes of administration

Oral use

Dosage and administration details

Saxagliptin 2.5 mg or 5 mg according to body weight once daily

Placebo

Arm description

Placebo matching saxagliptin

Arm type

Placebo

Investigational medicinal product name

Placebo matching saxagliptin

Investigational medicinal product code

Other name

Pharmaceutical forms

Film-coated tablet

Routes of administration

Oral use

Dosage and administration details

Placebo matching saxagliptin once daily

Number of subjects in period 1	Saxagliptin	Placebo
Started	4	4
Double-blind treatment period	4	4
Completed	3	3
Not completed	1	1
Consent withdrawn by subject	1	-
Poor/non-compliance	-	1

Baseline characteristics

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Reporting group title

Saxagliptin

Reporting group description

Saxagliptin 2.5 mg or 5 mg according to body weight

Reporting group title

Placebo

Reporting group description

Placebo matching saxagliptin

Reporting group values	Saxagliptin	Placebo	Total
Number of subjects	4	4	8
Age Categorical
Units: number
<= 18 years	4	4	8
Age continuous
Units: years
arithmetic mean (standard deviation)	14.5 ( 0.5 )	13.5 ( 1.8 )	-
Gender, Male/Female
Units: participant
Female	1	3	4
Male	3	1	4

Subject analysis set title

Saxagliptin

Subject analysis set type

Intention-to-treat

Subject analysis set description

Saxagliptin 2.5mg or 5 mg depending on body weight

Subject analysis set title

Placebo

Subject analysis set type

Intention-to-treat

Subject analysis set description

Placebo matching saxagliptin

Subject analysis sets values	Saxagliptin	Placebo
Number of subjects	4	4
Age Categorical
Units: number
<= 18 years	4	4
Age continuous
Units: years
arithmetic mean (standard deviation)	14.5 ( 0.5 )	13.5 ( 1.8 )
Gender, Male/Female
Units: participant
Female	1	3
Male	3	1

End points

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Reporting group title

Saxagliptin

Reporting group description

Saxagliptin 2.5 mg or 5 mg according to body weight

Reporting group title

Placebo

Reporting group description

Placebo matching saxagliptin

Subject analysis set title

Saxagliptin

Subject analysis set type

Intention-to-treat

Subject analysis set description

Saxagliptin 2.5mg or 5 mg depending on body weight

Subject analysis set title

Placebo

Subject analysis set type

Intention-to-treat

Subject analysis set description

Placebo matching saxagliptin

Primary: Mean change in HbA1c from baseline to Week 16

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End point title

Mean change in HbA1c from baseline to Week 16 ^[1]

End point description

End point type

Primary

End point timeframe

16 week double-blind treatment period

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis has been performed for this end point due to a small number of subjects in the study

End point values	Saxagliptin	Placebo
Number of subjects analysed	4	4
Units: percentage
arithmetic mean (standard deviation)	-0.7 ( 0.83 )	0.6 ( 1.53 )

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

52 week

Adverse event reporting additional description

16 week double-blind treatment period and 36 week long-term extension period

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

19.0

Reporting group title

Saxagliptin

Reporting group description

Saxagliptin 2.5 mg or 5 mg according to body weight

Reporting group title

Placebo

Reporting group description

Placebo matching saxagliptin

Serious adverse events	Saxagliptin	Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Infections and infestations
Pneumonia
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Saxagliptin	Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	3 / 4 (75.00%)	3 / 4 (75.00%)
Injury, poisoning and procedural complications
Fall
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0
Laceration
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Thermal burn
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Nervous system disorders
Headache
alternative assessment type: Systematic
subjects affected / exposed	1 / 4 (25.00%)	1 / 4 (25.00%)
occurrences all number	2	1
Blood and lymphatic system disorders
Lymphadenopathy
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
General disorders and administration site conditions
Peripheral swelling
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Eye disorders
Vision blurred
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Diarrhoea
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0
Gastrooesophageal reflux disease
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Vomiting
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
alternative assessment type: Systematic
subjects affected / exposed	2 / 4 (50.00%)	1 / 4 (25.00%)
occurrences all number	2	2
cough
alternative assessment type: Systematic
subjects affected / exposed	1 / 4 (25.00%)	1 / 4 (25.00%)
occurrences all number	1	1
Epistaxis
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Nasal congestion
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Wheezing
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0
erythema
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Hyperhidrosis
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Onycholysis
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0
Renal and urinary disorders
Pollakiuria
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Musculoskeletal and connective tissue disorders
Joint swelling
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Infections and infestations
Influenza
subjects affected / exposed	0 / 4 (0.00%)	1 / 4 (25.00%)
occurrences all number	0	1
Upper respiratory tract infection
subjects affected / exposed	1 / 4 (25.00%)	1 / 4 (25.00%)
occurrences all number	1	1
Viral upper respiratory tract infection
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0
Metabolism and nutrition disorders
Hypernatraemia
subjects affected / exposed	1 / 4 (25.00%)	0 / 4 (0.00%)
occurrences all number	1	0

More information

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Were there any global substantial amendments to the protocol? Yes

09 Sep 2011

The exclusion criteria concerning monogenic etiology of Type 2 DM and secondary diabetes was expanded to include previous diagnosis of genetic disorders with strong associations with insulin resistance/diabetes and/or obesity such as Turner’s Syndrome and Prader-Willi.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results:
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.

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Mean change in HbA1c from baseline to Week 16

Primary: Mean change in HbA1c from baseline to Week 16

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Clinical trials

Clinical Trial Results: 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.

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Mean change in HbA1c from baseline to Week 16

Primary: Mean change in HbA1c from baseline to Week 16

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
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.