Ertugliflozin Pediatric Study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Merck Sharp & Dohme LLC

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

29 Nov 2019 → 28 Apr 2025

Decision date (initial)

2022-11-28

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Merck Sharp & Dohme LLC · Pfizer USA

External identifiers

EU CT number

2022-501085-21-00

EudraCT number

2017-003455-35

WHO UTN

U1111-1279-3984

ClinicalTrials.gov

NCT04029480

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Pharmacokinetic, Efficacy

1. To compare the addition of ertugliflozin with the addition of placebo on the change from baseline in hemoglobin A1C (A1C) at 24 weeks.
2. To assess the safety and tolerability of ertugliflozin over 24 weeks and 54 weeks.

Secondary objectives 4

1. To compare the addition of ertugliflozin, with dosing optimized according to A1C response, to the addition of placebo on the change from baseline in A1C at 24 weeks.
2. To compare the addition of ertugliflozin 5 mg with the addition of placebo on the change from baseline in A1C at 24 weeks.
3. To compare the effect of the addition of ertugliflozin to the addition of placebo on the change from baseline in fasting plasma glucose (FPG) at 24 weeks.
4. To assess the within-group (ertugliflozin and placebo) changes from baseline at 54 weeks for A1C and FPG.

Conditions and MedDRA coding

Type 2 Diabetes Mellitus

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001533-PIP01-13

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Has diabetes diagnosed by one of the American Diabetes Association (ADA) criteria.
2. Has body mass index (BMI) ≥85th percentile at screening OR participant has a history of being overweight or obese at time of diagnosis of Type 2 diabetes mellitus (T2DM).
3. T2DM for ≥2 years, OR T2DM for <2 years and a fasting C-peptide value >0.6 ng/mL at Screening.
4. On stable metformin monotherapy (≥1500 mg/day, for ≥8 weeks prior to Screening, OR on a stable metformin dose (≥1500 mg/day, for ≥8 weeks prior to Screening and a stable dose of insulin for ≥8 weeks prior to Screening.
5. Contraceptive use by male participants should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
6. Is a non-sterilized female who is currently not sexually active OR who agrees to abstain from heterosexual activity OR who agrees to start contraception prior to initiating sexual activity and who agrees to use an adequate method of contraception. Contraceptive use by females should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
7. Have a family member or adult who, along with the participant, will be closely involved in the participant's daily activities (in the opinion of the investigator) and in the participant's treatment and study procedures.

Exclusion criteria 8

1. Has known type 1 diabetes mellitus or documented evidence of positive diabetes autoantibodies performed when participant was diagnosed with diabetes.
2. Has known monogenic diabetes, or secondary diabetes.
3. Has symptomatic hyperglycemia and/or moderate to large ketonuria requiring immediate initiation of another antihyperglycemic agent, including insulin.
4. Has a known hypersensitivity or intolerance to any sodium glucose co-transporter 2 (SGLT2) inhibitor.
5. Is pregnant, or breast feeding or is expecting to conceive or donate eggs during the study, including 14 days following the last dose of study medication.
6. Has previously taken an SGLT2 inhibitor (such as canagliflozin, dapagliflozin, empagliflozin, or ertugliflozin) or was enrolled in a study for these agents.
7. Has a history of idiopathic acute pancreatitis or chronic pancreatitis.
8. Has a history of severe hypoglycemia while on insulin.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. Change from Baseline in Hemoglobin A1C (HbA1C) at 24 weeks (pooled ertugliflozin 5 mg and 15 mg versus placebo)
2. Number of Participants Who Experience an Adverse Event (AE) over 24 weeks
3. Number of Participants Who Experience an AE over 54 weeks
4. Number of Participants Who Discontinue Study Treatment Due to an AE over 24 weeks
5. Number of Participants Who Discontinue Study Treatment Due to an AE over 54 weeks

Secondary endpoints 5

1. Change from Baseline in Hemoglobin A1C at Week 24 (dose-optimized ertugliflozin versus placebo)
2. Change from Baseline in Hemoglobin A1C at Week 24 (ertugliflozin 5 mg versus placebo)
3. Change from Baseline in Fasting Plasma Glucose (FPG) at 24 weeks
4. Change from Baseline in Hemoglobin A1C at 54 weeks
5. Change from Baseline in FPG at 54 weeks

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merck Sharp & Dohme LLC

Sponsor organisation

Merck Sharp & Dohme LLC

Address

126 East Lincoln Avenue

City

Rahway

Postcode

07065-4607

Country

United States

Scientific contact point

Organisation

Merck Sharp & Dohme LLC

Contact name

Ravi Shankar

Public contact point

Organisation

Merck Sharp & Dohme LLC

Contact name

Ravi Shankar

Third parties 6

Locations

3 EU/EEA countries · 15 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications