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Ertugliflozin Type 2 Diabetes Mellitus (T2DM) Pediatric Study (MK-8835/PF-04971729) (MK-8835-059)

Registration Number
NCT04029480
Lead Sponsor
Merck Sharp & Dohme LLC
Brief Summary

This study will evaluate the safety and efficacy of ertugliflozin (MK-8835) in pediatric participants with T2DM on metformin with/without insulin. The primary hypothesis of the study is that the addition of ertugliflozin reduces hemoglobin A1C (HbA1C) more than the addition of placebo after 24 weeks of treatment.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
165
Inclusion Criteria
  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
  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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Ertugliflozin 5 mg/15 mgErtugliflozin 5 mgAll participants will initially receive ERTU 5 mg QD and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at WK12, approximately half the participants who meet the up-titration criteria at the second randomization will up-titrate to ERTU 15 mg and placebo to ERTU 5 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a FFSG of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/5 mgErtugliflozin 5 mgAll participants will initially receive ertugliflozin (ERTU) 5 mg once daily (QD) and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at Week 12 (WK12), all participants that do not meet the up-titration criteria will remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Approximately half the participants who meet the up-titration criteria at the second randomization at WK12 will also remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a fasting fingerstick glucose (FFSG) of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
PlaceboPlacebo to ertugliflozin 5 mgAt the first randomization, participants receive placebo to ERTU 5 mg and placebo to ERTU 15 mg QD for 12 weeks. Participants in the placebo group with HbA1C ≥7.0% (53 mmol/mol) at WK12 will be mock titrated. Note: The up-titration criteria for participants on insulin will include a FFSG of ≥110 mg/dL (6.1 mmol/L) in addition to HbA1C ≥7.0% (53 mmol/mol) at WK12. Participants will continue to receive placebo to ERTU 5 mg and placebo to ERTU 15 mg QD from WK24 to WK54. Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/15 mgPlacebo to ertugliflozin 5 mgAll participants will initially receive ERTU 5 mg QD and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at WK12, approximately half the participants who meet the up-titration criteria at the second randomization will up-titrate to ERTU 15 mg and placebo to ERTU 5 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a FFSG of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/5 mgPlacebo to ertugliflozin 15 mgAll participants will initially receive ertugliflozin (ERTU) 5 mg once daily (QD) and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at Week 12 (WK12), all participants that do not meet the up-titration criteria will remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Approximately half the participants who meet the up-titration criteria at the second randomization at WK12 will also remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a fasting fingerstick glucose (FFSG) of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/15 mgErtugliflozin 15 mgAll participants will initially receive ERTU 5 mg QD and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at WK12, approximately half the participants who meet the up-titration criteria at the second randomization will up-titrate to ERTU 15 mg and placebo to ERTU 5 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a FFSG of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/15 mgPlacebo to ertugliflozin 15 mgAll participants will initially receive ERTU 5 mg QD and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at WK12, approximately half the participants who meet the up-titration criteria at the second randomization will up-titrate to ERTU 15 mg and placebo to ERTU 5 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a FFSG of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
PlaceboPlacebo to ertugliflozin 15 mgAt the first randomization, participants receive placebo to ERTU 5 mg and placebo to ERTU 15 mg QD for 12 weeks. Participants in the placebo group with HbA1C ≥7.0% (53 mmol/mol) at WK12 will be mock titrated. Note: The up-titration criteria for participants on insulin will include a FFSG of ≥110 mg/dL (6.1 mmol/L) in addition to HbA1C ≥7.0% (53 mmol/mol) at WK12. Participants will continue to receive placebo to ERTU 5 mg and placebo to ERTU 15 mg QD from WK24 to WK54. Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/5 mgInsulinAll participants will initially receive ertugliflozin (ERTU) 5 mg once daily (QD) and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at Week 12 (WK12), all participants that do not meet the up-titration criteria will remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Approximately half the participants who meet the up-titration criteria at the second randomization at WK12 will also remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a fasting fingerstick glucose (FFSG) of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/15 mgInsulinAll participants will initially receive ERTU 5 mg QD and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at WK12, approximately half the participants who meet the up-titration criteria at the second randomization will up-titrate to ERTU 15 mg and placebo to ERTU 5 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a FFSG of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/5 mgMetforminAll participants will initially receive ertugliflozin (ERTU) 5 mg once daily (QD) and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at Week 12 (WK12), all participants that do not meet the up-titration criteria will remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Approximately half the participants who meet the up-titration criteria at the second randomization at WK12 will also remain on ERTU 5 mg and placebo to ERTU 15 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a fasting fingerstick glucose (FFSG) of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Ertugliflozin 5 mg/15 mgMetforminAll participants will initially receive ERTU 5 mg QD and placebo to ERTU 15 mg QD for 12 weeks. At the second randomization at WK12, approximately half the participants who meet the up-titration criteria at the second randomization will up-titrate to ERTU 15 mg and placebo to ERTU 5 mg from WK12 to WK54. Note: For participants not on insulin, the up-titration criterion at the second randomization at WK12 is HbA1C ≥7.0% (53 mmol/mol) and for participants on insulin, a FFSG of ≥110 mg/dL (6.1 mmol/L) will be required in addition to HbA1C ≥7.0% (53 mmol/mol). Participants will remain on their background metformin with/without insulin treatment throughout the study.
Primary Outcome Measures
NameTimeMethod
Number of Participants Who Experience an Adverse Event (AE) over 24 weeksUp to 24 weeks

An adverse event is defined as any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening (i.e., any clinically significant adverse change in frequency and/or intensity) of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an adverse event.

Number of Participants Who Experience an AE over 54 weeksUp to 54 weeks

An adverse event is defined as any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening (i.e., any clinically significant adverse change in frequency and/or intensity) of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an adverse event.

Number of Participants Who Discontinue Study Treatment Due to an AE over 54 weeksUp to 54 weeks

An adverse event is defined as any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening (i.e., any clinically significant adverse change in frequency and/or intensity) of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an adverse event.

Change from Baseline in Hemoglobin A1C (HbA1C) at 24 weeks (pooled ertugliflozin 5 mg and 15 mg versus placebo)Baseline and 24 weeks

Hemoglobin A1C is a measure of the percentage of glycated HbA1C in the blood. Participant whole blood samples are collected at baseline and Week 24 to determine the least squares mean HbA1C change from baseline (i.e., HbA1C at Week 24 minus HbA1C at baseline).

Number of Participants Who Discontinue Study Treatment Due to an AE over 24 weeksUp to 24 weeks

An adverse event is defined as any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening (i.e., any clinically significant adverse change in frequency and/or intensity) of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an adverse event.

Secondary Outcome Measures
NameTimeMethod
Change from Baseline in FPG at 54 WeeksBaseline and 54 weeks

Blood glucose is measured on a fasting basis. FPG is expressed as mg/dL. Blood is drawn at predose on Day 1 and after 54 weeks of treatment to determine change in plasma glucose levels (i.e., FPG at Week 54 minus FPG at baseline).

Change from Baseline in Hemoglobin A1C at Week 24 (dose-optimized ertugliflozin versus placebo)Baseline and 24 weeks

Hemoglobin A1C is a measure of the percentage of glycated HbA1C in the blood. Participant whole blood samples are collected at baseline and Week 24 to determine the least squares mean HbA1C change from baseline (i.e., HbA1C at Week 24 minus HbA1C at baseline).

Change from Baseline in Hemoglobin A1C at Week 24 (ertugliflozin 5 mg versus placebo)Baseline and 24 weeks

Hemoglobin A1C is a measure of the percentage of glycated HbA1C in the blood. Participant whole blood samples are collected at baseline and Week 24 to determine the least squares mean HbA1C change from baseline (i.e., HbA1C at Week 24 minus HbA1C at baseline).

Change from Baseline in Fasting Plasma Glucose (FPG) at 24 WeeksBaseline and 24 weeks

Blood glucose is measured on a fasting basis. FPG is expressed as mg/dL. Blood is drawn at predose on Day 1 and after 24 weeks of treatment to determine change in plasma glucose levels (i.e., FPG at Week 24 minus FPG at baseline).

Change from Baseline in Hemoglobin A1C at 54 WeeksBaseline and 54 weeks

Hemoglobin A1C is a measure of the percentage of glycated HbA1C in the blood. Participant whole blood samples are collected at baseline and Week 54 to determine the least squares mean HbA1C change from baseline (i.e., HbA1C at Week 54 minus HbA1C at baseline).

Trial Locations

Locations (104)

The University of Alabama at Birmingham ( Site 2207)

🇺🇸

Birmingham, Alabama, United States

Children's Hospital - Los Angeles ( Site 2201)

🇺🇸

Los Angeles, California, United States

Center of Excellence in Diabetes and Endocrinology ( Site 2203)

🇺🇸

Sacramento, California, United States

Memorial Regional Hospital-Joe DiMaggio Children's Hospital Division of Pediatric Endocrinology ( Si

🇺🇸

Hollywood, Florida, United States

ICCT Research International, Inc. ( Site 2211)

🇺🇸

Chicago, Illinois, United States

Barry J. Reiner MD LLC ( Site 2204)

🇺🇸

Baltimore, Maryland, United States

William Beaumont Hospital ( Site 2219)

🇺🇸

Royal Oak, Michigan, United States

CHEAR Center LLC ( Site 2200)

🇺🇸

Bronx, New York, United States

Coastal Children''s Services ( Site 2202)

🇺🇸

Wilmington, North Carolina, United States

The Children's Hospital of Philadelphia ( Site 2205)

🇺🇸

Philadelphia, Pennsylvania, United States

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The University of Alabama at Birmingham ( Site 2207)
🇺🇸Birmingham, Alabama, United States

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