Colesevelam for Children With Type 2 Diabetes

Phase 4

Completed

• Conditions: Type 2 Diabetes Mellitus
• Interventions: Drug: High-dose colesevelam; Drug: Low-dose colesevelam

• Registration Number: NCT01258075
• Lead Sponsor: Daiichi Sankyo

Brief Summary

This study will evaluate the efficacy and safety of the study drug in treating type 2 diabetes in children 10 to 17 years old.

The groups will be low-dose (0.625 g Welchol) and high-dose (3.75 g Welchol). The children will have a 2 in 5 chance of being assigned to the low-dose group. They will have a 3 in 5 chance of being assigned to the high-dose group.

We believe the study drug will be safe, well tolerated, and improve blood sugar control in children 10 to 17 years old.

Detailed Description

Colesevelam oral suspension will be studied as treatment of type 2 diabetes mellitus (T2DM) to evaluate clinical safety and efficacy in patients aged 10-17 years. The patients may have been treated with Metformin or have had no antidiabetic drug treatment in the previous three months.

Study Hypothesis: Colesevelam oral suspension for pediatric subjects with T2DM is safe, well tolerated, and shows improved blood sugar control (as evidenced by a significant change from baseline in hemoglobin A1C \[HbA1c\]).

Study Timeline

• Study First Submitted: 2010-12-09
• Study First Submitted QC: 2010-12-09
• Study First Posted: 2010-12-10
• Study Start: 2011-02-24
• Primary Completion: 2019-11-01
• Study Completion: 2020-04-21
• Results First Submitted: 2020-11-25
• Results First Submitted QC: 2020-11-25
• Results First Posted: 2020-12-22
• Status Verified: 2021-04
• Last Update Submitted: 2021-04-16
• Last Update Posted: 2021-05-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 236

Inclusion Criteria

• Diagnosis of type 2 diabetes mellitus, as defined by the American Diabetes Association;

• Written informed consent of study participation

• Males and females aged 10 to 17 years, inclusive, at randomization (randomization must occur before 18th birthday);

• HbA1c at screening between 7.0% and 10.0%, inclusive;

• Fasting C-peptide >0.6 ng/mL; and

• Anti-diabetic treatment at screening:

  • Treatment-naïve or untreated; OR
  • On metformin monotherapy: Metformin monotherapy has been initiated prior to screening.

Exclusion Criteria

• Fasting plasma glucose >270 mg/dL;
• Diagnosis of type 1 diabetes;
• History of more than one episode of ketoacidosis after the initial diagnosis of type 2 diabetes mellitus;
• Clinical laboratory assessments/evaluations, eg. autoimmune markers, aminotransferases, triglycerides, creatinine clearance, and Hb variants, that are not within the protocol-defined parameters
• Systolic blood pressure ≥150 mmHg or diastolic blood pressure ≥95 mmHg
• Use of medications not allowed by protocol-defined parameters, eg. insulin or any medication that affects insulin sensitivity or secretion, growth hormones/somatotropin, or anabolic steroids
• Genetic syndrome or disorder known to affect glucose
• Participation in a weight loss program or another interventional research study within 60 days;
• Female participants who are lactating, pregnant, or plan to become pregnant within 1 year of screening;
• Female participants who are sexually active and unwilling to use appropriate contraception for the duration of the study;
• History of bowel obstruction;
• Other significant organ system illness or condition (including psychiatric or developmental disorder) that, in the opinion of the Investigator, would prevent full participation

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Colesevelam	High-dose colesevelam	High-dose colesevelam suspended in a drink for oral administration once daily with dinner
Placebo proxy	Low-dose colesevelam	Low-dose colesevelam suspended in a drink for oral administration once daily with dinner

Primary Outcome Measures

Name	Time	Method
Percent Change in Hemoglobin A1c (HbA1c) From Baseline to Month 6	Baseline to Month 6 post-dose	The percent change in HbA1c from baseline to Month 6 was assessed with the last observation after 1 month before any rescue therapy carried forward.

Secondary Outcome Measures

Name	Time	Method
Change in Fasting Plasma Glucose (FPG) From Baseline to Month 12	Baseline to Month 12 post-dose	Change from baseline was assessed for FPG values at Month 6 and Month 12 categorical time points.
Number of Participants Requiring Rescue Medication Who Initially Met Rescue Criteria	Baseline to Month 12 post-dose	Rescue criteria was defined as HbA1c levels ≥8.5% after 3 months or ≥7.5% after 6 months (≥173 days) (confirmed persistent hyperglycemia) of study medication treatment, as measured by the central laboratory.
Percent Change in Hemoglobin A1c (HbA1c) From Baseline to Month 12	Baseline to Month 12 post-dose	The percent change in HbA1c from baseline to Month 6 was assessed with the last observation after 1 month before any rescue therapy carried forward.
Number of Participants Achieving a Response to Therapy to Month 12	Baseline to Month 12 post-dose	Participants achieving a response to therapy, ie, glycemic control, were assessed with the last observation after 1 month before any rescue therapy carried forward at Month 6 and Month 12. Response to therapy was defined as HbA1c \<7.0% or \<6.5%, reduction in HbA1c ≥0.7% or ≥0.5% from baseline, and/or reduction in FPG ≥30 mg/dL from baseline.
Percent Change From Baseline to Month 6 in Triglycerides	Baseline to Month 6 post-dose	The percent change in triglycerides from baseline to Month 6 was assessed with the last observation after 1 month before any rescue therapy carried forward.
Percent Change From Baseline to Month 6 in Plasma Lipids	Baseline to Month 6 post-dose	The percent change in plasma lipids from baseline to Month 6 was assessed with the last observation after 1 month before any rescue therapy carried forward.