Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial

Introduction Type 1 diabetes mellitus (T1DM) is a disorder that arises following the selective autoimmune destruction of the insulin-producing beta cells. Beta-cell protective or beta-cell regenerative approaches have gained wider attention, and pharmacological approaches to protect the patient’s ow...

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Main Authors: Piotr Jaroslaw Chmura, Thomas Danne, Colin Dayan, Thomas R Pieber, Chantal Mathieu, Rachel Stenson, Julie Wych, Martina Brunner, Adrian Paul Mander
Format: Article
Language:English
Published: BMJ Publishing Group 2024-11-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/14/11/e091597.full
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author Piotr Jaroslaw Chmura
Thomas Danne
Colin Dayan
Thomas R Pieber
Chantal Mathieu
Rachel Stenson
Julie Wych
Martina Brunner
Adrian Paul Mander
author_facet Piotr Jaroslaw Chmura
Thomas Danne
Colin Dayan
Thomas R Pieber
Chantal Mathieu
Rachel Stenson
Julie Wych
Martina Brunner
Adrian Paul Mander
author_sort Piotr Jaroslaw Chmura
collection DOAJ
description Introduction Type 1 diabetes mellitus (T1DM) is a disorder that arises following the selective autoimmune destruction of the insulin-producing beta cells. Beta-cell protective or beta-cell regenerative approaches have gained wider attention, and pharmacological approaches to protect the patient’s own insulin-producing beta-cell mass have been proposed. Verapamil is an L-type calcium channel blocker that has been reported to effectively lowers beta-cell thioredoxin-interacting protein expression in rodent beta cells and islets, as well as in human islets, and thus promotes functional beta-cell mass.Methods and analysis The trial is a multicentre, randomised, double-blind, placebo-controlled trial in participants with T1DM, investigating the effect of verapamil on preservation of beta-cell function (Ver-A-T1D). A total of 120 participants will be randomised in a 2:1 ratio between 360 mg verapamil and placebo, administered orally once daily. T1DM patients aged ≥18 and <45 years will be eligible for recruitment within 6 weeks of diagnosis (defined as day of starting insulin therapy). The primary objective will be to determine the changes in stimulated C-peptide response during the first 2 hours of a mixed meal tolerance test at baseline and after 12 months for 360 mg verapamil administered orally once daily versus placebo. Secondary objectives include the effects of 360 mg verapamil on (1) fasting C-peptide, (2) dried blood spot C-peptide, (3) glycated haemoglobin, (4) daily total insulin dose, (5) time in range by intermittent continuous glucose monitoring measures, (6) other biomarkers related to immunological changes and beta-cell death and (6) safety (vital signs, ECG).Ethics and dissemination Ethics approval was sought from the research ethics committee of all participating countries. All participants provided written informed consent before joining the study. Ver-A-T1D received first regulatory and ethical approvals in Austria. The publication policy is set in the innovative approach towards understanding and arresting type 1 diabetes grant agreement (www.innodia.eu).Trial registration number EudraCT, 2020-000435-45; ClinicalTrials.gov, NCT04545151. Protocol version: Version 8.0 (08 November 2021).
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spelling doaj-art-397dbfd57a634ce2977b6cc88dca62952025-08-20T01:56:34ZengBMJ Publishing GroupBMJ Open2044-60552024-11-01141110.1136/bmjopen-2024-091597Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trialPiotr Jaroslaw Chmura0Thomas Danne1Colin Dayan2Thomas R Pieber3Chantal Mathieu4Rachel Stenson5Julie Wych6Martina Brunner7Adrian Paul Mander8Center for Protein Research, Kobenhavns Universitet Sundhedsvidenskabelige Fakultet, Kobenhavn, DenmarkDepartment of General Pediatrics Endocrinology/Diabetology & Clinical Research, Auf der Bult Children`s Hospital, Hannover, GermanyCardiff University School of Medicine, Cardiff University, Cardiff, UKDepartment of Internal Medicine, Division of Endocrinology and Diabetology, Medical University of Graz, Graz, AustriaKatholieke Universiteit Leuven, UZ Leuven, Leuven, BelgiumDiabetes Research Group, Cardiff University, Cardiff, UKCentre for Trials Research, Cardiff University, Cardiff, UKDepartment of Internal Medicine, Division of Endocrinology and Diabetology, Medical University of Graz, Graz, AustriaCentre for Trials Research, Cardiff University, Cardiff, UKIntroduction Type 1 diabetes mellitus (T1DM) is a disorder that arises following the selective autoimmune destruction of the insulin-producing beta cells. Beta-cell protective or beta-cell regenerative approaches have gained wider attention, and pharmacological approaches to protect the patient’s own insulin-producing beta-cell mass have been proposed. Verapamil is an L-type calcium channel blocker that has been reported to effectively lowers beta-cell thioredoxin-interacting protein expression in rodent beta cells and islets, as well as in human islets, and thus promotes functional beta-cell mass.Methods and analysis The trial is a multicentre, randomised, double-blind, placebo-controlled trial in participants with T1DM, investigating the effect of verapamil on preservation of beta-cell function (Ver-A-T1D). A total of 120 participants will be randomised in a 2:1 ratio between 360 mg verapamil and placebo, administered orally once daily. T1DM patients aged ≥18 and <45 years will be eligible for recruitment within 6 weeks of diagnosis (defined as day of starting insulin therapy). The primary objective will be to determine the changes in stimulated C-peptide response during the first 2 hours of a mixed meal tolerance test at baseline and after 12 months for 360 mg verapamil administered orally once daily versus placebo. Secondary objectives include the effects of 360 mg verapamil on (1) fasting C-peptide, (2) dried blood spot C-peptide, (3) glycated haemoglobin, (4) daily total insulin dose, (5) time in range by intermittent continuous glucose monitoring measures, (6) other biomarkers related to immunological changes and beta-cell death and (6) safety (vital signs, ECG).Ethics and dissemination Ethics approval was sought from the research ethics committee of all participating countries. All participants provided written informed consent before joining the study. Ver-A-T1D received first regulatory and ethical approvals in Austria. The publication policy is set in the innovative approach towards understanding and arresting type 1 diabetes grant agreement (www.innodia.eu).Trial registration number EudraCT, 2020-000435-45; ClinicalTrials.gov, NCT04545151. Protocol version: Version 8.0 (08 November 2021).https://bmjopen.bmj.com/content/14/11/e091597.full
spellingShingle Piotr Jaroslaw Chmura
Thomas Danne
Colin Dayan
Thomas R Pieber
Chantal Mathieu
Rachel Stenson
Julie Wych
Martina Brunner
Adrian Paul Mander
Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial
BMJ Open
title Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial
title_full Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial
title_fullStr Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial
title_full_unstemmed Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial
title_short Investigating the effect of verapamil on preservation of beta-cell function in adults with newly diagnosed type 1 diabetes mellitus (Ver-A-T1D): protocol for a randomised, double-blind, placebo-controlled, parallel-group, multicentre trial
title_sort investigating the effect of verapamil on preservation of beta cell function in adults with newly diagnosed type 1 diabetes mellitus ver a t1d protocol for a randomised double blind placebo controlled parallel group multicentre trial
url https://bmjopen.bmj.com/content/14/11/e091597.full
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