Table of Contents

• Trial overview
• Who is being studied
• Study designs and phases
• What is being measured
• Trial status and enrollment
• Simple explanation of key terms

Trial overview

Two interventional studies are investigating Verapamil Hydrochloride in adults with type 1 diabetes mellitus.^[1][2] Both studies focus on whether treatment can help preserve beta-cell function, which means the work of the pancreas cells that make insulin.^[1][2]

The first study is a randomised, double-blind, placebo-controlled, parallel-group, multi-centre Phase 2 trial in adults with newly diagnosed type 1 diabetes mellitus.^[1] The second study is an open-label extension, multi-centre Phase 2 trial in adults with type 1 diabetes who are continuing long-term follow-up.^[2]

Who is being studied

The main trial enrolled adult subjects with newly diagnosed type 1 diabetes mellitus.^[1] This means the study looked at people soon after diagnosis, when some insulin-making function may still be present.^[1]

The extension trial includes adult subjects diagnosed with type 1 diabetes mellitus and specifically notes follow-up in people who previously had a fasting C-peptide of at least 50 pmol/L and had earlier received placebo or Verapamil Hydrochloride in the first study.^[2] This shows the extension is focused on a selected group with measurable remaining insulin production.^[2]

Study designs and phases

The first study used a randomised design, which means participants were assigned by chance to a treatment group.^[1] It was also double-blind and placebo controlled, so neither the participants nor the study team knew who received active treatment or placebo during the trial.^[1]

The first study compared placebo with Verapamil Hydrochloride 360 mg given orally once daily for 12 months.^[1] The extension study is open-label, which means the treatment is known, and it follows participants for longer-term therapy over 24 months.^[2]

Both studies are Phase 2 trials.^[1][2] Phase 2 trials are early clinical studies that mainly look at whether a treatment may work in the target group and how outcomes change over time.^[1][2]

What is being measured

The main outcome in the first study is the area under the stimulated C-peptide response curve over the first two hours of a mixed meal tolerance test (MMTT) after 12 months of therapy compared with placebo.^[1] This outcome checks how much C-peptide the body makes after a meal-like test.^[1]

The extension study measures change over time in C-peptide area under the curve in adults receiving 360 mg oral Verapamil Hydrochloride daily during MMTT at baseline and after 24 months of therapy.^[2] In simple terms, the study is asking whether the body keeps making more of its own insulin over time.^[2]

C-peptide is a marker of natural insulin production, so it helps researchers understand how well the beta cells are still working.^[1][2]

Trial status and enrollment

The first trial, NCT04545151, is listed as completed and enrolled 120 participants.^[1] The second trial, 2024-515234-33-00, is listed as authorised and plans to enroll 40 participants.^[2]

These numbers show that the research is relatively small and focused on a specific patient group with type 1 diabetes.^[1][2]

Simple explanation of key terms

• Placebo means a treatment with no active study drug, used for comparison.^[1]

• Parallel-group means different groups are followed at the same time.^[1]

• Multi-centre means the trial takes place at more than one study site.^[1][2]

• Open-label extension means a follow-up study where treatment is known and the study continues for a longer time.^[2]

• Fasting C-peptide means the C-peptide level measured when a person has not eaten, which helps show remaining insulin production.^[2]