Clinical Study for Dimethyl Fumarate in Preserving Islet β-Cell Function in Type 1 Diabetes Mellitus

Official title

A Single-Center, Randomized, Double-Blind, Placebo-Controlled Clinical Trial Evaluating the Efficacy and Safety of Dimethyl Fumarate in Preserving Islet β-Cell Function in Patients With Type 1 Diabetes Mellitus

Overview

Purpose of the Clinical Trial: This clinical trial aims to investigate whether dimethyl fumarate can treat adults with newly diagnosed type 1 diabetes and to evaluate the safety profile of dimethyl fumarate. Primary Research Questions: Does dimethyl fumarate protect pancreatic beta-cell function in adults with newly diagnosed type 1 diabetes? What medical issues may arise in individuals taking dimethyl fumarate? Study Design: Researchers will compare dimethyl fumarate with a placebo (an identical substance without active ingredients) to determine whether Dimethyl fumarate can effectively treat type 1 diabetes. Participant Activities: Take dimethyl fumarate or placebo orally twice daily for 24 weeks. Attend on-site visits every 4 weeks during the intervention period and every 12 weeks after the intervention for examinations and assessments. Record symptoms, blood glucose control, islet function, and insulin usage throughout the trial.

Interventions

• Drug Dimethyl Fumarate Enteric-coated Capsules
  The dosing regimen for Dimethyl fumarate enteric-coated capsules initiates at 120 mg twice daily (bid). After 7 days, the dose should be escalated to the maintenance level of 240 mg bid. This investigational product is administered concurrently with standard insulin therapy for glycemic control in Type 1 Diabetes Mellitus (T1DM).
• Drug Matching placebo capsules
  The placebo capsules initiate at a dosage of 120 mg twice daily (bid). After 7 days, the dose should be increased to the maintenance level of 240 mg bid, administered concomitantly with standard insulin-based antihyperglycemic therapy for Type 1 Diabetes Mellitus (T1DM).

Primary outcome measures

• Baseline-adjusted geometric mean area under the serum C-peptide curve (C-peptide AUC) during a 2-hour mixed-meal tolerance test (MMTT) 24 weeks post-intervention. [Time frame: Post-intervention Weeks 24]

Secondary outcome measures (10)

• Baseline-adjusted geometric mean area under the curve (AUC) for serum C-peptide during a 2-hour mixed-meal tolerance test (MMTT) at 24 weeks of intervention and 52 weeks after the end of intervention. [Time frame: Week 24 of intervention and 52 weeks post-intervention]
• Changes from baseline in the geometric mean area under the C-peptide curve (AUC-C-peptide) during the 2-hour Mixed-Meal Tolerance Test (MMTT) at Intervention Week 24 and at Weeks 24 and 52 after the end of the intervention. [Time frame: Intervention Week 24, and Post-intervention Weeks 24 and 52]
• The number of subjects who remained C-peptide positive at 52 weeks after the end of intervention (defined as a stimulated peak serum C-peptide concentration >= 200 pmol/L during a 2-hour MMTT). [Time frame: Post-intervention Week 52]
• Glycemic Control Status [Time frame: Intervention Week 24, and Post-intervention Weeks 24 and 52]
• Mean Daily Dose of Exogenous Insulin Used During the 7 Days Preceding Each Study Visit [Time frame: Intervention Week 24, and Post-intervention Weeks 24 and 52]
• Immunological markers [Time frame: Baseline, Week 24 During Intervention, and 24,52 Weeks After Intervention]
• Incidence Rates of Flushing, Abdominal Pain, Diarrhea, Nausea, Vomiting, Pruritus, Rash, Proteinuria, Erythema, and Dyspepsia [Time frame: Week 4, 8, 12, 16, and 24 During Intervention]
• Incidence Rates of Anaphylaxis, Angioedema, and Opportunistic Infections [Time frame: Week 4, 8, 12, 16, and 24 During Intervention]
• Incidence Rates of Elevated Aspartate Aminotransferase (AST), Elevated Total Bilirubin (TBIL), and Lymphocytopenia [Time frame: Week 4, 8, 12, 16, and 24 During Intervention]
• Incidence Rates of Hypoglycemia/Severe Hypoglycemia and Ketosis/Diabetic Ketoacidosis (DKA) [Time frame: Baseline, Weeks4, 8, 12, 16, 20 and 24 During Intervention, and 12, 24, 36, and 52 Weeks After Intervention]

Eligibility criteria

Inclusion criteria

• Subjects who provide written informed consent.
• Aged 18-65 years.
• Diagnosed with Type 1 Diabetes Mellitus (per ADA 2024 criteria).
• Positive for ≥2 autoantibodies: Insulin autoantibody (IAA) Glutamic acid decarboxylase autoantibody (GADA) Protein tyrosine phosphatase antibody (IA-2A) Islet cell antibody (ICA) Zinc transporter 8 autoantibody (ZnT8A) Note: For IAA-positive subjects with insulin use >14 days, ≥2 additional autoantibodies must be positive.
• Disease duration ≤100 days post-T1DM diagnosis.
• Random C-peptide ≥ 200 pmol/L.

Exclusion criteria

• Pregnancy, lactation, or women of childbearing potential not using contraception.
• Well-controlled glycemia with oral hypoglycemic agents alone.
• Participation in other diabetes/immune-modulating trials.
• ALT/AST >3× upper limit of normal (ULN).
• History of malignancy, uncontrolled autoimmune disorders, or active infections.
• Alcohol/drug abuse, psychiatric disorders, or conditions unsuitable for trial participation.
• Use of immunosuppressants within 12 weeks prior.
• Participation in other drug trials within 12 weeks prior.
• History of drug allergies, hypersensitivity, or drug addiction.
• Any condition deemed by investigators to compromise study integrity.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

China · 1 center

• Deparement of Endocrinology and Metabolism, The First Affiliated Hospital with Nanjing Med — Nanjing

Identifiers

NCT: NCT07258394 · 2025-SR-439

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗