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

Plain English Summary

Clinical Study for Dimethyl Fumarate in Preserving Islet β-Cell Function in Type 1 Diabetes Mellitus is a Phase 3 clinical trial sponsored by Nanjing Medical University studying Type 1 Diabetes. This trial tests if a drug called Dimethyl Fumarate can help preserve the body's natural insulin-making cells (beta-cells) in people newly diagnosed with Type 1 Diabetes. It is for adults aged 18-65 who have been recently diagnosed with Type 1 Diabetes and have specific markers of the disease. Participants will take either Dimethyl Fumarate or a placebo (inactive pill) twice daily for 24 weeks and attend regular clinic visits. The main alternative is standard insulin therapy, which manages blood sugar but does not aim to preserve beta-cell function. The trial aims to enroll 90 participants.

Official Summary

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.

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you are between 18 and 65 years old and have been diagnosed with Type 1 Diabetes within the last 100 days. You must have specific autoantibodies in your blood and a certain level of a substance called C-peptide, which indicates remaining insulin production. You cannot join if you are pregnant, breastfeeding, or of childbearing potential without using contraception. Exclusion criteria also include uncontrolled blood sugar with oral medications, participation in other diabetes trials, liver enzyme abnormalities, or a history of certain medical conditions like cancer or active infections. This trial is studying Type 1 Diabetes, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how well the drug preserves the body's ability to produce its own insulin, indicated by C-peptide levels, which is crucial for managing blood sugar naturally. The specific primary outcome measures are: 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. (Post-intervention Weeks 24). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses a critical need to preserve beta-cell function in newly diagnosed Type 1 Diabetes patients, potentially slowing disease progression and reducing reliance on insulin. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Type 1 Diabetes, where improved treatment options are needed.

Investor Insight

This Phase 3 trial for a repurposed drug in a large market (Type 1 Diabetes) signals potential for a new treatment option, with a moderate probability of approval if efficacy and safety are demonstrat Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor if Dimethyl Fumarate is a suitable option for you and what the potential benefits and risks are. You will need to take study medication daily and attend regular clinic visits for check-ups, blood tests, and assessments. You will also need to record your symptoms, blood sugar levels, and insulin use throughout the study. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 90 participants

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 Outcomes

• 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. (Post-intervention Weeks 24)

Secondary Outcomes

• 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. (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. (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). (Post-intervention Week 52)
• Glycemic Control Status (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 (Intervention Week 24, and Post-intervention Weeks 24 and 52)

Full Eligibility Criteria

Inclusion Criteria:

1. Subjects who provide written informed consent.
2. Aged 18-65 years.
3. Diagnosed with Type 1 Diabetes Mellitus (per ADA 2024 criteria).
4. 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.
5. Disease duration ≤100 days post-T1DM diagnosis.
6. Random C-peptide ≥ 200 pmol/L.

Exclusion Criteria:

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

Trial Locations

• Deparement of Endocrinology and Metabolism, The First Affiliated Hospital with Nanjing Medical University, Nanjing, Jiangsu, China

Frequently Asked Questions

Related Conditions

• Type 1 Diabetes
• Autoimmune Diseases
• Endocrinology
• Metabolic Disorders
• Beta-cell Function
• Insulin Resistance
• Glycemic Control
• Immunomodulation
• Inflammatory Diseases
• Diabetes Complications

More Type 1 Diabetes Trials

View all Type 1 Diabetes clinical trials