Open-Label Study of Dimethyl Fumarate in Adults With Type 1 Diabetes
ConditionType 1 Diabetes Mellitus
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18-65
SponsorNanjing Medical University
About this trial
This is a non-randomized, parallel-controlled, single-center, open-label clinical trial designed to evaluate the efficacy of dimethyl fumarate in preserving pancreatic beta-cell function in adults with type 1 diabetes, as well as its safety and tolerability in this population.
Eligible participants are adults aged 18 to 65 years who meet the ADA 2024 diagnostic criteria for type 1 diabetes, have at least 2 positive islet autoantibodies, and have residual beta-cell function as evidenced by a random C-peptide level of at least 200 pmol/L. A total of 96 participants are planned for enrollment, including 32 in the dimethyl fumarate treatment group and 64 in the standard-treatment control group.
Participants in the treatment group will receive dimethyl fumarate enteric-coated capsules in addition to standard insulin therapy for type 1 diabetes. Dimethyl fumarate will be initiated at 120 mg twice daily and increased after 7 days to a maintenance dose of 240 mg twice daily. Participants in the control group will receive standard insulin therapy alone. The intervention period will be 24 weeks, followed by 52 weeks of follow-up.
The primary efficacy endpoint is the baseline-adjusted geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test at Week 24. Secondary endpoints include measures of beta-cell function at multiple time points, changes in glycated hemoglobin, proportions of participants with good or poor glycemic control, insulin dose requirements, and immunologic markers including lymphocyte subsets, cytokine profiles, and islet autoantibody characteristics. Safety assessments will include the incidence of flushing, gastrointestinal adverse events, allergic reactions, opportunistic infections, liver function abnormalities, lymphopenia, renal abnormalities, hypoglycemia, severe hypoglycemia, ketosis, and ketoacidosis.
The total study duration is 36 months, from January 2026 to December 2028.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Willing and able to participate in the study and provide signed informed consent
Aged 18 to 65 years
Diagnosed with type 1 diabetes mellitus according to ADA 2024 criteria
Positive for at least 2 islet autoantibodies among insulin autoantibody (IAA), glutamic acid decarboxylase autoantibody (GADA), insulinoma-associated protein 2 autoantibody (IA-2A), islet cell antibody (ICA), and zinc transporter 8 autoantibody (ZnT8A)
Disqualifiers
Pregnant or breastfeeding women, positive urine pregnancy test at screening, or inability to rule out pregnancy in the opinion of the investigator
Good glycemic control with oral antidiabetic drugs alone
Participation in other studies involving diabetes treatment or immunomodulation
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal
Trial design
Design model
Parallel
Treatments tested in this trial
Dimethyl Fumarate Enteric-coated Capsules
DrugDimethyl fumarate enteric-coated capsules, initiated at 120 mg twice daily and increased after 7 days to 240 mg twice daily.
Insulin
DrugStandard insulin therapy for type 1 diabetes according to routine clinical practice.
Treatment groups
96 Participants
are divided into 2 treatment groupsGroup A: Dimethyl Fumarate Plus Standard Insulin TherapyExperimental treatment 2 interventions
Group B: Standard Insulin TherapyActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
Baseline-Adjusted Geometric Mean Area Under the Serum C-Peptide Curve During a 2-Hour Mixed-Meal Tolerance Test
The primary efficacy endpoint is the baseline-adjusted geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test (MMTT).
Time frame
24 weeks after end of intervention (48 weeks after enrollment)Secondary outcomes
1
Baseline-Adjusted Geometric Mean Area Under the Serum C-Peptide Curve During a 2-Hour Mixed-Meal Tolerance Test
Baseline-adjusted geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test (MMTT).
Time frame
End of intervention (24 weeks after enrollment) and 52 weeks after end of intervention (76 weeks after enrollment)2
Change From Baseline in Geometric Mean Area Under the Serum C-Peptide Curve During a 2-Hour Mixed-Meal Tolerance Test
Change from baseline in the geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test (MMTT).
Time frame
End of intervention (24 weeks after enrollment), 24 weeks after end of intervention (48 weeks after enrollment), and 52 weeks after end of intervention (76 weeks after enrollment)3
Number of Participants Maintaining Positive C-Peptide Response After a 2-Hour Mixed-Meal Tolerance Test
Number of participants maintaining positive C-peptide response at 52 weeks after end of intervention, defined as a peak C-peptide concentration of at least 200 pmol/L after a 2-hour mixed-meal tolerance test (MMTT).
Time frame
52 weeks after end of intervention (76 weeks after enrollment)4
Glycated Hemoglobin (HbA1c)
Glycated hemoglobin (HbA1c) level and change from baseline.
Time frame
End of intervention (24 weeks after enrollment), 24 weeks after end of intervention (48 weeks after enrollment), and 52 weeks after end of intervention (76 weeks after enrollment)Sponsors and contacts
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Source ClinicalTrials.gov