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NCT Number: NCT07548996

Open-Label Study of Dimethyl Fumarate in Adults With Type 1 Diabetes

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.

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Key information

Conditions

Type 1 Diabetes Mellitus Autoimmune Diseases Diabetes Mellitus Diabetes Mellitus, Type 1 Endocrine System Diseases Glucose Metabolism Disorders Immune System Diseases Metabolic Diseases Nutritional and Metabolic Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Jiangsu Provincial Hospital

Nanjing, Jiangsu, 210029, China

Location status: Recruiting

Location contact

Yong Gu

CONTACT

[email protected]

13814084876

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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)
  • Random C-peptide level greater than or equal to 200 pmol/L

Note:

  • For participants who have used insulin for more than 14 days, a positive IAA result must be accompanied by at least 2 additional positive autoantibodies other than IAA

Exclusion criteria

  • 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
  • Patients with renal insufficiency or evidence of renal impairment:

eGFR <60 mL/min/1.73 m²; or other renal diseases considered by the investigator to be unsuitable for study enrollment

  • History of malignancy, uncontrolled immune system disease, or uncontrolled infection
  • Alcohol abuse, drug abuse, psychiatric disorder, or other conditions considered unsuitable for participation in a drug trial
  • Use of other immunosuppressive agents within 12 weeks before enrollment
  • Participation in any other drug trial within 12 weeks before enrollment
  • History of multiple drug allergies, allergic diseases, hypersensitivity constitution, or drug dependence
  • Any disease or condition that, in the opinion of the investigator, may interfere with study participation or evaluation

Treatment and study plan

Dimethyl Fumarate Enteric-coated Capsules

Drug

Dimethyl fumarate enteric-coated capsules, initiated at 120 mg twice daily and increased after 7 days to 240 mg twice daily.

Other names: DMF

Insulin

Drug

Standard insulin therapy for type 1 diabetes according to routine clinical practice.

Primary outcomes

  1. Baseline-Adjusted Geometric Mean Area Under the Serum C-Peptide Curve During a 2-Hour Mixed-Meal Tolerance Test

    Time frame: 24 weeks after end of intervention (48 weeks after enrollment)

    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).

Secondary outcomes

  1. Baseline-Adjusted Geometric Mean Area Under the Serum C-Peptide Curve During a 2-Hour Mixed-Meal Tolerance Test

    Time frame: End of intervention (24 weeks after enrollment) and 52 weeks after end of intervention (76 weeks after enrollment)

    Baseline-adjusted geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test (MMTT).

  2. Change From Baseline in Geometric Mean Area Under the Serum C-Peptide Curve During a 2-Hour Mixed-Meal Tolerance Test

    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)

    Change from baseline in the geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test (MMTT).

  3. Number of Participants Maintaining Positive C-Peptide Response After a 2-Hour Mixed-Meal Tolerance Test

    Time frame: 52 weeks after end of intervention (76 weeks after enrollment)

    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).

  4. Glycated Hemoglobin (HbA1c)

    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)

    Glycated hemoglobin (HbA1c) level and change from baseline.

  5. Number of Participants With Poor Glycemic Control

    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)

    Number of participants with poor glycemic control, defined as HbA1c greater than 9%.

  6. Number of Participants With Good Glycemic Control

    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)

    Number of participants with good glycemic control, defined as HbA1c less than 6.5%.

  7. Average Exogenous Insulin Dose

    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)

    Average dose of exogenous insulin during the 7 days prior to each study visit, expressed as IU/kg/day.

  8. Change from baseline in proportion of peripheral blood CD4+ T cells

    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)

    Measured by flow cytometry and reported as percentage of lymphocytes.

  9. Change from baseline in proportion of peripheral blood B cells

    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)

    Measured by flow cytometry and reported as percentage of lymphocytes.

  10. Change from baseline in proportion of peripheral blood natural killer cells

    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)

    Measured by flow cytometry and reported as percentage of lymphocytes.

  11. Change from baseline in proportion of peripheral blood regulatory T cells

    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)

    Measured by flow cytometry and reported as percentage of lymphocytes.

  12. Change from baseline in serum interleukin-6 concentration

    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)

    Measured in serum

  13. Change from baseline in serum tumor necrosis factor-alpha concentration

    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)

    Measured in serum

  14. Change from baseline in number of positive islet autoantibodies

    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)

    Count of positive islet autoantibodies among GADA, IA-2A, ZnT8A, ICA, and IAA, reported as an integer from 0 to 5.

  15. Change from baseline in glutamic acid decarboxylase autoantibody titer

    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)

    Measured using an assay-specific method and reported in assay-specific units.

  16. Change from baseline in zinc transporter 8 autoantibody titer

    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)

    Measured using an assay-specific method and reported in assay-specific units.

  17. Number of participants with composite treatment-related symptomatic adverse reactions

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one occurrence of any of the following treatment-related symptomatic adverse reactions during the safety follow-up period: flushing, abdominal pain, diarrhea, nausea, vomiting, pruritus, rash, erythema, or dyspepsia.

  18. Number of participants with composite hypersensitivity or opportunistic infection events

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one occurrence of any of the following during the safety follow-up period: immediate hypersensitivity reaction, angioedema, or opportunistic infection.

  19. Number of participants with composite laboratory safety abnormalities

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one occurrence of any of the following during the safety follow-up period: elevated aspartate aminotransferase, elevated total bilirubin, or lymphopenia.

  20. Number of participants with composite renal safety events

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one occurrence of any of the following during the safety follow-up period: new-onset or worsening albuminuria, increased serum creatinine, or decreased estimated glomerular filtration rate.

  21. Number of participants with hypoglycemia

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one episode of hypoglycemia during the safety follow-up period.

  22. Number of participants with severe hypoglycemia

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one episode of severe hypoglycemia during the safety follow-up period.

  23. Number of participants with ketosis

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one episode of ketosis during the safety follow-up period.

  24. Number of participants with diabetic ketoacidosis

    Time frame: From first dose to 52 weeks after end of intervention (up to 76 weeks after enrollment)

    Participants with at least one episode of diabetic ketoacidosis during the safety follow-up period.

Study contacts

Contact information is provided by the study sponsor or research team.

Yong Gu

CONTACT

[email protected]

+86 13814084876

Sponsors and collaborators

Lead sponsor

Nanjing Medical University

Other

Registry information

Official study title

Non-Randomized, Parallel-Controlled, Single-Center, Open-Label Clinical Trial Evaluating the Efficacy and Safety of Dimethyl Fumarate in Preserving Pancreatic β-Cell Function in Adults With Type 1 Diabetes

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Apr 23, 2026
Registry last updated
Jul 21, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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