Tocilizumab for the Treatment of Refractory Granulomatous Lobular Mastitis

NCT07595900 | Not Yet Recruiting Phase 1

• 1 other identifier: SYSKY-2026-253-02
• Study Type: interventional
• Target: 31
• Locations: 0 countries
• Sites: N/A

Brief Summary

Non-Lactational Granulomatous Lobular Mastitis (NL-GLM) is an inflammatory disease of unknown etiology, characterized clinically by local breast masses, accompanied by redness and swelling of the overlying skin, sinus tract formation, and other symptoms. Currently, there is no universally accepted standard treatment for this condition; previous expert consensus or practice guidelines have mostly recommended systemic glucocorticoid therapy as the primary treatment approach. Our team's preliminary research has confirmed that local glucocorticoid injection achieves efficacy equivalent to systemic administration but with better safety, making it a first-line treatment option for NL-GLM. However, in our preliminary studies and literature reports, we found that some patients still exhibit glucocorticoid dependence or resistance (i.e., refractory NL-GLM) after receiving either local or systemic glucocorticoid therapy. The lack of high-quality evidence to support subsequent-line treatments has become a major bottleneck in clinical management. Additionally, some patients cannot tolerate glucocorticoid therapy due to its adverse effects. Research has shown that the IL-6 inflammatory pathway is significantly activated in the lesion tissues and peripheral blood of NL-GLM patients, and the IL-6 inhibitor tocilizumab has demonstrated efficacy in various autoimmune diseases. Based on this, this study intends to conduct a dual-center, single-arm clinical trial to systematically evaluate the efficacy and safety of tocilizumab in the treatment of refractory NL-GLM. The aim is to fill the treatment gap, provide high-level evidence for clinical practice, and ultimately improve patient outcomes.

Conditions

Granulomatous Lobular Mastitis; Granulomatous Mastitis

Outcome Measures

Primary Outcomes (1)

• 8-week inflammatory remission rate

  Week 8 after enrollment

Secondary Outcomes (1)

• 4/12/16-week inflammatory remission rate

  Week 4/12/16 after enrollment

Study Arms (1)

Tocilizumab Treatment Group

EXPERIMENTAL

Drug: Tocilizumab

Interventions

Tocilizumab DRUG

Enrolled patients received intravenous administration of tocilizumab at a dose of 8 mg/kg (maximum 400 mg) at Week 1 and Week 5 after enrollment.

Tocilizumab Treatment Group

Eligibility Criteria

• Age: 20 Years - 50 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Females aged 20 to 50 years;
• Clinically diagnosed (combined with pathology) with non-lactational granulomatous mastitis (cessation of lactation for more than 6 months);
• M-Activity-Score ≥ 2;
• Steroid-refractory NL-GLM or intolerant to steroid therapy.
• Female subjects of childbearing potential agree to use highly effective contraception starting at least 7 days before the first dose until 16 weeks after dosing. Pregnancy tests for female subjects of childbearing potential must be negative within 7 days before the first dose.

You may not qualify if:

• Bilateral mastitis occurring simultaneously or sequentially within six months.
• Clinical diagnosis (combined with pathological findings) of periductal mastitis.
• History of lymphoproliferative disorder; or presence of signs or symptoms suggestive of a possible lymphoproliferative disorder (including lymphadenopathy or splenomegaly); or active primary or recurrent malignancy; or clinically significant malignancy with a remission duration of less than 5 years.
• Patients with carcinoma in situ of the cervix may participate if successfully treated with no evidence of recurrence or metastasis for at least 3 years.
• Patients with basal cell or squamous cell carcinoma of the skin may participate if successfully treated with no evidence of recurrence for at least 3 years.
• Patients who are pregnant.
• Current or recent severe viral, bacterial, fungal, or parasitic infection, including but not limited to:
• Symptomatic herpes zoster infection within 12 weeks prior to screening.
• History of disseminated/complicated herpes zoster (e.g., multidermatomal involvement, herpes zoster ophthalmicus, CNS involvement, or postherpetic neuralgia).
• Symptomatic herpes simplex at the time of enrollment.
• Active or chronic infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV).
• Household contact with an individual with active tuberculosis (TB) and not having received appropriate and documented TB prophylaxis.
• Evidence of active TB, or history of active TB without appropriate and documented treatment.
• Clinically significant infection within 4 weeks prior to enrollment, or having received intravenous antibiotic therapy for an infection.
• Any other active or recent infection within 4 weeks prior to enrollment that, in the investigator's judgment, would pose an unacceptable risk to the patient.

Sponsors & Collaborators

• Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University: lead

MeSH Terms

Conditions

Granulomatous Mastitis

Interventions

tocilizumab

Condition Hierarchy (Ancestors)

Mastitis; Puerperal Disorders; Pregnancy Complications; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Central Study Contacts

Kai Chen

CONTACT

86-02034070166; ckaichen@126.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 13, 2026
• First Posted: May 19, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): October 30, 2028
• Study Completion (Estimated): October 30, 2028
• Last Updated: May 19, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share