Using semaglutide to treat systemic scleroderma

An Open-lable Trial of Subcutaneous Semaglutide in Systemic Scleroderma

This study is testing if a medication called semaglutide can help adults with systemic scleroderma improve their skin and overall health after other treatments didn't work.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Second Xiangya Hospital of Central South University (Academic / other)
• Drugs / interventions: Tofacitinib, Methotrexate, prednisone
• Locations: 1 site (Changsha, Hunan)
• Trial ID: NCT06149260 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and efficacy of subcutaneous semaglutide in adults diagnosed with systemic sclerosis, a rare autoimmune disease that causes skin fibrosis and affects multiple organs. The trial is prospective and open-label, enrolling 10 patients who have not responded well to previous hormone or immunosuppressive therapies. Participants will receive semaglutide for a duration of 24 weeks, with the primary endpoint being the change in modified Rodnan skin score (mRSS) at the end of the treatment period.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with a confirmed diagnosis of systemic sclerosis who have not achieved satisfactory results from prior treatments.

Not a fit: Patients with infections, tumors, or other connective tissue diseases may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with systemic sclerosis who have not responded to existing treatments.

How similar studies have performed: While this approach is novel in the context of systemic sclerosis, similar studies with semaglutide in other conditions have shown promising results.

Eligibility criteria

Inclusion Criteria:(Those who have not achieved good results after hormone or immunosuppressive therapy)

1. Gender unlimited;
2. Age 18-65 years old (including 65 years old);
3. Patients diagnosed with SSc who meet the 2013 European Union Against Rheumatology (EULAR)/American Society of Rheumatology (ACR) SSc diagnostic classification criteria and exclude infections, tumors, and other connective tissue diseases.
4. Has received one or more of the following standard systemic treatments allowed by the research protocol:

   1. Before the first subcutaneous injection of the study, oral corticosteroids (prednisone not exceeding 15mg/d or equivalent) were administered for ≥ 8 weeks, and stabilizers were administered for ≥ 4 weeks.
   2. Before the first subcutaneous injection of the study, patients were treated with Tofacitinib (5-10mg/d) for ≥ 8 weeks and received a stabilizer dose for ≥ 6 weeks.
   3. If one or more of the following immune modulators are used, treatment must be given for ≥ 12 weeks before the start of the study, and treatment with a stabilizer dose must be given for ≥ 6 weeks Oral mycophenolate mofetil (MMF) ≤ 1.5 g/day Methotrexate (MTX) oral ≤ 15 mg/week, combined with folic acid Cyclosporine If the subjects use ≥ 2 of the above immunomodulatory drugs in combination, the appropriateness of the subjects' participation in the study must be discussed with the medical supervisor and study chair before enrollment.
5. A modified Rodnan Skin Score (mRSS) of \> 14
6. Those who sign an informed consent form, voluntarily participate in this project, and are able to complete follow-up as required.

Exclusion Criteria:

1. Prior to the first dose, Body Mass Index (BMI) \< 18.5 kg/m2; weight loss of 10% within one month or 20% within six months.
2. Family or personal history of type 2 multiple endocrine neoplasia or medullary thyroid carcinoma, with family history involving first-degree relatives.
3. History of malignant tumors or a history of malignant tumors within the past 5 years before screening.
4. Presence of other inflammatory diseases that may interfere with efficacy assessment, including but not limited to rheumatoid arthritis (RA), overlap syndrome, psoriasis, dermatomyositis, multiple sclerosis, Crohn's disease, or active Lyme disease.
5. Severe gastrointestinal complications of systemic sclerosis (SSc), such as significant swallowing difficulties, and severe diseases affecting vital organ systems such as the heart, brain, lungs, liver, kidneys, or blood, as deemed unsuitable for participation in the study by the investigator.
6. Known current active or recurrent severe infections, including active tuberculosis.
7. Congenital immunodeficiency or congenital immunosuppression.
8. Substance abuse, alcoholism, or psychiatric disorders, rendering patients uncooperative or unable to adhere to treatment; poor predictability of compliance.
9. Women who are pregnant, breastfeeding, or planning to become pregnant.
10. Patients currently participating in other clinical trials.

Where this trial is running

Changsha, Hunan

• the Second Xiangya Hospital — Changsha, Hunan, China (Recruiting)

Study contacts

• Principal investigator: Licong Liu, MD — Second Xiangya Hospital
• Study coordinator: Rong Xiao, MD

Conditions Scleroderma, Systemic; Fibrosis; Semaglutide