Subcutaneous Semaglutide in Systemic Scleroderma

An Open-lable Trial of Subcutaneous Semaglutide in Systemic Scleroderma

This trial will study the safety and efficacy of subcutaneous semaglutide for the treatment of Systemic Sclerosis

Study Overview

• Status: Recruiting
• Conditions: Fibrosis; Scleroderma, Systemic; Semaglutide
• Intervention / Treatment: Drug: Semaglutide Pen Injector

Detailed Description

Systemic Sclerosis (Ssc) is a rare, systemic autoimmune disease characterized by skin fibrosis and vasculopathy. In addition to the skin, it is a heterogeneous disease that affects multiple organs, including the cardiac, pulmonary, and gastrointestinal systems. This is a small prospective and open-label clinical trial of semaglutide in adults with systemic slceorsis. 10 systemic sclerosis patients will be recruited and receive semaglutide for 24 weeks. The primary endpoint of the study is the change in mRSS at 24 weeks of treatment.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Rong Xiao, MD; Phone Number: 13808425555; Email: xiaorong65@csu.edu.cn
• Study Contact Backup: Name: Licong Liu, MD; Phone Number: 18573185298; Email: Liulicong1997@csu.edu.cn

Study Locations

• China
  • Hunan
    • Changsha, Hunan, China, 410011
      • Recruiting
      • The second xiangya hospital
      • Contact: Rong Xiao, MD; Phone Number: +8618573185298; Email: xiaorong65@csu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Semaglutide; Participants will receive once-weekly semaglutide subcutaneous injection at escalating doses from 0.25 mg/week to 0.5 mg/week.	Drug: Semaglutide Pen Injector; Inject semaglutide subcutaneously once weekly, on the same day each week, at any time of day, in a dose increasing mode: the initial dose of 0.25mg QW (once a week), increased to 0.5mg QW after 4 weeks, and then maintained 0.5mg until the end of treatment in total 24 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in modified Rodnan skin score (mRSS) at week 24	Change in modified Rodnan skin score (mRSS) at week 24 performed by the same investigator at week 0 and week 24 and the change in mRSS will be calculated following the formula: ΔmRSS= mRSSw24 - mRSSw0. To measure mRSS, skin thickness of the patient is rated by palpation at each of 17 anatomic sites using a scale of 0-3 (0 = normal skin; 1= mild thickness; 2= moderate thickness; 3=severe thickness with an inability to pinch the skin into a fold). The scores at each site are summed with a minimum of 0 and a maximum of 51 (17 sites)	Baseline and 24 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Incidence of Adverse Events	Baseline and 24 weeks
Incidence of Severe Adverse Events	Baseline and 24 weeks
the scleroderma health assessment questionnaire-disability index (sHAQ-DI)	Baseline and 24 weeks
High-resolution computer tomography (HRCT)	Baseline and 24 weeks
Forced Vital Capacity(FVC) and Diffusing capacity of the lung for carbon monoxid(DLCO)	Baseline and 24 weeks
St George' s Respiratory Questionnaire(SGRQ)	Baseline and 24 weeks
Pulmonary arterial hypertension by echocardiography	Baseline and 24 weeks
left ventricular ejection fraction by echocardiography	Baseline and 24 weeks
6-Minute Walk Test (6MWT) Distance	Baseline and 24 weeks
gastrointestinal tract (GIT) in scleroderma score	Baseline and 24 weeks

Collaborators and Investigators

• Sponsor: Second Xiangya Hospital of Central South University
• Investigators: Study Chair: Rong Xiao, MD, Department of Dermatology, Second Xiangya Hospital of Central South University, Changsha, China; Principal Investigator: Licong Liu, MD, Second Xiangya Hospital; Principal Investigator: Yaqian Shi, MD, Second Xiangya Hospital; Principal Investigator: Zhuotong Zeng, MD, Second Xiangya Hospital; Principal Investigator: Zhan Yi, MD, Second Xiangya Hospital; Principal Investigator: Xiangning Qiu, MD, Second Xiangya Hospital; Principal Investigator: Ruixuan Zhu, MD, Second Xiangya Hospital; Principal Investigator: Yi Wei, MD, Second Xiangya Hospital; Principal Investigator: Ke Chai, MD, Second Xiangya Hospital; Principal Investigator: Hao Ren, MD, Second Xiangya Hospital; Principal Investigator: Yangfan Xiao, MD, Second Xiangya Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 29, 2024
• Primary Completion (Estimated): August 29, 2024
• Study Completion (Estimated): December 1, 2024

Study Registration Dates

• First Submitted: November 16, 2023
• First Submitted That Met QC Criteria: November 20, 2023
• First Posted (Actual): November 28, 2023

Study Record Updates

• Last Update Posted (Actual): March 5, 2024
• Last Update Submitted That Met QC Criteria: March 2, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Connective Tissue Diseases; Scleroderma, Systemic; Scleroderma, Diffuse; Scleroderma, Localized; Hypoglycemic Agents; Physiological Effects of Drugs; Glucagon-Like Peptide-1 Receptor Agonists; Semaglutide
• Other Study ID Numbers: LYG2022061

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No