Clinical trial NCT06149260: Subcutaneous Semaglutide in Systemic Scleroderma
Official title: An Open-lable Trial of Subcutaneous Semaglutide in Systemic Scleroderma
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Scleroderma, Systemic
Fibrosis
Semaglutide
Lead sponsor: Second Xiangya Hospital of Central South University
Sponsor class: OTHER
First posted: 2023-11-28
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

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.

Primary outcomes:
measure: Change in modified Rodnan skin score (mRSS) at week 24
timeFrame: Baseline and 24 weeks
Secondary outcomes:
measure: Incidence of Adverse Events
timeFrame: Baseline and 24 weeks
measure: Incidence of Severe Adverse Events
timeFrame: Baseline and 24 weeks
measure: the scleroderma health assessment questionnaire-disability index (sHAQ-DI)
timeFrame: Baseline and 24 weeks
measure: High-resolution computer tomography (HRCT)
timeFrame: Baseline and 24 weeks
measure: Forced Vital Capacity(FVC) and Diffusing capacity of the lung for carbon monoxid(DLCO)
timeFrame: Baseline and 24 weeks
measure: St George' s Respiratory Questionnaire(SGRQ)
timeFrame: Baseline and 24 weeks
measure: Pulmonary arterial hypertension by echocardiography
timeFrame: Baseline and 24 weeks
measure: left ventricular ejection fraction by echocardiography
timeFrame: Baseline and 24 weeks
measure: 6-Minute Walk Test (6MWT) Distance
timeFrame: Baseline and 24 weeks
measure: gastrointestinal tract (GIT) in scleroderma score
timeFrame: Baseline and 24 weeks
Other outcomes:
Locations:
city: Changsha
country: China
facility: the Second Xiangya Hospital
state: Hunan
Study officials:
affiliation: Department of Dermatology, Second Xiangya Hospital of Central South University, Changsha, China
name: Rong Xiao, MD
role: STUDY_CHAIR
affiliation: Second Xiangya Hospital
name: Licong Liu, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Yaqian Shi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Zhuotong Zeng, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Zhan Yi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Xiangning Qiu, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Ruixuan Zhu, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Yi Wei, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Ke Chai, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Hao Ren, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Second Xiangya Hospital
name: Yangfan Xiao, MD
role: PRINCIPAL_INVESTIGATOR
References: