RY_SW01 Cell Injection Therapy in Systemic Sclerosis

NCT06058091 | Recruiting Phase 1

• 1 other identifier: RYSW202301
• Study Type: interventional
• Target: 81
• Locations: 1 country
• Sites: 1

Brief Summary

Systemic sclerosis (SSc) tends to progress to involve multiple vital organs within 5 years of diagnosis, significantly impacting patient prognosis and survival. Clinical indications suggest that early intervention is more favorable for long-term outcomes in patients. Although guidelines recommend various drugs for symptomatic treatment, there is currently no standard therapy or effective medication to slow the progression of the disease. Therefore, for patients with diffuse SSc, as defined by a skin score of 10≤mRSS≤30 points, who have been treated with at least two therapies, including steroids, immunosuppressive agents, biologics, etc., within 5 years of diagnosis, the applicant intends to develop a drug that can both modulate the immune system and counteract fibrosis. The goal is to provide long-term benefits to patients through early intervention.

Conditions

Systemic Sclerosis

Keywords

RY_SW01 cell injection

Outcome Measures

Primary Outcomes (2)

• Frequency of Adverse Events

  24 weeks

• Change of mRSS

  24 weeks

Secondary Outcomes (6)

• Change in mRSS from baseline

  12 weeks

• Change in lung function (FVC predicted percentage) from baselineRY_SW01 cell injection.

  24 weeks

• Proportion of subjects with treatment failure.

  24 weeks

• Proportion of subjects showing improvement in mRSScell injection (Improvement defined as a decrease in mRSS from baseline by ≥25%).

  24 weeks

• Change in Health Assessment Questionnaire-Disability Index (HAQ-DI) from baseline to 24 weeks after RY_SW01 cell injection.

  24 weeks

Study Arms (3)

Phase II-Placebo group

PLACEBO COMPARATOR

Receive the best basic treatment

Drug: Basic treatment

Phase II-low-does group

EXPERIMENTAL

Receive the best basic treatment and a million cells per kilogram of body weight

Drug: RY_SW01 cell injection; Drug: Basic treatment

Phase II-high does group

EXPERIMENTAL

Receive the best basic treatment and two million cells per kilogram of body weight

Drug: RY_SW01 cell injection; Drug: Basic treatment

Interventions

RY_SW01 cell injection DRUG

Administer basic treatment with an injection of RY\_SW01 cell solution

Also known as: UC-MSC treatment

Phase II-high does group; Phase II-low-does group

Basic treatment DRUG

Basic treatment for SSc

Also known as: immunosuppressor

Phase II-Placebo group; Phase II-high does group; Phase II-low-does group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Voluntarily sign the informed consent form.
• Aged between 18 and 65 years (inclusive), regardless of gender.
• Diagnosed with systemic sclerosis (SSc) based on the 2013 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) criteria for SSc.
• Screened as diffuse cutaneous SSc patients with a disease duration of ≤5 years (disease onset defined as the time of the initial diagnosis of SSc).
• Previously treated with at least two of the following therapies: corticosteroids, immunosuppressants, biologic agents, and others, and have a skin score of 10≤mRSS≤30 points.

You may not qualify if:

• At screening, subjects with a forced vital capacity (FVC) predicted percentage \<50%.
• Previously diagnosed with pulmonary arterial hypertension or, at rest, had a mean pulmonary arterial pressure \>25mmHg measured by right heart catheterization or had a systolic pulmonary artery pressure \>45mmHg measured by echocardiography at screening.
• Presence of clinical symptoms requiring hospitalization for one of the following conditions at screening, whether newly occurring or worsening of pre-existing symptoms within 6 months: myocardial infarction, stroke, renal crisis, severe uncontrolled hypertension (≥160/100mmHg); or within 3 months: unstable ischemic heart disease, uncontrolled arrhythmia, heart failure (New York Heart Association III/IV stage), left ventricular ejection fraction \<50% as indicated by echocardiography, renal insufficiency, or hypertensive crisis as judged by the investigator.
• Concurrent autoimmune connective tissue diseases other than systemic sclerosis, with the exception of patients with secondary Sjögren's syndrome.
• Presence of any of the following laboratory abnormalities at screening:
• Hematology abnormalities: Hemoglobin \<100g/L; White blood cell count \<3.0×109/L; Neutrophil absolute count \<1.5×109/L; Platelet count \<100×109/L.
• Hepatic function abnormalities: ALT or AST \>3 times the upper limit of normal (ULN); Total bilirubin \>3 times ULN.
• Renal function abnormalities: Estimated glomerular filtration rate (eGFR) \<60mL/min/1.73m2 or any clinically significant laboratory abnormalities that may affect the interpretation of study data or the subject's participation in the study as determined by the investigator.
• Positive testing for human immunodeficiency virus (HIV) antibody, active syphilis, active hepatitis C (positive HCV antibodies and positive HCV-RNA), HBsAg positive and HBV-DNA positive at screening; history of severe active bacterial, viral, fungal, parasitic, or other infections during the screening period.
• Receipt of live vaccines/attenuated vaccines within 2 months prior to enrollment.
• Occurrence of any of the following within 3 months prior to enrollment: a. Major trauma or major surgery (including joint surgery) or anticipated major surgery during the study, which the investigator believes would pose an unacceptable risk to the subject. b. Plasma exchange or extracorporeal photopheresis treatment. c. Participation in any other clinical trials.
• Prior treatment with stem cell-related drugs.
• History of any malignancy within the past 5 years prior to enrollment, except for adequately treated or excised basal cell carcinoma, squamous cell carcinoma of the skin, or in situ cervical carcinoma.
• Intolerance or contraindication to the study treatment, including any of the following: a. Allergy to albumin contained in the investigational product excipient. b. Lack of suitable peripheral venous access.
• History of smoking, alcohol abuse, or drug abuse within the past 12 months or during the screening period:

Sponsors & Collaborators

• Jiangsu Renocell Biotech Company: lead
• The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School: collaborator

Study Sites (1)

Department of Rheumatology and Immunology, Nanjing Drum Tower Hospital, the Affiliated Drum Tower Hospital of Nanjing University Medical School

Nanjing, Jiangsu, 210008, China

RECRUITING

MeSH Terms

Conditions

Scleroderma, Systemic

Interventions

macrophage-derived immunosuppressor factor

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Jing Wang

  Jiangsu Renocell Biotech Company

  STUDY DIRECTOR

Central Study Contacts

Ning Wei

CONTACT

15852926678; weining@rybiotech.cn

Jing Wang

CONTACT

025-86162919; wangjing@rybiotech.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School

Study Record Dates

• First Submitted: September 21, 2023
• First Posted: September 28, 2023
• Study Start: September 22, 2023
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2035
• Last Updated: January 18, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)