Systemic SclerosisPhase 1/2NCT06058091
RY_SW01 Cell Injection Therapy in Systemic Sclerosis
A Phase 1/2 study testing RY_SW01 cell injection and Basic treatment in people with Systemic Sclerosis, run by Jiangsu Renocell Biotech Company at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Systemic Sclerosis
- What is being tested?
- RY_SW01 cell injection and Basic treatment
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 81 participants
- Where
- 1 siteChina
- Study dates
- 22 Sep 2023to 31 Dec 2035
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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:
- Smoking defined as an average daily consumption of ≥5 cigarettes within the 3 months prior to screening.
- Alcohol abuse defined as consuming more than 14 units of alcohol per week (1 unit of alcohol = 350ml of beer, or 45ml of spirits, or 150ml of wine) within the 3 months prior to screening.
- Drug abuse defined as having a history of drug abuse.
- Plans for conception during the trial period until at least 1 year after cell infusion, unwillingness to use effective contraceptive measures with their partners, or plans for sperm or egg donation.
- Deemed unsuitable for participation in the study by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Department of Rheumatology and Immunology, Nanjing Drum Tower Hospital, the Affiliated Drum Tower Hospital of Nanjing University Medical School
Nanjing, Jiangsu, 210008
Sun Lingyun, MD
About this study
Official title: A Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of RY_SW01 Cell Injection Therapy in Systemic Sclerosis
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.
Read the detailed description
This clinical trial is a multicenter Phase I/II clinical trial, which includes two stages: Phase I dose-escalation and Phase II dose-expansion. The Phase I dose-escalation stage adopts a single-arm trial design, aiming to explore the safety, tolerability, and preliminary efficacy of RY_SW01 cell injection in treating patients with systemic sclerosis. The Phase II dose-expansion stage adopts a randomized, double-blind controlled trial design, intending to explore the safety, efficacy, and changes in disease-related biomarkers of RY_SW01 cell injection in treating systemic sclerosis patients.
Based on the characteristics of this product and pre-clinical study data, as well as integrating the safety and efficacy data of similar types of drugs and clinical trials for the same or similar indications published domestically and internationally, the researchers and sponsors jointly selected the trial exploration doses as low dose 1.0×10^6 cells/kg and high dose 2.0×10^6 cells/kg.
The trial will enroll systemic sclerosis patients aged ≥18 and ≤65 years, who must meet all inclusion criteria and none of the exclusion criteria. Approximately 81-87 subjects are planned to be enrolled to undergo dose-escalation and dose-expansion trials with RY_SW01 cell injection. The dose-escalation stage plans to enroll 6-12 evaluable subjects, and the dose-expansion stage plans to enroll 75 subjects.
- Conditions
- Systemic Sclerosis
- Being tested
- RY_SW01 cell injection, Basic treatment
- Keywords
- RY_SW01 cell injection
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RY_SW01 cell injectionDrug
Also called UC-MSC treatment
Administer basic treatment with an injection of RY_SW01 cell solution
Basic treatmentDrug
Also called immunosuppressor
Basic treatment for SSc
| Study group | Role | What they receive |
|---|---|---|
| Phase II-Placebo group | Placebo comparator | Receive the best basic treatment Receives: Drug: Basic treatment |
| Phase II-low-does group | Experimental | Receive the best basic treatment and a million cells per kilogram of body weight Receives: 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 Receives: Drug: RY_SW01 cell injection; Drug: Basic treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events
Measured: 24 weeks
Change of mRSS
Measured: 24 weeks
Secondary outcomes (6)
Change in mRSS from baseline
Measured: 12 weeks
Change in lung function (FVC predicted percentage) from baselineRY_SW01 cell injection.
Measured: 24 weeks
Proportion of subjects with treatment failure.
Measured: 24 weeks
Proportion of subjects showing improvement in mRSScell injection (Improvement defined as a decrease in mRSS from baseline by ≥25%).
Measured: 24 weeks
Change in Health Assessment Questionnaire-Disability Index (HAQ-DI) from baseline to 24 weeks after RY_SW01 cell injection.
Measured: 24 weeks
Biological markerTGF-β, VEGF, TNF-a, and Th17 cell subset proportions from baseline to 12 and 24 weeks after RY_SW01 cell injection.
TNFR1, anti-Scl-70 antibodies, IL-6, TGF-β，TNF-a,Th17
Measured: 12weeks 24weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start22 Sep 2023
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 81 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jiangsu Renocell Biotech Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
- Responsible party
- Principal investigator, Sun Lingyun, MD, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
- Investigators
- Jing Wang, Study director, Jiangsu Renocell Biotech Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06058091
- Sponsor's protocol ID
- RYSW202301
- Status last verified
- Jan 2024
- First submitted
- 21 Sep 2023
- Last updated
- 18 Jan 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of RY_SW01 Cell Injection Therapy in Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT06058091
- Acronym / sponsor ID
- RYSW202301
- Conditions
- Systemic Sclerosis
- Keywords
- RY_SW01 cell injection
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 81 participants (estimated)
- Lead sponsor
- Jiangsu Renocell Biotech CompanyIndustry (pharma, biotech, devices)
- Collaborators
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
- Study start
- 22 Sep 2023 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2035 (estimated)
- First posted
- 28 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This clinical trial is a multicenter Phase I/II clinical trial, which includes two stages: Phase I dose-escalation and Phase II dose-expansion. The Phase I dose-escalation stage adopts a single-arm trial design, aiming to explore the safety, tolerability, and preliminary efficacy of RY_SW01 cell injection in treating patients with systemic sclerosis. The Phase II dose-expansion stage adopts a randomized, double-blind controlled trial design, intending to explore the safety, efficacy, and changes in disease-related biomarkers of RY_SW01 cell injection in treating systemic sclerosis patients.
Based on the characteristics of this product and pre-clinical study data, as well as integrating the safety and efficacy data of similar types of drugs and clinical trials for the same or similar indications published domestically and internationally, the researchers and sponsors jointly selected the trial exploration doses as low dose 1.0×10^6 cells/kg and high dose 2.0×10^6 cells/kg.
The trial will enroll systemic sclerosis patients aged ≥18 and ≤65 years, who must meet all inclusion criteria and none of the exclusion criteria. Approximately 81-87 subjects are planned to be enrolled to undergo dose-escalation and dose-expansion trials with RY_SW01 cell injection. The dose-escalation stage plans to enroll 6-12 evaluable subjects, and the dose-expansion stage plans to enroll 75 subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Phase II-Placebo group | Placebo comparator | Drug: Basic treatmentReceive the best basic treatment |
| Phase II-low-does group | Experimental | Drug: RY_SW01 cell injection, Drug: Basic treatmentReceive the best basic treatment and a million cells per kilogram of body weight |
| Phase II-high does group | Experimental | Drug: RY_SW01 cell injection, Drug: Basic treatmentReceive the best basic treatment and two million cells per kilogram of body weight |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (20 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RY_SW01 cell injectionAlso: UC-MSC treatmentAdminister basic treatment with an injection of RY_SW01 cell solution | Drug | Phase II-high does group, Phase II-low-does group |
| Basic treatmentAlso: immunosuppressorBasic treatment for SSc | Drug | Phase II-Placebo group, Phase II-high does group, Phase II-low-does group |
Primary outcome
Frequency of Adverse Events
Time frame: 24 weeks
Change of mRSS
Time frame: 24 weeks
6 secondary outcomes
Change in mRSS from baseline
Time frame: 12 weeks
Change in lung function (FVC predicted percentage) from baselineRY_SW01 cell injection.
Time frame: 24 weeks
Proportion of subjects with treatment failure.
Time frame: 24 weeks
Proportion of subjects showing improvement in mRSScell injection (Improvement defined as a decrease in mRSS from baseline by ≥25%).
Time frame: 24 weeks
Change in Health Assessment Questionnaire-Disability Index (HAQ-DI) from baseline to 24 weeks after RY_SW01 cell injection.
Time frame: 24 weeks
Biological markerTGF-β, VEGF, TNF-a, and Th17 cell subset proportions from baseline to 12 and 24 weeks after RY_SW01 cell injection.
Time frame: 12weeks 24weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jing Wang study director
Jiangsu Renocell Biotech Company
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing RY_SW01 cell injection and Basic treatment in people with Systemic Sclerosis, run by Jiangsu Renocell Biotech Company at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ning Wei (15852926678, weining@rybiotech.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Adverse Events (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jan 2024.