At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- rapcabtagene autoleucel (biological), rituximab (biological)
- How long the study runs
- Study runs about 94 months (dates as stated)
- About the drug or intervention
- rapcabtagene autoleucel — biological: single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days. · rituximab — biological: rituximab intravenous infusion (i.v.) as per protocol
- Patient visit burden
- Not specified by the sponsor
In plain English
This is a phase 2 study looking at a treatment called rapcabtagene autoleucel in people with diffuse cutaneous systemic sclerosis, a severe form of scleroderma. The study is funded by Novartis Pharmaceuticals. It is testing whether this therapy can help people whose disease is severe and getting worse.
Who can take part
- You have systemic sclerosis that meets the 2013 classification criteria, and the diffuse type of the disease (affecting wider areas of skin).
- Your first symptoms (other than Raynaud's) started within the last 7 years — for example puffy hands, skin tightening, finger ulcers, joint pain, or breathlessness.
- Your disease is severe and getting worse in at least one of these ways: lung disease linked to scleroderma, severe and worsening skin disease, or heart involvement.
- You have had all recommended vaccines for people with a weakened immune system.
Who may not be able to
- A condition or medicines that would make the treatment unsafe or unsuitable, in the doctor's judgement.
- An allergy to ingredients in rapcabtagene autoleucel or to a medicine called rituximab.
- Rituximab is not suitable for you, in the doctor's opinion.
- Another health condition not linked to scleroderma that would make the study treatment hard to tolerate.
- Another rheumatic disease (apart from secondary Sjogren's syndrome or scleroderma muscle problems), including the limited type of scleroderma.
- Pulmonary hypertension (high pressure in the lung blood vessels) that you already had.
- Significant kidney disease.
- High blood pressure that is not controlled.
- A live vaccine in the 6 weeks before joining the study.
What taking part involves
- • Not stated — ask the trial team.
Time commitment: Not stated — ask the trial team about study visits, how long it lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years to 70 Years
- Who
- All
- Number of participants
- 96
- Started
- 2024-10-29
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for scleroderma, diffuse
- • Phase2 - 96 participants
- • The purpose of this study is to evaluate the efficacy, safety and tolerability of rapcabtagene autoleucel (administered once following lymphodepletion) in participants with severe refractory diffuse cutaneous systemic sclerosis relative to rituximab
Who can take part?
- • Ages 18 Years to 70 Years
- • Diagnosed with scleroderma, diffuse
Where?
- • Sheffield - Novartis Investigative Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this study is to evaluate the efficacy, safety and tolerability of rapcabtagene autoleucel (administered once following lymphodepletion) in participants with severe refractory diffuse cutaneous systemic sclerosis relative to rituximab.
More detail
This is a phase 2, multi-part, five-year, randomized, open-label, assessor-blinded, multicenter study to evaluate the efficacy and safety of rapcabtagene autoleucel versus rituximab in participants with severe refractory diffuse cutaneous systemic sclerosis (dcSSc). This study comprises two cohorts: * A Lead-in Cohort enrolling participants to receive rapcabtagene autoleucel. * A Randomized Cohort enrolling participants to receive rapcabtagene autoleucel or rituximab. Participants in the rituximab arm whose disease is not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible per protocol. After end of study, participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 70 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Disease onset from the first non-Raynaud symptoms attributable to SSc (e.g., puffy hands, scleroderma, digital...
- ✓Severe, progressive systemic sclerosis disease defined by at least one of the following:
- ✓Progressive systemic sclerosis-associated interstitial lung disease
- ✓Severe, progressive systemic sclerosis skin disease
- ✓Clinically significant systemic sclerosis-associated heart involvement at Screening
Who cannot take part
- ✗Participants with history of hypersensitivity to excipients in rapcabtagene autoleucel or to rituximab.
- ✗Any participant for whom treatment with rituximab is clinically inappropriate in the opinion of the investigator.
- ✗Rheumatic disease other than dcSSc, (except secondary Sjogren's syndrome or scleroderma myopathy),including limited...
- ✗Participants with pre-existing lung hypertension.
- ✗Significant kidney pathology at Screening.
See the full criteria
Where Is This Study? (1 UK site)
Novartis Investigative Site
Sheffield S10 2JF, United Kingdom