Azienda Unita Sanitaria Locale Di Bologna
Bologna, Italy
Not yet recruitingRare disease
Safety and dose‑finding study of CD19‑CART01 in patients with SLE, SSc, DM/PM, IMNM, AAV, MG, NMOSD, or chronic GVHD
Verified siteInvestigational
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What is this trial about?
A plain-language summary of the goals, design and what participants do
The trial includes patients with Dermatomyositis, Polymyositis, Systemic Sclerosis, Neuromyelitis Optica, ANCA-associated vasculitis, Myasthenia Gravis, Immune-Mediated Necrotizing Myopathy, Systemic Lupus Erythematosus and chronic Graft-versus-Host Disease. The investigational product is a laboratory‑grown cell therapy called CD19-CART01, which belongs to the class of CAR T-cell treatments and is administered by intravenous infusion.
The purpose of the study is to evaluate the safety and determine the appropriate dose of the therapy for these conditions. After an initial screening period, participants receive a single infusion of the product and remain in the clinic for a short observation period. Follow‑up visits are scheduled over the next several months to monitor health status, collect blood samples, and assess any side effects.
Safety monitoring focuses on reactions such as cytokine release syndrome, which feels like a flu‑like illness, and on infections that may arise. Blood tests track how long the engineered cells stay in the body and whether the targeted immune cells are reduced. At later visits, participants complete questionnaires that measure changes in symptoms and overall quality of life.
The research process
The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
49 criteria
- Age between 4 and 70 years, depending on the specific disease group.
- Life expectancy of at least 12 weeks (you are expected to live for the next three months).
- Ability to perform daily activities at a level of 50% or higher on the Karnofsky/Lansky scale (a measure of overall fitness).
- Good overall organ health, including:
- Liver function: total bilirubin no more than three times the normal limit, and liver enzymes (AST, ALT) no more than five times normal.
- Kidney function: not needing dialysis.
- Heart function: left‑ventricle ejection fraction (a measure of pumping ability) of at least 45%.
- Having a vein that can be used for leukapheresis (a procedure that collects white blood cells) or being able to have a catheter placed safely.
- Negative blood culture from any central line (no infection in the bloodstream) within the week before leukapheresis.
- For women who could become pregnant: a negative pregnancy test within 8 days before screening and agreement to use birth control during the study and for 12 months after the treatment.
- For men who could father a child: agreement to use birth control during the study and for 12 months after the treatment.
- Willingness and ability to attend all study visits and follow study procedures.
- Signing a voluntary informed consent form (or having a parent/guardian sign if you are under 18).
- Diagnosis of one of the target diseases (e.g., systemic lupus erythematosus, systemic sclerosis, dermatomyositis, polymyositis, immune‑mediated necrotizing myopathy, ANCA‑associated vasculitis, myasthenia gravis, neuromyelitis optica, or chronic graft‑versus‑host disease) confirmed by the official classification criteria for that disease.
- For systemic lupus erythematosus (SLE) (adults):
- Positive lupus‑specific antibodies (anti‑dsDNA, anti‑histone, anti‑nucleosome, or anti‑Sm).
- Active disease measured by a SLEDAI‑2K score of 6 or higher (or 4 with at least one clinical sign).
- Not responding adequately to steroids and at least two other lupus medicines (e.g., hydroxychloroquine, mycophenolate, belimumab, methotrexate, rituximab).
- For systemic lupus erythematosus (SLE) (children):
- Active disease defined by a SLEDAI‑2K score of 8 or higher.
- Insufficient response to steroids and at least two other lupus medicines.
- For systemic sclerosis (SSc) (adults):
- Diffuse skin disease with the typical auto‑antibody pattern.
- Evidence of rapid disease progression, such as disease duration ≤5 years, skin thickness score (mRSS) between 10‑40, and elevated inflammation markers (CRP ≥ 6 mg/L, ESR ≥ 28 mm/h, or high platelet count).
- Not responding adequately to steroids and at least two other medicines (e.g., mycophenolate, azathioprine, nintedanib, methotrexate, rituximab).
- For juvenile systemic sclerosis (children):
- Diffuse skin disease with an mRSS score of 10‑35.
- Active disease shown by worsening skin scores, lung involvement, heart involvement, high blood pressure in the lungs, or gastrointestinal problems.
- Insufficient response to steroids and at least two other medicines.
- For dermatomyositis / polymyositis (DM/PM) (adults):
- Meets the 2017 classification criteria and has at least one of: a specific muscle antibody, a positive muscle biopsy, or characteristic skin rash (Gottron’s sign/papules or heliotrope rash).
- Shows muscle weakness (MMT score < 142) plus two additional signs such as pain, physician rating, functional score (HAQ > 0.25), elevated muscle enzymes (CK or aldolase > 2 × normal), MRI evidence of muscle inflammation, or abnormal EMG.
- Not responding adequately to steroids and at least two other medicines (e.g., mycophenolate, cyclosporine, azathioprine, tacrolimus, methotrexate, rituximab, IV immunoglobulin).
- For juvenile dermatomyositis (children):
- Meets the 2017 criteria for definite or probable disease.
- Active disease shown by low muscle function scores (CMAS < 48 or MMT8 < 78) and a physician global assessment > 2.5 /10, or very high muscle enzyme levels (≥ 3 × normal) or MRI evidence of inflammation.
- Insufficient response to steroids and at least two other medicines.
- For immune‑mediated necrotizing myopathy (IMNM) (adults):
- Meets the ENMC criteria, meaning you have high muscle enzyme levels, proximal muscle weakness, and either:
- Anti‑HMGCR antibodies, or
- Meets the ENMC criteria, meaning you have high muscle enzyme levels, proximal muscle weakness, and either:
- Anti‑SRP antibodies, or
- No specific antibodies but a muscle biopsy showing inflammation.
- For ANCA‑associated vasculitis (adults and children):
- Meets the 2022 classification criteria for MPA, GPA, or EGPA.
- Has ANCA antibodies directed against proteinase 3 or myeloperoxidase.
- Shows disease activity on the Birmingham Vasculitis Activity Score (BVAS) – at least one major sign or three minor signs, or two kidney signs (blood and protein in urine).
- Did not respond adequately to steroids combined with cyclophosphamide or a B‑cell‑targeting drug.
- For myasthenia gravis (MG) (adults):
- Presence of antibodies against the acetylcholine receptor, MuSK, or LRP4.
- Classification as MGFA class IIA‑IV (moderate to severe disease).
- Baseline MG‑ADL (activities of daily living) score ≥ 6.
- At least one of the following:
- Symptoms remain despite immunosuppressive therapy and second‑line drugs.
- Intolerable side effects or contraindications to second‑line drugs.
- Need for plasma exchange or monthly IV immunoglobulin in the past 6 months.
- At least one myasthenic crisis (severe breathing difficulty) in the past 6 months.
- For neuromyelitis optica spectrum disorder (NMOSD) (adults and children):
- Positive AQP4‑IgG antibody test (a blood test specific for NMOSD).
- Either:
- At least one relapse after three months of approved NMOSD therapy (e.g., eculizumab, inebilizumab, satralizumab, ravulizumab) in the past year, or two relapses in the past two years (including one in the past year),
- Or inability to use those approved drugs because of side effects or medical reasons.
- For chronic graft‑versus‑host disease (cGVHD) (adults and children):
- Disease that has not improved after at least three different prior treatments.
- Involvement of at least one organ (skin, joints/fascia, lung, or liver) with a moderate to severe score (NIH score 2‑3) as defined by the study.
Who cannot join the trial?
20 criteria
- The doctor believes another approved or easier treatment would be better for you.
- Your white blood cell count called ANC is lower than 1,000 cells per microliter (very low neutrophils).
- Your hemoglobin (the protein that carries oxygen) is less than 8 g/dL, meaning you are anemic.
- Your platelet count is less than 30,000 per microliter, which can increase bleeding risk.
- Your CD3+ T‑cell count is below 100 cells per microliter, indicating very few immune cells.
- Any medical problem or lab test result that puts you at high risk or would make the study results unclear.
- Uncontrolled serious other illnesses, severe liver disease (a Child‑Pugh score ≥ 10), or unstable heart disease.
- A severe or uncontrolled infection at the time of screening.
- Infection with HIV, hepatitis B (positive HBcAb or HBsAg) or active hepatitis C that was treated less than 12 weeks ago.
- Being pregnant or breastfeeding.
- Known allergy to any of the medicines used in the study.
- Having received CAR T‑cell therapy for an autoimmune disease before.
- Taking immunosuppressive or chemotherapy drugs within 7 days before the blood collection; cyclophosphamide must be stopped at least 3 weeks earlier.
- Taking systemic steroids (prednisone‑like) at a dose higher than 10 mg per day (or more than 0.3 mg per kg body weight) starting 7 days before blood collection.
- Having an active cancer diagnosed within the past 5 years, except certain skin cancers, treated in‑situ cervical or breast cancer, or certain blood cancers after a stem‑cell transplant with no evidence of disease.
- Taking systemic steroids higher than 40 mg per day (or more than 0.4 mg per kg) starting 2 weeks before the cell infusion, or higher than 20 mg per day (or more than 0.3 mg per kg) starting 1 week before infusion.
- Taking immunosuppressive or chemotherapy drugs within 7 days before the cell infusion.
- Receiving B‑cell‑depleting medicines (such as rituximab) within 28 days before the infusion.
- The collected blood product must contain at least 1 billion (1 × 10⁹) peripheral blood mononuclear cells; a lower amount cannot be used.
- Problems with the manufacturing of the CAR T‑cells, such as less than 20 % of cells successfully modified, cell viability below 80 %, killing ability under 60 %, or too many genetic copies (>10 per cell).
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Rome, Italy
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Investigated drugs
CD19-CART01 is a gene‑engineered cell therapy where a patient’s own immune cells (T‑cells) are modified in the lab to recognize and attack cells that have a protein called CD19 on their surface. CD19 is found on many B‑cells, which are part of the immune system and can be over‑active in certain autoimmune diseases and in chronic graft‑versus‑host disease. After the cells are engineered, they are given back to the patient through an intravenous (IV) infusion as a liquid suspension. In this trial, the therapy is being tested to see if it is safe and to find the best amount to use for conditions such as lupus, scleroderma, inflammatory muscle diseases, vasculitis, myasthenia gravis, neuromyelitis optica, and chronic graft‑versus‑host disease.
What is already known about the treatment
CD19-CART01 - This medication is given as an intravenous infusion of a cell suspension that contains genetically engineered T‑cells. It is currently an experimental therapy being studied in clinical trials for several rare autoimmune diseases, including systemic lupus erythematosus, systemic sclerosis, dermatomyositis/polymyositis, immune‑mediated necrotizing myopathy, ANCA‑associated vasculitis, myasthenia gravis, neuromyelitis optica, and chronic graft‑versus‑host disease. The treatment works by modifying the patient’s own T‑cells to recognize and destroy CD19‑positive B‑cells, which reduces the abnormal immune response that drives these conditions. CD19‑CART01 belongs to the class of CAR‑T cell therapies, a type of cellular immunotherapy that targets specific proteins on immune cells.
Investigated diseases
Dermatomyositis/Polymyositis
An inflammatory muscle disease that causes muscle weakness and, in dermatomyositis, a characteristic skin rash. The muscle inflammation progresses, leading to increasing difficulty with everyday activities, and can also involve the lungs and joints over time.
Systemic sclerosis
A connective‑tissue disorder that causes thickening and hardening of the skin and internal organs. As the disease advances, skin tightness worsens and fibrosis can develop in the lungs, heart, kidneys, and gastrointestinal tract.
Neuromyelitis optica spectrum disorder
An autoimmune condition that attacks the optic nerves and spinal cord, resulting in vision loss and limb weakness. Recurrent attacks can accumulate, leading to permanent visual impairment and increasing disability.
ANCA-associated vasculitis
Inflammation of small blood vessels driven by specific antibodies, causing damage to organs such as the kidneys, lungs, and skin. Disease activity can flare, leading to progressive loss of organ function if not controlled.
Myasthenia gravis
An autoimmune disorder where antibodies disrupt communication between nerves and muscles, producing muscle fatigue. Weakness typically begins with eye muscles and may spread to facial, limb, and respiratory muscles over time.
Immune‑mediated necrotizing myopathy
A rare muscle disease in which the immune system attacks muscle fibers, causing severe weakness. The condition can progress rapidly, sometimes affecting respiratory muscles and limiting daily activities.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2025-522821-37-00Protocol codeBETELGEUSEEstimated enrolment24 patientsSponsorAzienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
sourced from the EU Clinical Trials Register and site verification
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