Early Phase 1 Vasculitis Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Major organ function must meet minimum levels set by the trial, including bone marrow, liver, kidney, heart, and lung function, based on blood and other tests (confirm specific values with trial site).
• Kidney function must generally be at or above a certain level, though people with lower kidney function or those on dialysis may still be considered if the treating doctor determines the potential benefit outweighs the risk and full informed consent is given.
• Heart scans must show no major structural problems and the heart must be pumping adequately (at least 55% efficiency).
• Lung function must be sufficient, with blood oxygen levels at or above 92%.
• Women who could become pregnant must use medically accepted contraception or abstain from sex during treatment and for at least 12 months after treatment ends.
• Written informed consent must be provided by the participant, or by a parent or guardian if the participant is a minor.

For Lupus (SLE) specifically:

• People aged 5 years or older.
• Diagnosis must meet recognised international classification criteria for lupus (2012 SLICC or 2019 EULAR/ACR criteria).
• Disease must remain active despite adequate treatment with steroids and at least two other immune-suppressing or biologic medicines for at least 3 months, or steroid doses cannot be reduced to a low level after 6 months of standard treatment.
• People who have experienced intolerable side effects from standard treatment, have reasons they cannot take standard treatment, or have had multiple treatment failures may also be considered (confirm with trial site).
• Disease activity must be at or above a certain score on a standard lupus activity scale (SLEDAI-2K ≥ 8, or ≥ 6 with additional organ involvement criteria — confirm with trial site).
• No episode of a serious complication called macrophage activation syndrome in the month before screening.
• Presence of lupus affecting the brain or nervous system (such as seizures, psychosis, or stroke-like events) requiring treatment within 60 days before screening.

For Drug-Resistant Kidney Disease (MDR-SRNS) specifically:

• People aged 3 years or older.
• Diagnosis of steroid-resistant nephrotic syndrome according to 2021 international kidney disease guidelines.
• Has not responded to at least 12 months of treatment with two different standard medicines (at least one being a calcineurin inhibitor such as cyclosporine or tacrolimus), or has relapsed after achieving remission.
• People who have not responded after 3 to 6 months of adequate treatment with one calcineurin inhibitor may also be considered if the treating doctor determines the benefit outweighs the risk and full informed consent is given.
• People unable to tolerate standard treatment may be considered if the treating doctor determines the benefit outweighs the risk and informed consent is given.
• Kidney biopsy must confirm a specific tissue type: either minimal change disease (MCD) or focal segmental glomerulosclerosis (FSGS).

For IgA Kidney Disease (IgA Nephropathy) specifically:

• People aged 5 years or older.
• Diagnosis confirmed by kidney biopsy.
• Must have been treated with a specific type of blood pressure and kidney-protecting medicine (ACEI or ARB) for at least 3 months.
• Must also meet at least one of: having been on steroids plus at least one other immune medicine for at least 3 months with significant protein in the urine; OR a rapid decline in kidney function (greater than 50% drop in eGFR within 3 months); OR unable to tolerate standard treatment, with the treating doctor determining benefit outweighs risk and informed consent obtained.
• People with other identified secondary causes of the disease, or with uncontrolled blood pressure, are not eligible.

For Systemic Sclerosis (Scleroderma) specifically:

• People aged 5 years or older.
• Diagnosis must meet 2013 international classification criteria for scleroderma.
• Certain scleroderma-related antibodies must be present in blood tests.
• A standard skin scoring measure must reach at least 15 out of 51.
• Must have been treated with steroids plus at least one other immune-modifying medicine for more than 3 months without significant improvement; OR has rapidly progressing disease unresponsive to standard treatment (with treating doctor determining benefit outweighs risk and informed consent obtained); OR is unable to tolerate standard treatment (with treating doctor determining benefit outweighs risk and informed consent obtained).

For ANCA-Associated Vasculitis (a type of blood vessel inflammation) specifically:

• People aged 5 years or older.
• Diagnosis must meet recognised international criteria for ANCA-associated vasculitis, including two specific subtypes (MPA or GPA).
• Disease must be classified as refractory (not responding well enough to standard treatment) or must have returned after remission requiring restart of intensive treatment.
• People with severe vasculitis that has not responded adequately to standard care may also be considered if the treating doctor determines benefit outweighs risk and informed consent is given.
• Active vasculitis must be confirmed by a standard activity score: at least 10 out of 63 on the appropriate scale for the participant's age.

Who may not be able to join:

• People with a known severe allergic reaction or contraindication to any medicines used in the trial, including cyclophosphamide or tocilizumab.
• People with severe heart failure (Grade III or IV on a standard medical scale).
• People with a history of congenital heart disease or a heart attack within the past 6 months, serious abnormal heart rhythms, significant fluid around the heart, serious heart muscle inflammation, or unstable vital signs requiring blood pressure-raising medicines.
• People with an active, uncontrolled infection, or an infection requiring systemic treatment at the time of screening.
• People with active tuberculosis of the lungs at the time of screening.
• People with active hepatitis B, active hepatitis C, HIV, syphilis, or active cytomegalovirus (CMV) infection, based on blood test results.
• People with a history of severe herpes infection (such as herpes affecting the brain or eyes, or widespread herpes), or who have had signs of herpes or chickenpox/shingles infection within 12 weeks before screening.
• People with active disease affecting the brain or central nervous system (other than lupus-related CNS involvement as specified in the inclusion criteria — confirm with trial site).
• People with a current or prior diagnosis of cancer or malignant disease.
• People with a secondary or inherited immune deficiency condition.
• People with a significant history of major medical conditions affecting the heart, hormones, blood, liver, immune system, metabolism, urinary system, lungs, nerves, skin, mental health, or kidneys that would prevent safe participation in the trial (except where the condition is lupus).
• People who have received a solid organ transplant or stem cell transplant within 3 months before screening, or who have had significant graft-versus-host disease within 2 weeks before screening.
• People who received a live vaccine within 4 weeks before screening.
• People who test positive on a blood pregnancy test.
• People who have taken part in another clinical trial and received any intervention within 3 months before enrolment.
• People with abnormal laboratory test results that the treating doctor considers clinically significant enough to prevent safe participation.
• People who, in the treating doctor's judgment, have any situation that may increase risk or affect the trial results.

Important: Always verify eligibility with the trial site directly before applying.