US Zamto-cel Autoimmune Diseases
A Phase I Multicohort Trial of Zamtocabtagene Autoleucel (Zamto-Cel) in Subjects With Severe Refractory Autoimmune Diseases
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Procedures mentioned: Infusions
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "The CAR-T cell infusion will be administered intravenously at a dose of 2.5 x 10\^6 or 1.0 x 10\^6 CAR+ cells/kg body weight, based on the dose level assigned to the cohort."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This early-stage study tests the safety of zamtocabtagene autoleucel (zamto-cel) and how well people tolerate it. It is for people with lupus, lupus kidney disease, or systemic sclerosis whose disease has not responded well to standard treatment. These are autoimmune diseases, in which the body's defense system attacks its own tissues.
AID is a phase I multi-cohort study to assess the safety and tolerability of zamtocabtagene autoleucel (zamto-cel) in patients with refractory autoimmune diseases (SLE-Non renal, SLE-LN, SSc/dcSSc) after receiving standard therapy.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- All groups: You must have a confirmed diagnosis of one of the autoimmune diseases listed above.
- Both lupus groups: Blood tests must show anti-double-stranded DNA or anti-Smith antibodies—proteins that can attack your own body.
- Lupus without kidney disease: You must have a lupus activity score of at least 8 and severe disease in at least one organ on the study's rating scales.
- Lupus without kidney disease: Steroids and at least 2 of the listed lupus medicines must not have worked well enough. Each of those medicines must have been used for at least 3 months.
- Lupus kidney disease: A kidney biopsy—a small tissue sample—must show severe, active disease during screening or within the past 6 months.
- Lupus kidney disease: Disease must be worsening despite the highest doses you can tolerate of certain kidney-protecting blood pressure medicines, unless you are allergic to them or cannot tolerate them.
- Lupus kidney disease: Steroids, hydroxychloroquine, and at least 1 of the listed treatments must not have worked well enough, with the required treatment use of at least 3 months each.
- Systemic sclerosis: You must meet the study's rules for active, worsening skin thickening or worsening lung scarring with breathing symptoms and CT scan changes. Standard treatment must not have worked, such as at least 2 medicines that lower immune activity.
Who can't join
- All groups: You have had gene therapy, or you have had active cancer within the past 5 years.
- All groups: You have a serious, active fungal or bacterial infection.
- All groups: You have or have had lupus affecting the brain or spinal cord, or another disease of the brain or spinal cord.
- All groups: Your kidney filtering test, called eGFR, is below 45.
- All groups: Your bilirubin blood level is outside the normal range, unless a condition you were born with, such as Gilbert syndrome, explains the high level.
- Lupus without kidney disease: You have lupus affecting mental health or the nervous system, or lupus caused by a medicine.
- Lupus kidney disease: You have rapidly worsening kidney inflammation, shown by a doubling of blood creatinine within 3 months before joining or judged by the study doctor.
- Systemic sclerosis: You have active bleeding from widened blood vessels in the stomach, including bleeding within the past 6 months or as judged by the doctor; a sudden severe kidney problem from systemic sclerosis within the past year; or kidney damage from another condition.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
General Key Inclusion/Exclusion Criteria Across All Cohorts Inclusion Criteria: •Confirmed diagnosis of autoimmune disease (SLE-Non-renal, SLE-LN, SSc/ dcSSc) Exclusion Criteria: * Prior gene therapy treatment * Active malignancy within past 5 years * Significant active fungal or bacterial infection * History or presence of CNS lupus or other CNS disease * eGFR \< 45 mL/min/1.73 m\^2 * Total bilirubin outside the normal range (unless congenital hyperbilirubinemia such as Gilbert syndrome has been confirmed). Systemic Lupus Erythematosus-Non-renal Key Inclusion/Exclusion Criteria Inclusion Criteria: * Positive for at least 1 of the following autoantibodies at Screening: anti- double stranded DNA or anti-Smith * Systemic Lupus Erythematosus Disease Activity Index-2000 score ≥ 8 AND at least 1 British Isles Lupus Assessment Group (BILAG)-2004 Class A (severe manifestation) organ scores * Inadequate response to glucocorticoids and to at least 2 of the following treatments, used for at least 3 months each: cyclophosphamide, mycophenolic acid or its derivatives, belimumab, azathioprine, anifrolumab, methotrexate, rituximab, or obinutuzumab Exclusion Criteria: * Subjects with neuropsychiatric SLE. * Drug-induced SLE. Systemic Lupus Erythematosus - Lupus Nephritis Key Inclusion/Exclusion Criteria Inclusion Criteria: * Positive for at least 1 of the following autoantibodies at Screening: anti- double stranded DNA or anti-Smith * Confirmed LN diagnosis by kidney biopsy during screening or within the previous 6 months, with severe active phase of the disease. * Progressing despite maintenance on maximally tolerated doses of renin- angiotensin system (RAS) blocking agents, unless allergic to or intolerant of ACE inhibitors and ARBs * Inadequate response to glucocorticoids and hydroxychloroquine and at least 1 of the following treatments, used for at least 3 months each: cyclophosphamide, mycophenolic acid derivatives, belimumab, azathioprine, methotrexate, rituximab, obinutuzumab, calcineurin inhibitor (cyclosporin, tacrolimus or voclosporin) Exclusion Criteria: •Evidence of Rapidly progressive glomerulonephritis (defined as a doubling of serum creatinine within 3 months prior to enrollment) or as determined by the study investigator. Systemic Sclerosis/Diffuse Cutaneous Systemic Sclerosis Cohort Key Inclusion/ Exclusion Criteria Inclusion Criteria: * Active disease defined as: * Modified Rodnan skin score (mRSS) ≥ 16 units, in the prior 6 months, with 1 or more of the following: * Increase in mRSS by ≥ 3 units or 10% * Involvement of 1 new body area with increase in mRSS by ≥ 2 units * Involvement of 2 new body areas with increase by ≥ 1 mRSS unit OR * Progressive interstitial lung disease (ILD) defined as: \- Worsening of respiratory symptoms and an increased extent of fibrosis evaluated by high-resolution computed tomography * Lack of response to standard therapy (e.g., failure of ≥ 2 immunosuppressive therapies) Exclusion Criteria: * "Active" gastric antral vascular ectasia, as evidenced by bleeding (ie, on esophagogastroduodenoscopy) in the past 6 months or as per Investigator's assessment. * History of SSc renal crisis within 1 year prior to Screening; presence of kidney impairment due to conditions other than SSc
Study sites (1)
WI
- Froedtert Hospital and the Medical College of Wisconsin · Milwaukee, WI 53226Kiley Timler · Study contact · ktimler@mcw.eduMore trials in Milwaukee