Lupus NephritisPhase 1NCT06708845
US Zamto-cel Autoimmune Diseases
A Phase 1 study testing zamtocabtagene autoleucel, Cyclophosphamide and Fludarabine in people with Lupus Nephritis and Systemic Lupus Erythematosus, run by Miltenyi Biomedicine GmbH at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lupus Nephritis, Systemic Lupus Erythematosus, Systemic Sclerosis (SSc)
- What is being tested?
- zamtocabtagene autoleucel, Cyclophosphamide and Fludarabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2026to Jul 2028
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- •Confirmed diagnosis of autoimmune disease (SLE-Non-renal, SLE-LN, SSc/ dcSSc)
- 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
- 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.
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- 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)
- 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)
Who cannot join
- 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).
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- Systemic Lupus Erythematosus-Non-renal Key Inclusion/Exclusion Criteria
- Subjects with neuropsychiatric SLE.
- Drug-induced SLE.
- Systemic Lupus Erythematosus - Lupus Nephritis Key Inclusion/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
- "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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Froedtert Hospital and the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Kiley Timler, ktimler@mcw.edu
About this study
Official title: A Phase I Multicohort Trial of Zamtocabtagene Autoleucel (Zamto-Cel) in Subjects With Severe Refractory Autoimmune Diseases
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.
Read the detailed description
This is a Phase 1, multicohort, dose-finding study evaluating autologous T cells engineered to target dual CD19 and CD20 antigens in subjects with refractory autoimmune diseases following standard therapy. The investigational product, Zamto-cel, is a chimeric antigen receptor T-cell (CAR-T) therapy genetically engineered to enable subjects' T cells to express CARs on their surfaces.
Eligible subjects will undergo leukapheresis for the collection of cells required for manufacturing. Prior to infusion of the fresh CAR-T product, subjects will receive a lymphodepleting regimen consisting of cyclophosphamide and fludarabine. 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.
The study will initially enroll 3 subjects per cohort in a staggered manner to evaluate safety. Upon confirmation of safety, the study will proceed to cohort-specific recommended Phase 2 dose (RP2D) and dose expansion phases. Subjects will be monitored for up to 1 year to assess safety, preliminary efficacy, and health-related quality of life (HRQoL). Additional long-term follow-up will be conducted under a separate long-term follow-up protocol.
- Conditions
- Lupus Nephritis, Systemic Lupus Erythematosus, Systemic Sclerosis (SSc), Diffuse Cutaneous Systemic Sclerosis
- Being tested
- zamtocabtagene autoleucel, Cyclophosphamide, Fludarabine
- Keywords
- Chimeric antigen receptor, CAR T, Zamtocabtagene autoleucel, Autoimmune Disease, Immune System Diseases, SLE-Non renal, SLE-LN, SSc, dcSSc, Lupus
- Medical subject headings
- Lupus Nephritis, Lupus Erythematosus, Systemic, Scleroderma, Systemic, Scleroderma, Diffuse, Autoimmune Diseases, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zamtocabtagene autoleucelBiological
chimeric antigen receptor T-cell (CAR-T) therapy
CyclophosphamideDrug
Lymphodepleting chemotherapy
FludarabineDrug
Lymphodepleting chemotherapy
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1 | Experimental | Not described Receives: Biological: zamtocabtagene autoleucel; Drug: Cyclophosphamide; Drug: Fludarabine |
| Dose Level 2 | Experimental | Not described Receives: Biological: zamtocabtagene autoleucel; Drug: Cyclophosphamide; Drug: Fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence and severity of adverse events (AEs), adverse events of special interest (AESIs), and serious adverse events (SAEs)
Measured: From enrollment through study completion 12 months post zamto-cel infusion
The proportion of subjects with dose-limiting toxicities (DLTs) up to Day 28 and determination of recommended Phase 2 dose (RP2D)
Measured: From enrollment through Day 28 post zamto-cel infusion
Secondary outcomes (4)
The incidence and severity of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS)
Measured: From enrollment through study completion 12 months post zamto-cel infusion
Clinical response at Week 4, 12, 24, and 52 evaluated by defined disease-specific activity measures in SLE-Non renal, SLE-LN, and SSc/dcSSc
Measured: From enrollment through study completion 12 months post zamto-cel infusion
The duration of remission or low disease activity status in respective diseases under the study
Measured: From enrollment through study completion 12 months post zamto-cel infusion
Persistence, maximal drug concentration (Cmax), time to reach Cmax, area under the concentration curve, and phenotype of zamto-cel
Measured: From enrollment through study completion 12 months post zamto-cel infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2024
- Study startJul 2026 (estimated)
- Primary completionJan 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lupus NephritisStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Miltenyi Biomedicine GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Esther Eromosele, MD, Study director, Miltenyi Biomedicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06708845
- Sponsor's protocol ID
- M-2024-423
- Status last verified
- Jun 2026
- First submitted
- 26 Nov 2024
- Last updated
- 11 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Multicohort Trial of Zamtocabtagene Autoleucel (Zamto-Cel) in Subjects With Severe Refractory Autoimmune Diseases
- ClinicalTrials.gov ID
- NCT06708845
- Acronym / sponsor ID
- M-2024-423
- Conditions
- Lupus Nephritis, Systemic Lupus Erythematosus, Systemic Sclerosis (SSc), Diffuse Cutaneous Systemic Sclerosis
- Keywords
- Chimeric antigen receptor, CAR T, Zamtocabtagene autoleucel, Autoimmune Disease, Immune System Diseases, SLE-Non renal, SLE-LN, SSc
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Miltenyi Biomedicine GmbHIndustry (pharma, biotech, devices)
- Study start
- Jul 2026 (estimated)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 27 Nov 2024 (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.
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.
Detailed description
This is a Phase 1, multicohort, dose-finding study evaluating autologous T cells engineered to target dual CD19 and CD20 antigens in subjects with refractory autoimmune diseases following standard therapy. The investigational product, Zamto-cel, is a chimeric antigen receptor T-cell (CAR-T) therapy genetically engineered to enable subjects' T cells to express CARs on their surfaces.
Eligible subjects will undergo leukapheresis for the collection of cells required for manufacturing. Prior to infusion of the fresh CAR-T product, subjects will receive a lymphodepleting regimen consisting of cyclophosphamide and fludarabine. 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.
The study will initially enroll 3 subjects per cohort in a staggered manner to evaluate safety. Upon confirmation of safety, the study will proceed to cohort-specific recommended Phase 2 dose (RP2D) and dose expansion phases. Subjects will be monitored for up to 1 year to assess safety, preliminary efficacy, and health-related quality of life (HRQoL). Additional long-term follow-up will be conducted under a separate long-term follow-up protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1 | Experimental | Biological: zamtocabtagene autoleucel, Drug: Cyclophosphamide, Drug: Fludarabine |
| Dose Level 2 | Experimental | Biological: zamtocabtagene autoleucel, Drug: Cyclophosphamide, Drug: Fludarabine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- •Confirmed diagnosis of autoimmune disease (SLE-Non-renal, SLE-LN, SSc/ dcSSc)
- 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
- Positive for at least 1 of the following autoantibodies at Screening: anti- double stranded DNA or anti-Smith
Key exclusion criteria (14 listed)
- 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
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 |
|---|---|---|
| zamtocabtagene autoleucelchimeric antigen receptor T-cell (CAR-T) therapy | Biological | Dose Level 1, Dose Level 2 |
| CyclophosphamideLymphodepleting chemotherapy | Drug | Dose Level 1, Dose Level 2 |
| FludarabineLymphodepleting chemotherapy | Drug | Dose Level 1, Dose Level 2 |
Primary outcome
The incidence and severity of adverse events (AEs), adverse events of special interest (AESIs), and serious adverse events (SAEs)
Time frame: From enrollment through study completion 12 months post zamto-cel infusion
The proportion of subjects with dose-limiting toxicities (DLTs) up to Day 28 and determination of recommended Phase 2 dose (RP2D)
Time frame: From enrollment through Day 28 post zamto-cel infusion
4 secondary outcomes
The incidence and severity of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS)
Time frame: From enrollment through study completion 12 months post zamto-cel infusion
Clinical response at Week 4, 12, 24, and 52 evaluated by defined disease-specific activity measures in SLE-Non renal, SLE-LN, and SSc/dcSSc
Time frame: From enrollment through study completion 12 months post zamto-cel infusion
The duration of remission or low disease activity status in respective diseases under the study
Time frame: From enrollment through study completion 12 months post zamto-cel infusion
Persistence, maximal drug concentration (Cmax), time to reach Cmax, area under the concentration curve, and phenotype of zamto-cel
Time frame: From enrollment through study completion 12 months post zamto-cel infusion
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 11 Jun 2026.
- 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.
Esther Eromosele, MD study director
Miltenyi Biomedicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing zamtocabtagene autoleucel, Cyclophosphamide and Fludarabine in people with Lupus Nephritis and Systemic Lupus Erythematosus, run by Miltenyi Biomedicine GmbH at 1 site in United States.
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 16 inclusion and 14 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 11 Jun 2026. 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 United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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 Sadie Swift (617-218-0044, clinicaltrials@miltenyi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence and severity of adverse events (AEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) (time frame: From enrollment through study completion 12 months post zamto-cel infusion). 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 11 Jun 2026.