Systemic SclerosisPhase 1/2NCT06400303
KYSA-5: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Systemic Sclerosis
A Phase 1/2 study testing KYV-101 and Standard lymphodepletion regimen in people with Systemic Sclerosis and Systemic Sclerosis - Diffuse Cutaneous, run by Kyverna Therapeutics at 2 sites in United States.
Trial summary
A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Subjects with Systemic Sclerosis Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis, Systemic Sclerosis - Diffuse Cutaneous, Systemic Sclerosis - 2013 ACR/EULAR Classification Criteria
- What is being tested?
- KYV-101 and Standard lymphodepletion regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesUnited States
- Study dates
- 6 Aug 2024to 16 Jun 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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
- Clinical diagnosis of SSc according to 2013 ACR/EULAR classification
- Clinical disease as follows: Classified as diffuse cutaneous SSc; ≤ 6 years since first non-Raynaud's sign or symptom; active disease
- Up to date on all recommended vaccinations per CDC or institutional guidelines for immune-compromised individuals
Who cannot join
- Clinically significant ILD
- Prior treatment with cellular therapy (CAR-T) or gene therapy product directed at any target
- History of allogeneic or autologous stem cell transplant
- Evidence of active hepatitis B or hepatitis C infection
- Positive serology for HIV
- Primary immunodeficiency
Show all 13Show fewer
- History of splenectomy
- History of stroke, seizure, dementia, Parkinson's disease, coordination movement disorder, cerebellar diseases, psychosis, paresis, aphasia, and any other neurologic disorder investigator considers would increase the risk for the subject
- Impaired cardiac function or clinically significant cardiac disease
- Previous or concurrent malignancy with the following exceptions:
- Adequately treated basal cell or squamous cell carcinoma (adequate wound healing is required prior to screening)
- In situ carcinoma of the cervix or breast, treated curatively and without evidence of recurrence for at least 3 years prior to screening
- A primary malignancy which has been completely resected, or treated, and is in complete remission for at least 5 years prior to screening
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Northwell Health
Great Neck, New York, 11021
Stanford University Medical Center
Palo Alto, California, 94305
About this study
Official title: KYSA-5: A Phase 1/2, Open-Label, Multicentre Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T Cell (CD19 CAR T) Therapy, in Subjects With Systemic Sclerosis
A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Subjects with Systemic Sclerosis
Read the detailed description
SSc is an immune-mediated rheumatic disease that is characterized by fibrosis of the skin and internal organs and vasculopathy. B-cells play a role in SSc, and the disease is characterized by the presence of autoantibodies such as anti-Scl-70 and anti-RNAP III antibodies. CD19-targeted chimeric antigen receptor (CAR) T-cells harness the ability of cytotoxic T-cells to directly and specifically lyse target cells to effectively deplete B-cells in the circulation and in lymphoid and potentially non-lymphoid tissues. KYV-101, a fully human anti-CD19 CAR T-cell therapy, will be investigated in adult subjects with systemic sclerosis.
- Conditions
- Systemic Sclerosis, Systemic Sclerosis - Diffuse Cutaneous, Systemic Sclerosis - 2013 ACR/EULAR Classification Criteria
- Being tested
- KYV-101, Standard lymphodepletion regimen
- Keywords
- KYV-101, systemic sclerosis, autoimmune disease, anti-CD19 CAR-T therapy, cellular therapy, Scleroderma
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KYV-101Biological
Anti-CD19 CAR-T cell therapy
Standard lymphodepletion regimenDrug
Also called Cyclophosphamide, Fludarabine
Standard lymphodepletion regimen
| Study group | Role | What they receive |
|---|---|---|
| KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 1) | Experimental | Dosing with KYV-101 CAR T cells Receives: Biological: KYV-101; Drug: Standard lymphodepletion regimen |
| KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 2) | Experimental | Recommended Phase 2 Dose Receives: Biological: KYV-101; Drug: Standard lymphodepletion regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events and laboratory abnormalities (Phase 1)
Measured: Up to 2 years
Frequency of Dose-Limiting Toxicities (DLTs) at each dose level (Phase 1)
Measured: Up to 2 years
To evaluate efficacy of KYV-101(Phase 2)
via revised Composite Response Index in Systemic Sclerosis (rCRISS) 30/5
Measured: 52 weeks
Secondary outcomes (6)
To evaluate pharmacodynamics (PK) of KYV-101 in blood (Phase 1 and Phase 2)
Chimeric antigen receptor-positive (CAR-positive) T-cell counts in blood
Measured: Up to 2 years
To evaluate pharmacodynamics (PD) of KYV-101 in blood (Phase 1 and Phase 2)
Levels of B-cells in blood
Measured: Up to 2 years
To evaluate pharmacodynamics (PD) of KYV-101 in blood (Phase 1 and Phase 2)
Levels of cytokines in serum
Measured: Up to 2 years
To evaluate efficacy of KYV-101 (Phase 1 and Phase 2)
revised Composite Response Index in Systemic Sclerosis (rCRISS) 30/5 response rate
Measured: 12, 24, 52 weeks
To evaluate immunogenicity (humoral response) of KYV-101 (Phase 1 and Phase 2)
Percentage of participants who develop anti-KYV-101 antibodies by immunoassays
Measured: Up to 2 years
To define the Recommended Phase 2 Dose (RP2D) (Phase 1)
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2024
- Study start6 Aug 2024
- Primary completion16 Jun 2026
- Study completion16 Jun 2026
Why it stopped: Study discontinued due to sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Systemic SclerosisStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyverna Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Kyverna Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06400303
- Sponsor's protocol ID
- KYSA-5
- Other IDs
- KYV101-005
- Status last verified
- Aug 2026
- First submitted
- 29 Aug 2023
- Last updated
- 31 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Bosello S, Angelucci C, Lama G, Alivernini S, Proietti G, Tolusso B, Sica G, Gremese E, Ferraccioli G. Characterization of inflammatory cell infiltrate of scleroderma skin: B cells and skin score progression. Arthritis Res Ther. 2018 Apr 18;20(1):75. doi: 10.1186/s13075-018-1569-0.
PubMed 29669578
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- KYSA-5: A Phase 1/2, Open-Label, Multicentre Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T Cell (CD19 CAR T) Therapy, in Subjects With Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT06400303
- Acronym / sponsor ID
- KYSA-5
- Conditions
- Systemic Sclerosis, Systemic Sclerosis - Diffuse Cutaneous, Systemic Sclerosis - 2013 ACR/EULAR Classification Criteria
- Keywords
- KYV-101, systemic sclerosis, autoimmune disease, anti-CD19 CAR-T therapy, cellular therapy, Scleroderma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study discontinued due to sponsor decision
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Kyverna TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 6 Aug 2024 (actual)
- Primary completion
- 16 Jun 2026 (actual)
- Study completion
- 16 Jun 2026 (actual)
- First posted
- 6 May 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.
A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Subjects with Systemic Sclerosis
Detailed description
SSc is an immune-mediated rheumatic disease that is characterized by fibrosis of the skin and internal organs and vasculopathy. B-cells play a role in SSc, and the disease is characterized by the presence of autoantibodies such as anti-Scl-70 and anti-RNAP III antibodies. CD19-targeted chimeric antigen receptor (CAR) T-cells harness the ability of cytotoxic T-cells to directly and specifically lyse target cells to effectively deplete B-cells in the circulation and in lymphoid and potentially non-lymphoid tissues. KYV-101, a fully human anti-CD19 CAR T-cell therapy, will be investigated in adult subjects with systemic sclerosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 1) | Experimental | Biological: KYV-101, Drug: Standard lymphodepletion regimenDosing with KYV-101 CAR T cells |
| KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 2) | Experimental | Biological: KYV-101, Drug: Standard lymphodepletion regimenRecommended Phase 2 Dose |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Clinical diagnosis of SSc according to 2013 ACR/EULAR classification
- Clinical disease as follows: Classified as diffuse cutaneous SSc; ≤ 6 years since first non-Raynaud's sign or symptom; active disease
- Up to date on all recommended vaccinations per CDC or institutional guidelines for immune-compromised individuals
Key exclusion criteria (13 listed)
- Clinically significant ILD
- Prior treatment with cellular therapy (CAR-T) or gene therapy product directed at any target
- History of allogeneic or autologous stem cell transplant
- Evidence of active hepatitis B or hepatitis C infection
- Positive serology for HIV
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 |
|---|---|---|
| KYV-101Anti-CD19 CAR-T cell therapy | Biological | KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 1), KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 2) |
| Standard lymphodepletion regimenAlso: Cyclophosphamide, FludarabineStandard lymphodepletion regimen | Drug | KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 1), KYV-101 CAR-T cells with lymphodepletion conditioning (Phase 2) |
Primary outcome
Incidence of adverse events and laboratory abnormalities (Phase 1)
Time frame: Up to 2 years
Frequency of Dose-Limiting Toxicities (DLTs) at each dose level (Phase 1)
Time frame: Up to 2 years
To evaluate efficacy of KYV-101(Phase 2)
Time frame: 52 weeks
via revised Composite Response Index in Systemic Sclerosis (rCRISS) 30/5
6 secondary outcomes
To evaluate pharmacodynamics (PK) of KYV-101 in blood (Phase 1 and Phase 2)
Time frame: Up to 2 years
To evaluate pharmacodynamics (PD) of KYV-101 in blood (Phase 1 and Phase 2)
Time frame: Up to 2 years
To evaluate pharmacodynamics (PD) of KYV-101 in blood (Phase 1 and Phase 2)
Time frame: Up to 2 years
To evaluate efficacy of KYV-101 (Phase 1 and Phase 2)
Time frame: 12, 24, 52 weeks
To evaluate immunogenicity (humoral response) of KYV-101 (Phase 1 and Phase 2)
Time frame: Up to 2 years
To define the Recommended Phase 2 Dose (RP2D) (Phase 1)
Time frame: Up to 2 years
Results and safety
The study was stopped early (reason given: Study discontinued due to sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2026.
- Background: PubMed 29669578Bosello S, Angelucci C, Lama G, Alivernini S, Proietti G, Tolusso B, Sica G, Gremese E, Ferraccioli G. Characterization of inflammatory cell infiltrate of scleroderma skin: B cells and skin score progression. Arthritis Res Ther. 2018 Apr 18;20(1):75. doi: 10.1186/s13075-018-1569-0.
- 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. This study is not recruiting at the moment.
MD study director
Kyverna Therapeutics, Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing KYV-101 and Standard lymphodepletion regimen in people with Systemic Sclerosis and Systemic Sclerosis - Diffuse Cutaneous, run by Kyverna Therapeutics at 2 sites 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 3 inclusion and 13 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: stopped early, as of the sponsor's last update on 31 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study discontinued due to sponsor decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events and laboratory abnormalities (Phase 1) (time frame: Up to 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06400303, which also lists 1 linked publication.
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 31 Aug 2026.