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Pemphigus VulgarisPhase 1/2NCT04422912

A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CART Cells (Desmoglein 3 Chimeric Autoantibody Receptor T Cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T Cells [CABA-201]) in Subjects With Active, Pemphigus Vulgaris (RESET-PV)

A Phase 1/2 study testing DSG3-CAART and CABA-201 in people with Pemphigus Vulgaris, run by Cabaletta Bio at 13 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

A phase 1/2, open-label, safety and dosing study of autologous CART cells (desmoglein 3 chimeric autoantibody receptor T cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T cells [CABA-201]) in subjects with active, pemphigus vulgaris Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pemphigus Vulgaris
What is being tested?
DSG3-CAART and CABA-201
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
13 sitesUnited States
Study dates
29 Sep 2020to Jan 2029
Phase
Phase 1/2
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 mPV by prior or screening biopsy and prior positive anti- DSG3 antibody ELISA
  • mPV inadequately managed by at least one standard immunosuppressive therapies
  • Active mPV at screening
  • Anti-DSG3 antibody ELISA positive at screening
  • Inclusion Criteria for CABA-201 sub-study: Open to enrollment
  • Age ≥18
Show all 10Show fewer
  • Confirmed diagnosis of PV by prior or screening biopsy and prior positive DSG3 ELISA, IIF, and/or DIF
  • PV inadequately managed by at least one standard immunosuppressive therapy
  • Active PV at screening
  • DSG3 ELISA positive at screening

Who cannot join

  • Active cutaneous lesions associated with PV that indicates mucocutaneous rather than mucosal-dominant disease
  • Rituximab in last 12 months unless PV symptoms have recently worsened or anti-DSG3 antibody titers have recently increased
  • Prednisone > 0.25mg/kg/day
  • Other autoimmune disorder requiring immunosuppressive therapies
  • Investigational treatment in last 3 months
  • Have paraneoplastic pemphigus or active malignancy (not including non-melanoma skin cancer) or malignancy diagnosed within the previous 5 years
Show all 10Show fewer
  • Have received rituximab or other anti-CD20 or anti-CD19 therapies in last 12 months unless anti-DSG3 antibody titers have recently increased or PV symptoms have recently worsened
  • Prednisone > 0.25mg/kg/day
  • Other autoimmune disorder requiring immunosuppressive therapies
  • Treatment with any investigational agent within 4 weeks or 5 half-lives, whichever is longer.

Trial locations and contacts

13 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Cabaletta Bio contact

+1 267 759 3100 ext. 4444

clinicaltrials@cabalettabio.com

13 sites in 1 country

United States13 sites, 10 recruiting

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

    Sydney Chase, 617-525-7638, schase@bwh.harvard.edu

    Rebecca Wenzel-Rideout, rwenzel-rideout@bwh.harvard.edu

    Recruiting

  • University of North Carolina, Department of Dermatology

    Chapel Hill, North Carolina, 27516

    Withdrawn

  • Northwestern University

    Chicago, Illinois, 60611

    NU Dermatology CTU, 312-503-5944, NUderm-research@northwestern.edu

    Alan Zhou, MD

    Recruiting

  • UT Southwestern Medical Center, Dept. of Dermatology

    Dallas, Texas, 75235

    Aleuna Lee, 214-645-8968, aleuna.lee@utsouthwestern.edu

    Arturo Dominguez, MD

    Recruiting

  • MD Anderson Texas Medical Center

    Houston, Texas, 77030

    Alda Ashu, 713-792-1280, abmiraso@mdanderson.org

    Omar Pacha, MD

    Recruiting

  • University of Iowa

    Iowa City, Iowa, 52242

    Amanda Howell, 319-384-6843, amanda-steahr@uiowa.edu

    Emma Killoran, MD

    Recruiting

  • Yale University

    New Haven, Connecticut, 06520

    Kacie Carlson, 203-785-7432, kacie.carlson@yale.edu

    Mary Tomayko, MD, PhD

    Recruiting

  • Mount Sinai - Icahn School of Medicine

    New York, New York, 10029

    Withdrawn

  • Columbia University

    New York, New York, 10032

    Hei Ton "Jason" Chan, 646-317-6360, htc2110@cumc.columbia.edu

    Alexandra Coromilas, MD

    Recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Marie DeMarco, 215-349-5699, marie.demarco@pennmedicine.upenn.edu

    David Porter, MD

    Recruiting

  • Stanford University, Dept. of Dermatology

    Redwood City, California, 94063

    Kunju Sridhar, PhD, 650-721-4902, kunju@stanford.edu

    M. Peter Marinkovich, MD

    Recruiting

  • UC Davis, Dept. of Dermatology

    Sacramento, California, 95816

    Lauren Downing, 916-551-2635, ladowning@ucdavis.edu

    Mehrdad Abedi, MD

    Recruiting

  • University of Washington

    Seattle, Washington, 98109

    Completed

Other trials in United States

About this study

Official title: A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CART Cells (Desmoglein 3 Chimeric Autoantibody Receptor T Cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T Cells [CABA-201]) in Subjects With Active, Pemphigus Vulgaris

A phase 1/2, open-label, safety and dosing study of autologous CART cells (desmoglein 3 chimeric autoantibody receptor T cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T cells [CABA-201]) in subjects with active, pemphigus vulgaris

Read the detailed description

Pemphigus vulgaris (PV) is a B-cell mediated autoimmune disorder in which painful blisters are formed on the skin or mucosal membrane, including the mouth, nose, throat, eyelids, anus, and genitals.

This phase 1/2 study is being conducted in two parts. The first part is the main study conducted to find the maximum tolerated dose and optimal fractionated infusion schedule of an investigational cell therapy, DSG3-CAART, that can be given to patients with mucosal PV who are inadequately managed by standard therapies. This study is closed to enrollment.

The second part is a sub-study is being conducted to investigate if CABA-201, also called resecabtagene autoleucel, or "rese-cel", can be safely administered while achieving clinical responses without the need for preconditioning in mucosal-dominant PV (mPV) and mucocutaneous PV (mcPV) patients. This sub-study is open to enrollment.

DSG3-CAART or CABA-201 may potentially lead to complete and durable remission of disease.

Being tested
DSG3-CAART, CABA-201
Keywords
Pemphigus, Pemphigus Vulgaris, CAAR-T Therapy, CAR-T Therapy, Desmoglein 3, Cell Therapy, Autoimmune Disease, Autoimmunity, Skin Diseases, Vesiculobullous, Immunotherapy, Adoptive, Immune System Diseases, CABA-201, Anti-CD19 CAR-T therapy, Resecabtagene Autoleucel, Rese-cel
Medical subject headings
Pemphigus, Autoimmune Diseases, Skin Diseases, Vesiculobullous, Immune System Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DSG3-CAARTBiological

    Intravenous infusions of DSG3-CAART alone at different doses and different fractionations, with or without intravenous immunoglobulin, cyclophosphamide, and/or fludarabine.

  • CABA-201Biological

    Single intravenous infusion of CABA-201 at escalating doses, with or without preconditioning.

Study groups in NCT04422912
Study groupRoleWhat they receive
DSG3-CAARTExperimentalSingle or multiple intravenous infusion(s) of DSG3-CAART at varying dose levels. This study is now closed to enrollment.
Receives: Biological: DSG3-CAART
CABA-201ExperimentalInfusion of CABA-201 with or without cyclophosphamide and fludarabine preconditioning, or with or without cyclophosphamide preconditioning. This sub-study is open to enrollment.
Receives: Biological: CABA-201

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse events, including Dose Limit Toxicity

    Incidence of adverse events that are related to DSG3-CAART therapy

    Measured: 3 months

  2. For CABA-201 Sub-study: To evaluate adverse events reported by subjects

    Incidence and severity of AEs

    Measured: Up to 28 days after CABA-201 infusion

Secondary outcomes (14)

  1. Percent of CAAR-transduced cells

    Percent of total cells for infusion that are CAAR-transduced cells by flow cytometry

    Measured: Baseline

  2. Total DSG3-CAART positive cells

    Total DSG3-CAART positive cells for each manufacturing run by flow cytometry

    Measured: Baseline

  3. Cellular kinetics profile of DSG3-CAART

    Cellular kinetics profile of DSG3-CAART assessed by quantitative polymerase chain reaction

    Measured: Up to 36 months

  4. Change in DSG3 autoantibody titer

    Change in DSG3 autoantibody titer by ELISA compared to pre-infusion visit

    Measured: Up to 36 months

  5. Serologic remission

    Proportion of subjects achieving serologic remission, determined by negative DSG3 ELISA titer

    Measured: Up to 36 months

  6. Pemphigus Disease Area Index (PDAI)

    Change in PDAI compared to pre-infusion visit, scored on a 0-250 scale where a greater number represents more disease activity

    Measured: Up to 36 months

  7. Clinical remission: complete remission off therapy and complete remission on minimal therapy

    Proportion of subjects achieving complete remission, determined by a PDAI activity score of 0 for at least 2 months, either off therapy or on minimal therapy

    Measured: Up to 36 months

  8. Time to clinical remission and time to serologic remission

    Time to clinical remission and time to serologic remission from the last infusion

    Measured: up to 36 months

  9. Duration of clinical remission and duration of serologic remission

    Duration of clinical remission and duration of serologic remission sustained after achieving the initial remission

    Measured: up to 36 months

  10. For CABA-201 Sub-study: To evaluate adverse events reported by subjects

    Incidence and severity of AEs

    Measured: Up to 156 weeks after CABA-201 infusion

  11. For CABA-201 Sub-study: To characterize the pharmacodynamics (PD)

    Levels of B cells in the blood

    Measured: Up to 156 weeks

  12. For CABA-201 Sub-study: To characterize the pharmacokinetics (PK)

    Levels of CABA-201-positive T cells in the blood

    Measured: Up to 156 weeks

  13. For CABA-201 Sub-study: To evaluate autoantibody -related biomarkers

    Levels of serum anti-DSG3 and anti-DSG1 antibodies

    Measured: Up to 156 Weeks

  14. For CABA-201 Sub-study: To evaluate efficacy

    Absolute and percent change in disease activity by Pemphigus Disease Area Index (PDAI)

    Measured: Up to 156 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jun 2020
  2. Study start29 Sep 2020
  3. Primary completionJan 2029 (estimated)
  4. Study completionJan 2029 (estimated)

Resources

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
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04422912
Sponsor's protocol ID
CAB-101
Status last verified
Oct 2026
First submitted
2 Jun 2020
Last updated
15 Oct 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Nunez D, Stadanlick J, Furmanak T, Goldenberg J, Choudhary GS, Ishikawa L, Werner M, Vorndran Z, Hadi-Nezhad F, Thompson D, Braccia D, Ellis A, Cicarelli J, Flanagan S, Williams J, Kobulsky D, Toreki A, Schreiber C, Bass N, Impagliazzo A, Lam Q, Dominguez A, Awan F, Zhou X, Brieva J, Abedi M, Maverakis E, Kresa-Reahl K, Tummala R, Chang D, Binder GK, Volkov J, Basu S. CD19 CAR T-cell therapy is feasible for patients with pemphigus vulgaris treated without lymphodepletion in the RESET-PV trial. Blood. 2026 Jul 30;148(5):574-580. doi: 10.1182/blood.2025032093.

    PubMed 42201777

  • Shu J, Xie W, Chen Z, Offringa R, Hu Y, Mei H. The enchanting canvas of CAR technology: Unveiling its wonders in non-neoplastic diseases. Med. 2024 Jun 14;5(6):495-529. doi: 10.1016/j.medj.2024.03.016. Epub 2024 Apr 11.

    PubMed 38608709

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing DSG3-CAART and CABA-201 in people with Pemphigus Vulgaris, run by Cabaletta Bio at 13 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 10 inclusion and 10 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 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 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?

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 Cabaletta Bio (+1 267 759 3100 ext. 4444, clinicaltrials@cabalettabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse events, including Dose Limit Toxicity (time frame: 3 months). 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?

On the ClinicalTrials.gov record for NCT04422912, which also lists 2 linked publications.

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 15 Oct 2026.

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