- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04422912
Open-label Study to Determine the Maximum Tolerated Dose of DSG3-CAART in Mucosal-dominant PV Patients (mPV)
A Phase 1, Open-label, Safety and Dosing Study of Autologous Desmoglein 3 Chimeric Autoantibody Receptor T Cells (DSG3-CAART) in Subjects With Active, Anti-DSG3, Mucosal-dominant Pemphigus Vulgaris
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Anticipated)
Phase
- Phase 1
Contacts and Locations
Study Locations
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California
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Redwood City, California, United States, 94063
- Recruiting
- Stanford University, Dept. of Dermatology
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Contact:
- Isin Sinem Bagci, MD
- Phone Number: 650-724-8829
- Email: isbagci@stanford.edu
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Contact:
- Kunju Sridhar, PhD
- Phone Number: 650 721 4902
- Email: kunju@stanford.edu
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Principal Investigator:
- M. Peter Marinkovich, MD
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Sacramento, California, United States, 95816
- Recruiting
- UC Davis, Dept. of Dermatology
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Contact:
- Lauren Downing
- Phone Number: 916-551-2635
- Email: ladowning@ucdavis.edu
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Illinois
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Chicago, Illinois, United States, 60611
- Recruiting
- Northwestern University
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Contact:
- NU Dermatology CTU
- Phone Number: 312-503-5944
- Email: NUderm-research@northwestern.edu
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Iowa
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Iowa City, Iowa, United States, 52242
- Recruiting
- University of Iowa
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Contact:
- Amanda Steahr
- Phone Number: 319-384-6843
- Email: amanda-steahr@uiowa.edu
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Principal Investigator:
- Janet Fairley, MD
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New York
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New York, New York, United States, 10029
- Recruiting
- Mount Sinai - Icahn School of Medicine
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Contact:
- Jonathan Lagdameo
- Phone Number: 212-241-8552
- Email: jonathan.lagdameo@mssm.edu
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Principal Investigator:
- Keren Osman, MD
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North Carolina
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Chapel Hill, North Carolina, United States, 27516
- Recruiting
- University of North Carolina, Department of Dermatology
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Contact:
- Suzy Caballero
- Phone Number: 984-974-3682
- Email: nieves_caballero@med.unc.edu
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Principal Investigator:
- Donna A Culton, MD, PhD
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Recruiting
- University of Pennsylvania
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Contact:
- Joshua Bryer, BA, CCRC
- Phone Number: 267-251-6819
- Email: jbryer@pennmedicine.upenn.edu
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Principal Investigator:
- David Porter, MD
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Texas
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Dallas, Texas, United States, 75235
- Recruiting
- UT Southwestern Medical Center, Dept. of Dermatology
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Contact:
- Aleuna Lee
- Phone Number: 214-645-8968
- Email: aleuna.lee@utsouthwestern.edu
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Principal Investigator:
- Arturo Dominguez, MD
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Houston, Texas, United States, 77030
- Recruiting
- MD Anderson Texas Medical Center
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Contact:
- Dermatology Department
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Principal Investigator:
- Omar Pacha, MD
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Washington
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Seattle, Washington, United States, 98109
- Recruiting
- University of Washington
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Contact:
- Susan Ra
- Phone Number: 206-667-5310
- Email: sra@fredhutch.org
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Principal Investigator:
- Michi Shinohara, MD
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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 6 months
- Absolute lymphocyte count < 1,000/µL at screening
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: DSG3-CAART
Cohort A: Fractionated infusions of DSG3-CAART at increasing dose levels (6-9 groups) administered as a single cycle. Cohort B: Consolidation of infusion of DSG3-CAART to fewer fractionations than in Cohort A using the selected dose from Cohort A (1 group) administered as a single cycle. Cohort C: Infusion of final selected dose and fractionation of DSG3-CAART from Cohorts A and B (1 group) administered as a single cycle |
Intravenous infusions of DSG3-CAART alone at different doses and different fractionations.
Subjects may also receive varying doses of DSG3-CAART as part of a sub-study, which will employ pre-treatment with intravenous immunoglobulin, cyclophosphamide, and with or without fludarabine to potentially increase the in vivo expansion, persistence and activity of DSG3-CAART.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Adverse events, including Dose Limit Toxicity
Time Frame: 3 months
|
The primary endpoint of the study is the incidence of adverse events that are related to DSG3-CAART therapy within 3 months of DSG3-CAART cell infusion.
|
3 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percent of CAAR-transduced cells
Time Frame: Baseline
|
Percent of total cells for infusion that are CAAR-transduced cells by flow cytometry
|
Baseline
|
Total DSG3-CAART positive cells
Time Frame: Baseline
|
Total DSG3-CAART positive cells for each manufacturing run by flow cytometry
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Baseline
|
Cellular kinetics profile of DSG3-CAART
Time Frame: Up to 36 months
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Cellular kinetics profile of DSG3-CAART assessed by quantitative polymerase chain reaction
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Up to 36 months
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Change in DSG3 autoantibody titer
Time Frame: Up to 36 months
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Change in DSG3 autoantibody titer by ELISA compared to pre-infusion visit
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Up to 36 months
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Serologic remission
Time Frame: Up to 36 months
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Proportion of subjects achieving serologic remission, determined by negative DSG3 ELISA titer
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Up to 36 months
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Pemphigus Disease Area Index (PDAI)
Time Frame: Up to 36 months
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Change in PDAI compared to pre-infusion visit, scored on a 0-250 scale where a greater number represents more disease activity
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Up to 36 months
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Clinical remission: complete remission off therapy and complete remission on minimal therapy
Time Frame: Up to 36 months
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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
|
Up to 36 months
|
Time to clinical remission and time to serologic remission
Time Frame: up to 36 months
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Time to clinical remission and time to serologic remission from the last infusion
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up to 36 months
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Duration of clinical remission and duration of serologic remission
Time Frame: up to 36 months
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Duration of clinical remission and duration of serologic remission sustained after achieving the initial remission
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up to 36 months
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Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Cabaletta Bio, Cabaletta Bio
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CAB-101
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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