Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03239470|
Recruitment Status : Active, not recruiting
First Posted : August 4, 2017
Last Update Posted : February 2, 2021
T cells, a type of white blood cell called a lymphocyte, play an important role in the immune system. One subtype, the regulatory T cell (Treg) helps to regulate the immune system and may provide protection against the development of autoimmune disease. The hope is that these naturally occurring Treg cells can be utilized for the treatment of autoimmune disease and potentially replace the use of chronic immunosuppressive therapies that are associated with multiple side effects. There has been a small study showing safe administration of Tregs with decreased disease activity in patients with insulin-dependent diabetes. Tregs are being studied in lupus, cancer and organ transplantation.
This phase I trial will be conducted as an open-label, dose-escalation, multicenter trial in adult participants with active pemphigus.The purpose of this study is to test the safety and effect of Treg therapy in participants who have skin (cutaneous) involvement due to pemphigus.
|Condition or disease||Intervention/treatment||Phase|
|Pemphigus Foliaceus Pemphigus Vulgaris||Biological: Cohort 1: 1.0 x 10^8 PolyTregs Biological: Cohort 2: 2.5x10^8 PolyTregs||Phase 1|
Up to 12 adults between the ages of 18 and 75 years of age who have been diagnosed with pemphigus and meet all other entry criteria will be enrolled to receive one infusion of their own expanded Tregs at one of the following doses:
- 1.0 x 10^8 PolyTregs or
- 2.5 x 10^8 PolyTregs.
Safety, disease activity, and mechanism of action will be assessed over a three year period, using biospecimens from blood and skin. Study therapy administration will occur during an overnight stay, followed by 2 weekly visits, then monthly visits from Week 8 to Week 12, then quarterly visits from Week 26 to Week 52, then twice a year visits until Week 156.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I, Open-Label, Multicenter Trial Exploring the Safety and Tolerability of Autologous Polyclonal Regulatory T Cell Therapy in Adults With Active Pemphigus (APG01)|
|Actual Study Start Date :||October 10, 2017|
|Actual Primary Completion Date :||December 10, 2020|
|Estimated Study Completion Date :||December 2022|
Experimental: Cohort 1: 1.0 x 10^8 PolyTregs
A single intravenous infusion of 1.0 x 10^8 PolyTregs will be administered.
Biological: Cohort 1: 1.0 x 10^8 PolyTregs
Each participant will receive a target cell dose of 1.0 x 10^8 polyclonal Tregs.
Experimental: Cohort 2: 2.5x10^8 PolyTregs
A single intravenous infusion of 2.5x10^8 PolyTregs will be administered.
Biological: Cohort 2: 2.5x10^8 PolyTregs
Each participant will receive a target cell dose of 2.5x10^8 polyclonal Tregs.
- Number of Significant Events as Defined by Grade 3 or higher adverse event(s) by Week 52 [ Time Frame: Baseline (Visit 0) to Week 52 ]Severity [Adverse Events (AEs) and Serious Adverse Events (SAEs)] will be reported using NCI-CTCAE grading criteria. Reference: the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0 http://ctep.cancer.gov/reporting/ctc.html.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03239470
|United States, California|
|University of California San Francisco School of Medicine: Department of Dermatology|
|San Francisco, California, United States, 94115|
|United States, Iowa|
|University of Iowa Health Care: Department of Dermatology|
|Iowa City, Iowa, United States, 52242|
|United States, North Carolina|
|Duke University Medical Center: Department of Dermatology|
|Durham, North Carolina, United States, 27710|
|United States, Texas|
|University of Texas Southwestern Medical Center: Department of Dermatology|
|Dallas, Texas, United States, 75390|
|Study Chair:||Haley Naik, MD,MHSc||University of California San Francisco School of Medicine: Department of Dermatology|
|Study Chair:||Anna Haemel, MD||University of California San Francisco School of Medicine: Department of Dermatology|
|Study Chair:||Michael Rosenblum, MD, Ph.D.||University of California San Francisco School of Medicine: Department of Dermatology|
|Study Chair:||Jeffrey Bluestone, Ph.D.||UCSF School of Medicine: UCSF Diabetes Clinic|
|Study Chair:||David Wofsy, M.D.||University of California San Francisco School of Medicine: Lupus Clinic and Rheumatology Clinical Research Center|