NCT03239470

Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus

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)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) · NIH
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Pemphigus Foliaceus
• Pemphigus Vulgaris

Interventions

Type	Name	Description
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.
BIOLOGICAL	Cohort 2: 2.5x10^8 PolyTregs	Each participant will receive a target cell dose of 2.5x10\^8 polyclonal Tregs.

Timeline

• Start date: 2017-10-10
• Primary completion: 2020-12-10
• Completion: 2023-01-09
• First posted: 2017-08-04
• Last updated: 2024-02-15
• Results posted: 2021-11-22

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study