NCT03773328

A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS)

Phase I Trial To Evaluate The Safety Of CK0801 In Treatment-Resistant Guillain-Barré Syndrome (GBS)

• Status: Withdrawn
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Cellenkos, Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

The goal of this research study is to determine whether it is safe and practical to give CK0801 (a cord-blood derived T-regulatory cell product) to patients with Guillain-Barré Syndrome (GBS). Researchers also want to determine the highest possible dose that is safe to be given and to learn if CK0801 may improve the symptoms of GBS. There will be three doses of CK0801 given during this study. A minimum of three patients will be treated in each dose level. The dose a patient receives is dependent on the timing of when they enter the study, as after each dose level is completed the following patients will receive the next highest dose level.

Detailed description

CK0801 (Cord blood-derived T-regulatory cells) consists of ex vivo expanded T-regulatory cells with a flow cytometry phenotype of ≥ 60% CD4+CD25+ T-regulatory (Treg) cells and \< 10% CD4-CD8+ T-cytotoxic/suppressor cells. The cellular starting material of CK0801 is a single unit of umbilical cord blood (CBU) from a normal, healthy unrelated donor. Because Treg cells are present only at low frequency in circulating blood or umbilical cord blood, production of clinically relevant Treg cell doses requires ex vivo enrichment and expansion of Treg cells with a CD4+CD25+ phenotype. In their natural state, T-regulatory (Treg) cells play an important role in maintaining immune homeostasis and limiting autoimmune responses by modulating both innate and adaptive immunity. Based on literature reports of animal studies showing induction of immune tolerance by Treg cells in autoimmune diseases, graft-versus-host disease, and solid organ transplant rejection, the administration of Treg cells in human clinical trials has become an attractive strategy to induce immune tolerance in patients in a variety of clinical settings.

Conditions

• Guillain-Barré Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	CK0801	CK0801 (Cord blood-derived T-regulatory cells)

Timeline

• Start date: 2022-11-01
• Primary completion: 2025-02-01
• Completion: 2027-02-01
• First posted: 2018-12-12
• Last updated: 2023-01-04

Regulatory

• FDA-regulated drug study