Clinical Trial

Disease: Systemic Lupus Erythematosus, SLE, (NCT06373991)

Disease info:

Systemic lupus erythematosus (SLE) is the most common form of lupus, a chronic autoimmune disease whereby the immune system attacks the body's own tissue and organs. SLE causes inflammation in connective tissues, such as cartilage and the lining of blood vessels. SLE can be mild or severe and can affect many parts of the body, including joints, skin, kidneys, heart, lungs, blood vessels, and brain. Individuals with SLE often experience periods of disease symptom flares and symptom remission. The symptoms of SLE vary within individuals and can range from mild to severe. 

Variants in multiple genes have been implicated in increasing the risk of developing SLE. The majority of these genes are involved in immune system function.

The first symptoms of SLE often manifest as fatigue, malaise, loss of appetite and weight loss. A characteristic symptom of SLE is the appearance of a red rash across the nose and cheeks, referred to as a “butterfly rash”. Skin problems are common in SLE, including a sun-reactive skin rash, calcium deposits under the skin known as calcinosis, damaged blood vessels and small red spots called petechiae. Kidney disease is also associated with SLE, as well as heart problems and symptoms arising from nervous system damage such as seizures. 
Other symptoms of lupus include arthritis, an unexplained fever, chest pain especially when inhaling or lying down, hair loss, discoloured fingers or toes, sun sensitivity, swelling in legs and around eyes, mouth ulcers and swollen glands, headaches, and dizziness.
There is currently no cure for lupus. Treatment involves managing symptom flares and reducing organ damage.

Frequency:
Overall prevalence is estimated to be 1-5 in 10,000. SLE affects women approximately nine times more often than men.
Official title:
A Phase 1 Study to Evaluate the Safety and Efficacy of ATHENA CAR-T in Subjects With Moderate or Severe Systemic Lupus Erythematosus
Who:

Contact 

Name: Chao Liu

Phone: (86)-10-80733899 ext 8039

Email: cliu@edigene.com

Contact Backup

Name: Yiding Zhao, PhD

Phone: (86)-10-80733899 ext 8103

Email: ydzhao@edigene.com

Sponsor:
Partners:
Locations:

China, Henan

The First Affiliated Hospital of Henan University of Science and Technology, Luoyang, Henan, China, 471003

Study start:
Jul. 24, 2024
Enrollment:
12 participants
Gene editing method:
CRISPR-Cas
Type of edit:
Gene knockout
Gene:
TRAC and Power3 gene
Delivery method:
Lentivirus - Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

The goal of this clinical trial is to test ATHENA CAR-T injection in adults with moderate to severe Systemic Lupus Erythematosus. The main question it aims to answer is:

• To evaluate the safety and tolerability of ATHENA CAR-T.

After screening, participants will be subjected to lymphodepletion regimen. After recovery, participants will be injected with ATHENA CAR-T injection and followed up to 24 months.

This study is a single center, one-arm, open label, phase I study aimed at evaluate the safety and effectiveness of ATHENA CAR-T treating moderate to severe SLE patients.

A traditional "3+3" design is used with two doses. DLT is monitored. Safety and effectiveness are followed up until 24 months post infusion of ATHENA CAR-T. Besides safety monitoring, efficacy is evaluated via SLEDAI-2000, BILAG-2004, PGA.

Last updated: Oct. 4, 2024
close
Search CRISPR Medicine