Purpose of this Study
Participants will take part in the main study, including screening, treatment, and follow up, for about 3 years. After receiving the study treatment, they will continue in a long term safety monitoring program for an additional 15 years so researchers can track their health and any delayed side effects.
Who Can Participate?
Eligibility
Participants may be able to join if they are 6 to 17 years old and have a definite or probable inflammatory muscle disease, including juvenile inflammatory muscle disease. They must have blood test results showing certain disease-related antibodies and signs of active disease, such as abnormal blood tests, MRI findings, muscle biopsy results, or a skin rash related to the disease.
Age Range
6-17
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is testing a personalized CAR T cell therapy called CABA-201 in people with inflammatory muscle diseases, including juvenile forms of these diseases. Researchers want to learn if the treatment is safe and well tolerated. The treatment uses a participant's own immune cells, which are modified in a laboratory and then returned to the body to help remove immune cells that may be causing the disease.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of
Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Subjects with Active Idiopathic Inflammatory Myopathy or Active
Juvenile Idiopathic Inflammatory Myopathy
Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Subjects with Active Idiopathic Inflammatory Myopathy or Active
Juvenile Idiopathic Inflammatory Myopathy
Principal Investigator
Kaveh
Ardalan
Protocol Number
PRO00116563
NCT ID
NCT06154252
Phase
I/II
Enrollment Status
Open to Enrollment