Purpose of this Study
We are doing this study to find out if an experimental drug called CABA-201 (the study drug) is a safe and effective option for people with systemic sclerosis.
Who Can Participate?
Eligibility
Adults ages 18-75 who:<ul>
<li>Are diagnosed with systemic sclerosis</li>
<li>Have tried at least 2 different medications for at least 12 weeks each to treat their scleroderma and had to stop because the medications did not work adequately or caused unacceptable side effects</li></ul>
Previous medications that failed to be successful can include options like azathioprine, corticosteroids, hydroxychloroquine, intravenous immunoglobulin, JAK inhibitors, mycophenolate, methotrexate, rituximab, and tocilizumab.
For more information about this study, contact the study team at <a href= "mailto: khalia.stewart@duke.edu">khalia.stewart@duke.edu.</a>
<li>Are diagnosed with systemic sclerosis</li>
<li>Have tried at least 2 different medications for at least 12 weeks each to treat their scleroderma and had to stop because the medications did not work adequately or caused unacceptable side effects</li></ul>
Previous medications that failed to be successful can include options like azathioprine, corticosteroids, hydroxychloroquine, intravenous immunoglobulin, JAK inhibitors, mycophenolate, methotrexate, rituximab, and tocilizumab.
For more information about this study, contact the study team at <a href= "mailto: khalia.stewart@duke.edu">khalia.stewart@duke.edu.</a>
Age Range
18-75
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
If you choose to join this study, you will get a single dose of the study drug. The study drug is a type of intervention known as chimeric antigen receptor T cell (CAR-T) therapy. The study drug is made in a lab out of your own T cells that we will collect from your blood through a process called apheresis. This process involves passing your blood through a machine that removes T cells from the blood, and it then returns the rest of your blood products (e.g., red blood cells, white blood cells, plasma, and platelets) back to your body.
Before you get the study drug, you will come to Duke Hospital for a "pre-conditioning" visit that will involve giving you two drugs called cyclophosphamide and fludarabine. These drugs are commonly used to pre-condition cancer patients who receive CAR-T therapy. There is evidence that these medications can help the CAR-T cells multiply more quickly in people's bodies and to remain longer. The study doctor may decide to have you stay in the hospital overnight after you get your pre-conditioning regimen.
After pre-conditioning, we will schedule you to come in to get a dose of the study drug. The study drug is given as an infusion into a vein (IV). The study doctor might keep you in the hospital for up to 4 days after the infusion.
This study is intensive and will require you and a caregiver of yours to stay within 1 hour of Duke Hospital for the first 14 days after the study drug infusion. We will schedule you to come in for follow-up visits after you get the study drug. Your participation might last for up to 15 years, but you will not need to make in-person visits after year 5.
Before you get the study drug, you will come to Duke Hospital for a "pre-conditioning" visit that will involve giving you two drugs called cyclophosphamide and fludarabine. These drugs are commonly used to pre-condition cancer patients who receive CAR-T therapy. There is evidence that these medications can help the CAR-T cells multiply more quickly in people's bodies and to remain longer. The study doctor may decide to have you stay in the hospital overnight after you get your pre-conditioning regimen.
After pre-conditioning, we will schedule you to come in to get a dose of the study drug. The study drug is given as an infusion into a vein (IV). The study doctor might keep you in the hospital for up to 4 days after the infusion.
This study is intensive and will require you and a caregiver of yours to stay within 1 hour of Duke Hospital for the first 14 days after the study drug infusion. We will schedule you to come in for follow-up visits after you get the study drug. Your participation might last for up to 15 years, but you will not need to make in-person visits after year 5.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
DRA01: 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 Systemic Sclerosis
Principal Investigator
Ankoor
Shah
Protocol Number
PRO00115528
NCT ID
NCT06328777
Phase
I/II
Enrollment Status
Open to Enrollment