Gene Transfer for Patients With Sickle Cell Disease Using a Gamma Globin Lentivirus Vector
Brief description of study
This Phase 1/2 study will use a novel therapy, ARU-1801, for Sickle Cell Disease, using gene transfer of recombinant -globin lentivirus (LV) vector. The purpose of this study, which
involves research, is to determine whether an experimental procedure called
“gene transfer” is safe and effective.
If you are interested in learning more, or would like to find out if you are eligible, please contact the research team using the form at the end of the page.
Detailed description of study
Watch a a patient testimonial video here.
This experimental procedure is designed
to allow your body to produce red blood cells that do not sickle. It involves
the following:
Subjects will need to return to
the study site for multiple follow-up visits for 2 years after the ARU-1801
infusion. Then, a separate long-term follow-up (LTFU) clinical study will be
initiated. All subjects who complete the Year 2 study visit in this study will
be asked to consent and enroll into the LTFU study and will be followed for a
total of 15 years after the ARU-1801 infusion. Follow-up after Year 2 may continue
in this Phase 1/2 study until subjects have transitioned into the LTFU study.
a) Collection of
your own stem cells (blood-making cells)
b) Administration
of a chemotherapy drug called melphalan to prepare your body to receive the
gene modified stem cells
c) Infusion of
gene modified stem cells
Eligibility of study
You may be eligible for this study if you meet the following criteria:
-
Conditions:
sickle cell,gene transfer
-
Age: Between 18 Years - 99 Years
-
Gender: All
Updated on
07 Oct 2021.
Study ID: 843697
