A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with Severe Combined Immune Deficiency undergoing blood stem cell transplantation
A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with SCID undergoing blood stem cell transplantation. Blood Stem Cell transplantation offers the only potentially curative therapy for SCID. The biological conditioning regimen, JSP191, is an antibody that binds to CD117. CD117 is the receptor for Stem Cell Factor on blood forming cells. CD117 binding to Stem Cell Factor is critical for survival and maintenance of blood forming stem cells. The binding of JSP191 to CD117 blocks CD117 from binding to Stem Cell Factor on blood forming stem cells. In the absence of CD117/Stem Cell Factor binding, hematopoietic stem cells that are currently occupying the bone marrow niches in SCID patients exit from the bone marrow.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
23
Procedure: single intravenous infusion of JSP191 antibody
UCLA Mattel Children's Hospital
Los Angeles, California, United States
Lucile Packard Children's Hospital
Palo Alto, California, United States
UCSF Benioff's Children's Hospital
San Francisco, California, United States
Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events
The number of subjects experiencing dose limiting toxicities including adverse events and serious adverse events will be assessed.
Time frame: Up to 5 years post Donor Cell Transplant (28 days dose limiting toxicity period)
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
To enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism
Time frame: Up to 24 weeks post Donor Cell Transplant
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
To enable immune reconstitution, as determined by the production of naive T cells
Time frame: Weeks 36-104 post Donor Cell Transplant
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Children's National Medical Center
Washington D.C., District of Columbia, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
National Institutes of Health Clinical Center
Bethesda, Maryland, United States
University of Minnesota
Minneapolis, Minnesota, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States