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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
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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.
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Inclusion and exclusion criteria
Key Inclusion Criteria:
Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but not limited to the following subtypes:
Patients with human leukocyte antigen (HLA) matched related or unrelated donors
Adequate end organ function as defined in study protocol
Age ≤ 12 years
Prior donor of appropriate age (≥ 5 years old) available for re-collection of stem cells
Previous allogeneic Hematopoietic Cell Transplantation HCT (≥ 6 months post initial transplant) with poor graft function
Key Exclusion Criteria:
Primary purpose
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40 participants in 1 patient group
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Clinical Trials Jasper Therapeutics, Inc.
Data sourced from clinicaltrials.gov
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