Brief Title
JSP191 Antibody Targeting Conditioning in SCID Patients
Official Title
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of JSP191 for Hematopoietic Cell Transplantation Conditioning to Achieve Engraftment and Immune Reconstitution in Subjects With SCID
Brief Summary
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
Detailed Description
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 are depleted.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events
Condition
SCID
Intervention
Humanized anti-CD117 Monoclonal Antibody (JSP191)
Study Arms / Comparison Groups
Blood Stem Cell Transplant w/ anti-CD117 conditioning
Description: The study will enroll two groups: Group A: previously transplanted SCID patients; Group B: newly diagnosed SCID. The study plans to assess JSP191 in different dose cohorts. Patients will receive a single dose of intravenous JSP191 antibody followed by monitoring for antibody clearance. Once the antibody has cleared below a certain level, patients will receive stem cell transplant and be monitored for hematopoietic recovery.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
40
Start Date
March 20, 2017
Completion Date
August 2027
Primary Completion Date
August 2024
Eligibility Criteria
Key Inclusion Criteria:
All patient groups must have:
1. Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but
not limited to the following subtypes:
1. T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient
2. T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
3. T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient OR Variant
SCID with absent or low T cell function, Omenn syndrome, Leaky SCID, Reticular
dysgenesis, Adenosine deaminase deficiency, and Purine nucleoside phosphorylase
deficiency may be included after consultation with the medical monitor.
2. Patients with human leukocyte antigen (HLA) matched related or unrelated donors
3. Adequate end organ function as defined in study protocol
Key Exclusion Criteria:
1. Patients with any acute or uncontrolled infections
2. Patients receiving any other investigational agents, or concurrent biological,
chemotherapy, or radiation therapy
3. Patients with active malignancies
4. Active GVHD within 6 months prior to enrollment, or on immunosuppressive therapy for
GVHD
Gender
All
Ages
3 Months - N/A
Accepts Healthy Volunteers
No
Contacts
Rajni A. Agarwal-Hashmi, M.D., 650-549-1270, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02963064
Organization ID
JAS-BMT-CP-001
Responsible Party
Sponsor
Study Sponsor
Jasper Therapeutics, Inc.
Study Sponsor
Rajni A. Agarwal-Hashmi, M.D., Principal Investigator, Lucile Packard Children's Hospital
Verification Date
May 2021