AMG191 Conditioning/CD34+CD90 Stem Cell Transplant Study for SCID Patients

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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


Primary Outcome

Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events




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.


* 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


Estimated Enrollment


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




3 Months - N/A

Accepts Healthy Volunteers



Rajni A. Agarwal-Hashmi, M.D., 650-549-1270, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Jasper Therapeutics, Inc.

Study Sponsor

Rajni A. Agarwal-Hashmi, M.D., Principal Investigator, Lucile Packard Children's Hospital

Verification Date

May 2021