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

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Severe combined immunodeficiency
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Brief Title

AMG 191 Antibody Targeting Conditioning in SCID Patients

Official Title

A Phase 1 Study to Evaluate the Safety and Tolerability of Tandemly-purified Allogeneic CD34+CD90+ HSC Administered Following Conditioning With AMG 191 to Achieve Engraftment and Immune Reconstitution in Patients With SCID

Brief Summary

      This is a Phase 1 study to evaluate the safety and tolerability of an antibody conditioning
      regimen known as AMG 191, in patients with Severe Combined Immune Deficiency undergoing blood
      stem cell transplantation

Detailed Description

      This is a Phase 1 study to evaluate the safety and tolerability of an antibody conditioning
      regimen known as AMG 191, 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, AMG 191, 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 AMG 191
      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

Study Type

Interventional

Primary Outcome

Safety and tolerability of AMG 191 as conditioning therapy in SCID patients undergoing HCT: adverse events

Condition

SCID

Intervention

Humanized anti-CD117 Monoclonal Antibody (AMG 191)

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 AMG 191 in different dose cohorts. Patients will receive a single dose of intravenous AMG 191 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

34

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. Primary Immune Deficiency as defined by specific criteria, 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

          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., 650-549-1425, [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, Stanford University

Verification Date

August 2020