JSP191 Antibody Targeting Conditioning in SCID Patients

NCT ID: NCT02963064

Last Updated: 2025-07-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-20

Study Completion Date

2025-07-07

Brief Summary

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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

Detailed Description

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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.

Conditions

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SCID

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Blood Stem Cell Transplant w/ anti-CD117 conditioning

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.

Group Type EXPERIMENTAL

Humanized anti-CD117 Monoclonal Antibody (JSP191)

Intervention Type BIOLOGICAL

Procedure: single intravenous infusion of JSP191 antibody

Interventions

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Humanized anti-CD117 Monoclonal Antibody (JSP191)

Procedure: single intravenous infusion of JSP191 antibody

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

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 (no longer enrolling)
2. T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
3. T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient (no longer enrolling) 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
4. Age ≤ 12 years
5. Prior donor of appropriate age (≥ 5 years old) available for re-collection of stem cells
6. Previous allogeneic Hematopoietic Cell Transplantation HCT (≥ 6 months post initial transplant) with poor graft function

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
Minimum Eligible Age

3 Months

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Jasper Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rajni A. Agarwal-Hashmi, M.D.

Role: PRINCIPAL_INVESTIGATOR

Lucile Packard Children's Hospital

Christopher C. Dvorak, M.D.

Role: PRINCIPAL_INVESTIGATOR

UCSF Benioff's Children's Hospital

Joseph H. Oved, M.D.

Role: PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center

Theodore B. Moore, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Sharat Chandra, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Christen L Ebens, M.D., MPH

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Harry L Malech, M.D.

Role: PRINCIPAL_INVESTIGATOR

National Institutes of Health Clinical Center (CC)

Shanmuganathan Chandrakasan, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Healthcare of Atlanta

Neena Kapoor, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Los Angeles

Elizabeth D Hicks, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's National Research Institute

Susan Prockop, M.D.

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

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UCLA Mattel Children's Hospital

Los Angeles, California, United States

Site Status

Lucile Packard Children's Hospital

Palo Alto, California, United States

Site Status

UCSF Benioff's Children's Hospital

San Francisco, California, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Countries

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United States

Other Identifiers

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JAS-BMT-CP-001

Identifier Type: -

Identifier Source: org_study_id

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