JSP191 Antibody Targeting Conditioning in SCID Patients

NCT02963064 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: JAS-BMT-CP-001
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 9

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

Conditions

SCID

Keywords

Immunodeficiency; Pediatric; SCID; Bone Marrow Transplantation; GVHD; Stem Cells; Chimerism; Transplant; BMT

Outcome Measures

Primary Outcomes (3)

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

  The number of subjects experiencing dose limiting toxicities including adverse events and serious adverse events will be assessed.

  Up to 5 years post Donor Cell Transplant (28 days dose limiting toxicity period)

• Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients

  To enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism

  Up to 24 weeks post Donor Cell Transplant

• Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients

  To enable immune reconstitution, as determined by the production of naive T cells

  Weeks 36-104 post Donor Cell Transplant

Study Arms (1)

Blood Stem Cell Transplant w/ anti-CD117 conditioning

EXPERIMENTAL

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.

Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)

Interventions

Humanized anti-CD117 Monoclonal Antibody (JSP191) BIOLOGICAL

Procedure: single intravenous infusion of JSP191 antibody

Blood Stem Cell Transplant w/ anti-CD117 conditioning

Eligibility Criteria

• Age: 3 Months - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but not limited to the following subtypes:
• T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient (no longer enrolling)
• T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
• 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.
• 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

You may not qualify if:

• Patients with any acute or uncontrolled infections
• Patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
• Patients with active malignancies
• Active GVHD within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD

Sponsors & Collaborators

• Jasper Therapeutics, Inc.: lead

Study Sites (9)

UCLA Mattel Children's Hospital

Los Angeles, California, 90095, United States

Location

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

Location

UCSF Benioff's Children's Hospital

San Francisco, California, 94158, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Immunologic Deficiency Syndromes

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Rajni A. Agarwal-Hashmi, M.D.

  Lucile Packard Children's Hospital

  PRINCIPAL INVESTIGATOR

• Christopher C. Dvorak, M.D.

  UCSF Benioff's Children's Hospital

  PRINCIPAL INVESTIGATOR

• Joseph H. Oved, M.D.

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

• Theodore B. Moore, M.D.

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

• Sharat Chandra, M.D.

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

• Christen L Ebens, M.D., MPH

  University of Minnesota

  PRINCIPAL INVESTIGATOR

• Harry L Malech, M.D.

  National Institutes of Health Clinical Center (CC)

  PRINCIPAL INVESTIGATOR

• Shanmuganathan Chandrakasan, M.D.

  Children's Healthcare of Atlanta

  PRINCIPAL INVESTIGATOR

• Neena Kapoor, M.D.

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

• Elizabeth D Hicks, M.D.

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

• Susan Prockop, M.D.

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2016
• First Posted: November 15, 2016
• Study Start: March 20, 2017
• Primary Completion: July 7, 2025
• Study Completion: July 7, 2025
• Last Updated: July 28, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)