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JSP191 Antibody Targeting Conditioning in SCID Patients

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ClinicalTrials.gov Identifier: NCT02963064
Recruitment Status : Recruiting
First Posted : November 15, 2016
Last Update Posted : June 1, 2021
Sponsor:
Information provided by (Responsible Party):
Jasper Therapeutics, Inc.

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

Condition or disease Intervention/treatment Phase
SCID Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191) Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : March 20, 2017
Estimated Primary Completion Date : August 2024
Estimated Study Completion Date : August 2027


Arm Intervention/treatment
Experimental: 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.
Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)
Procedure: single intravenous infusion of JSP191 antibody




Primary Outcome Measures :
  1. Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events [ Time Frame: Up to 5 years post Donor Cell Transplant (28 days dose limiting toxicity period) ]
    The number of subjects experiencing dose limiting toxicities including adverse events and serious adverse events will be assessed.

  2. Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients [ Time Frame: Up to 24 weeks post Donor Cell Transplant ]
    To enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism

  3. Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients [ Time Frame: Weeks 36-104 post Donor Cell Transplant ]
    To enable immune reconstitution, as determined by the production of naive T cells



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Ages Eligible for Study:   3 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02963064


Contacts
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Contact: Clinical Trials Jasper Therapeutics, Inc. 650-549-1270 ClinicalTrials@JasperTherapeutics.com

Locations
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United States, California
UCLA Mattel Children's Hospital Recruiting
Los Angeles, California, United States, 90095
Contact: Andres Vargas    310-871-0614    andresvargas@mednet.ucla.edu   
Principal Investigator: Theodore B. Moore, M.D.         
Lucile Packard Children's Hospital Recruiting
Palo Alto, California, United States, 94304
Contact: Elisabeth Merkel, RN    650-721-0644    merkel@stanford.edu   
Principal Investigator: Rajni A. Agarwal-Hashmi, M.D.         
UCSF Benioff's Children's Hospital Recruiting
San Francisco, California, United States, 94158
Contact: Kenny Truong    415-502-2080    kenny.truong@ucsf.edu   
Principal Investigator: Christopher C. Dvorak, M.D.         
United States, Georgia
Children's Healthcare of Atlanta Recruiting
Atlanta, Georgia, United States, 30322
Contact: Rebecca Hanrahan    404-785-9831    Rebecca.Hanrahan@choa.org   
Contact: Shanmuganathan Chandrakasan, M.D.    404-727-8877    shanmuganathan.chandrakasan@emory.edu   
United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: Pamela Graham, RN, BSN, MSA    301-761-6732    pamela.graham@nih.gov   
Principal Investigator: Harry L. Malech, M.D.         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Tamara Griffin       griffint@umn.edu   
Contact: Lauren Matzke, RN    612-624-5831    matzk042@umn.edu   
Principal Investigator: Christen L. Ebens, M.D., MPH         
United States, New York
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10065
Contact: Susan E. Prockop, MD    212-639-6715    prockops@mskcc.org   
Principal Investigator: Susan E. Prockop, M.D.         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Jamie Wilhelm    513-803-1102    jamie.wilhelm@cchmc.org   
Principal Investigator: Sharat Chandra, M.D.         
Sponsors and Collaborators
Jasper Therapeutics, Inc.
Investigators
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Principal Investigator: 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: Susan E. Prockop, M.D. Memorial Sloan Kettering Cancer Center
Principal Investigator: Theodore B. Moore, M.D. UCLA Mattel Children's Hospital
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
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Responsible Party: Jasper Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02963064    
Other Study ID Numbers: JAS-BMT-CP-001
First Posted: November 15, 2016    Key Record Dates
Last Update Posted: June 1, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Data collected is for future publication

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Jasper Therapeutics, Inc.:
Immunodeficiency
Pediatric
SCID
Bone Marrow Transplantation
GVHD
Stem Cells
Chimerism
Transplant
BMT
Additional relevant MeSH terms:
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Severe Combined Immunodeficiency
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Antibodies
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs