Back to resultsJSP191 Antibody Targeting Conditioning in SCID Patients
Primary Purpose
SCID
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Humanized anti-CD117 Monoclonal Antibody (JSP191)
Sponsored by
About this trial
This is an interventional treatment trial for SCID focused on measuring Immunodeficiency, Pediatric, SCID, Bone Marrow Transplantation, GVHD, Stem Cells, Chimerism, Transplant, BMT
Eligibility Criteria
Key Inclusion Criteria:
All patient groups must have:
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
- T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
- 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.
- Patients with human leukocyte antigen (HLA) matched related or unrelated donors
- Adequate end organ function as defined in study protocol
Key Exclusion Criteria:
- 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
Sites / Locations
- UCLA Mattel Children's HospitalRecruiting
- Lucile Packard Children's HospitalRecruiting
- UCSF Benioff's Children's HospitalRecruiting
- Children's Healthcare of AtlantaRecruiting
- National Institutes of Health Clinical CenterRecruiting
- University of MinnesotaRecruiting
- Memorial Sloan Kettering Cancer CenterRecruiting
- Cincinnati Children's Hospital Medical CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Blood Stem Cell Transplant w/ anti-CD117 conditioning
Arm 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.
Outcomes
Primary Outcome Measures
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.
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
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
To enable immune reconstitution, as determined by the production of naive T cells
Secondary Outcome Measures
Full Information
NCT ID
NCT02963064
First Posted
November 2, 2016
Last Updated
November 16, 2021
Sponsor
Jasper Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT02963064
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2021
Overall Recruitment Status
Recruiting
Study Start Date
March 20, 2017 (Actual)
Primary Completion Date
August 2024 (Anticipated)
Study Completion Date
August 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jasper Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
SCID
Keywords
Immunodeficiency, Pediatric, SCID, Bone Marrow Transplantation, GVHD, Stem Cells, Chimerism, Transplant, BMT
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Blood Stem Cell Transplant w/ anti-CD117 conditioning
Arm Type
Experimental
Arm 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.
Intervention Type
Biological
Intervention Name(s)
Humanized anti-CD117 Monoclonal Antibody (JSP191)
Intervention Description
Procedure: single intravenous infusion of JSP191 antibody
Primary Outcome Measure Information:
Title
Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events
Description
The number of subjects experiencing dose limiting toxicities including adverse events and serious adverse events will be assessed.
Time Frame
Up to 5 years post Donor Cell Transplant (28 days dose limiting toxicity period)
Title
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
Description
To enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism
Time Frame
Up to 24 weeks post Donor Cell Transplant
Title
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
Description
To enable immune reconstitution, as determined by the production of naive T cells
Time Frame
Weeks 36-104 post Donor Cell Transplant
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
All patient groups must have:
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
T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
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.
Patients with human leukocyte antigen (HLA) matched related or unrelated donors
Adequate end organ function as defined in study protocol
Key Exclusion Criteria:
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Clinical Trials Jasper Therapeutics, Inc.
Phone
650-549-1270
Email
ClinicalTrials@JasperTherapeutics.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rajni A. Agarwal-Hashmi, M.D.
Organizational Affiliation
Lucile Packard Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Christopher C. Dvorak, M.D.
Organizational Affiliation
UCSF Benioff's Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Joseph H. Oved, M.D.
Organizational Affiliation
Memorial Sloan Kettering Cancer Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Theodore B. Moore, M.D.
Organizational Affiliation
UCLA Mattel Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Sharat Chandra, M.D.
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Christen L Ebens, M.D., MPH
Organizational Affiliation
University of Minnesota
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Harry L Malech, M.D.
Organizational Affiliation
National Institutes of Health Clinical Center (CC)
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Shanmuganathan Chandrakasan, M.D.
Organizational Affiliation
Children's Healthcare of Atlanta
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCLA Mattel Children's Hospital
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Andres Vargas
Phone
310-871-0614
Email
andresvargas@mednet.ucla.edu
First Name & Middle Initial & Last Name & Degree
Theodore B. Moore, M.D.
Facility Name
Lucile Packard Children's Hospital
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elisabeth Merkel, RN
Phone
650-721-0644
Email
merkel@stanford.edu
First Name & Middle Initial & Last Name & Degree
Rajni A. Agarwal-Hashmi, M.D.
Facility Name
UCSF Benioff's Children's Hospital
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kenny Truong
Phone
415-502-2080
Email
kenny.truong@ucsf.edu
First Name & Middle Initial & Last Name & Degree
Christopher C. Dvorak, M.D.
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rebecca Hanrahan
Phone
404-785-9831
Email
Rebecca.Hanrahan@choa.org
First Name & Middle Initial & Last Name & Degree
Shanmuganathan Chandrakasan, M.D.
Phone
404-727-8877
Email
shanmuganathan.chandrakasan@emory.edu
Facility Name
National Institutes of Health Clinical Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pamela Graham, RN, BSN, MSA
Phone
301-761-6732
Email
pamela.graham@nih.gov
First Name & Middle Initial & Last Name & Degree
Harry L. Malech, M.D.
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tamara Griffin
Email
griffint@umn.edu
First Name & Middle Initial & Last Name & Degree
Lauren Matzke, RN
Phone
612-624-5831
Email
matzk042@umn.edu
First Name & Middle Initial & Last Name & Degree
Christen L. Ebens, M.D., MPH
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Georgia Flynn
Email
flynng@mskcc.org
First Name & Middle Initial & Last Name & Degree
Joseph H. Oved, M.D.
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jamie Wilhelm
Phone
513-803-1102
Email
jamie.wilhelm@cchmc.org
First Name & Middle Initial & Last Name & Degree
Sharat Chandra, M.D.
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
Data collected is for future publication
Learn more about this trial
JSP191 Antibody Targeting Conditioning in SCID Patients
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