JSP191 Antibody Targeting Conditioning in SCID Patients

SCID Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02963064
• Other study ID #: JAS-BMT-CP-001
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: March 20, 2017
• Est. completion date: August 2027

Study information

• Verified date: February 2024
• Source: Jasper Therapeutics, Inc.
• Contact: Clinical Trials Jasper Therapeutics, Inc.
• Phone: 650-549-1270
• Email: ClinicalTrials[@]JasperTherapeutics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: August 2027
• Est. primary completion date: August 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Months and older

Eligibility

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-2Rc? deficient, JAK3-deficient

2. T-, B-, NK+: RAG1/2 deficient, Artemis-deficient

3. T-, B+, NK+: IL7Ra 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

Related Conditions & MeSH terms

• SCID
• Severe Combined Immunodeficiency

Intervention

Biological:
• Humanized anti-CD117 Monoclonal Antibody (JSP191)
Procedure: single intravenous infusion of JSP191 antibody

Locations

Country	Name	City	State
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	National Institutes of Health Clinical Center	Bethesda	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	UCLA Mattel Children's Hospital	Los Angeles	California
United States	University of Minnesota	Minneapolis	Minnesota
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	Lucile Packard Children's Hospital	Palo Alto	California
United States	UCSF Benioff's Children's Hospital	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Jasper Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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
Primary	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