Clinical trial NCT02963064: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: SCID
Keywords: Immunodeficiency
Pediatric
SCID
Bone Marrow Transplantation
GVHD
Stem Cells
Chimerism
Transplant
BMT
Lead sponsor: Jasper Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2016-11-15
Enrollment: 40
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 exit from the bone marrow.

Eligibility criteria: Key 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

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

Primary outcomes:
measure: Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events
timeFrame: Up to 5 years post Donor Cell Transplant (28 days dose limiting toxicity period)
measure: Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
timeFrame: Up to 24 weeks post Donor Cell Transplant
measure: Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
timeFrame: Weeks 36-104 post Donor Cell Transplant
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Los Angeles
country: United States
facility: UCLA Mattel Children's Hospital
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital
state: California
city: San Francisco
country: United States
facility: UCSF Benioff's Children's Hospital
state: California
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Lucile Packard Children's Hospital
name: Rajni A. Agarwal-Hashmi, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: UCSF Benioff's Children's Hospital
name: Christopher C. Dvorak, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Memorial Sloan Kettering Cancer Center
name: Joseph H. Oved, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, Los Angeles
name: Theodore B. Moore, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Medical Center, Cincinnati
name: Sharat Chandra, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Minnesota
name: Christen L Ebens, M.D., MPH
role: PRINCIPAL_INVESTIGATOR
affiliation: National Institutes of Health Clinical Center (CC)
name: Harry L Malech, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Healthcare of Atlanta
name: Shanmuganathan Chandrakasan, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Los Angeles
name: Michael Pulsipher, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's National Research Institute
name: Elizabeth D Hicks, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital
name: Susan Prockop, M.D.
role: PRINCIPAL_INVESTIGATOR
References: