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
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
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Jasper Therapeutics, Inc. |
United States,
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 |
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