JSP191 Antibody Targeting Conditioning in SCID Patients

About

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

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 1/Phase 2

Eligibility

Gender

All

Healthy Volunteers

No

Minimum Age

3 Months

Maximum Age

12 Years

Key Inclusion Criteria:

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 (no longer enrolling)
- T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
- 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.
Patients with human leukocyte antigen (HLA) matched related or unrelated donors
Adequate end organ function as defined in study protocol
Age ≤ 12 years
Prior donor of appropriate age (≥ 5 years old) available for re-collection of stem cells
Previous allogeneic Hematopoietic Cell Transplantation HCT (≥ 6 months post initial transplant) with poor graft function

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

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