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JSP191 Antibody Targeting Conditioning in SCID Patients

J

Jasper Therapeutics

Status and phase

Enrolling
Phase 2
Phase 1

Conditions

SCID

Treatments

Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)

Study type

Interventional

Funder types

Industry

Identifiers

NCT02963064
JAS-BMT-CP-001

Details and patient eligibility

About

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

Full 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.

Enrollment

40 estimated patients

Sex

All

Ages

3 months to 12 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

40 participants in 1 patient group

Blood Stem Cell Transplant w/ anti-CD117 conditioning
Experimental group
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.
Treatment:
Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)

Trial contacts and locations

12

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Central trial contact

Clinical Trials Jasper Therapeutics, Inc.

Data sourced from clinicaltrials.gov

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