What is the mechanism of action of this treatment?

The most common treatments for Severe Combined Immunodeficiency (SCID) include hematopoietic stem cell transplantation (HSCT) and gene therapy. The antibody conditioning regimen, such as JSP191, works by targeting and depleting the patient's existing immune cells to create space for the new, healthy stem cells to engraft and proliferate. This is crucial for SCID patients because their immune systems are severely compromised, making them highly susceptible to infections. By effectively conditioning the patient, the new stem cells can develop into functional immune cells, thereby restoring the immune system and improving the patient's ability to fight infections.

What side effects are associated with this type of intervention?

Common treatments for Severe Combined Immunodeficiency (SCID) include antibody-based conditioning regimens like JSP191, which are generally used to prepare patients for blood stem cell transplantation. Known side effects of such antibody treatments can include infusion reactions, infections due to immunosuppression, and potential organ toxicity. Other common treatments for SCID, such as hematopoietic stem cell transplantation, may also lead to graft-versus-host disease, increased risk of infections, and organ damage. It is crucial to monitor patients closely for these adverse effects to manage them promptly.

What prior FDA approvals exist?

The FDA has approved several treatments for Severe Combined Immunodeficiency (SCID), primarily focusing on gene therapy and enzyme replacement therapy. For example, Strimvelis, a gene therapy for ADA-SCID, and enzyme replacement therapies like PEG-ADA have been approved. While JSP191 is an antibody conditioning regimen currently under study, similar antibody-based treatments for SCID have not been explicitly mentioned in the provided context. Therefore, the focus remains on gene and enzyme replacement therapies as the primary FDA-approved treatments for SCID.

What other types of research exist?

Promising, novel alternatives to JSP191 for SCID patients may include other targeted antibody therapies such as alemtuzumab, which has been used in conditioning regimens for other hematologic conditions. Additionally, gene therapy approaches and newer monoclonal antibodies targeting different immune pathways are being actively researched and could offer viable alternatives in the near future. Consulting with a healthcare provider specializing in SCID is essential to explore the most current and suitable treatment options.

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Monoclonal Antibodies

JSP191 for Severe Combined Immunodeficiency (SCID)

Phase 1 & 2

Recruiting

Led By Susan Prockop, M.D.

Research Sponsored by Jasper Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with human leukocyte antigen (HLA) matched related or unrelated donors

Patients with Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including specified subtypes

Must not have

Patients with any acute or uncontrolled infections

Patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years post donor cell transplant (28 days dose limiting toxicity period)

Awards & highlights

No Placebo-Only Group

Summary

This trial tests JSP191, an antibody, in SCID patients needing a blood stem cell transplant. JSP191 helps by clearing out old blood-forming cells to make room for new, healthy ones.

Who is the study for?

This trial is for patients with Severe Combined Immunodeficiency (SCID) who have a matching donor for blood stem cell transplantation. They must have normal organ function and typical SCID as defined by specific criteria. People with active cancer, ongoing treatments like chemotherapy or radiation, uncontrolled infections, or recent graft-versus-host disease are not eligible.

What is being tested?

The study is testing the safety and effectiveness of JSP191, a humanized anti-CD117 monoclonal antibody used as part of the conditioning regimen before blood stem cell transplants in SCID patients. This Phase 1/2 trial will assess how well patients tolerate this new treatment approach.

What are the potential side effects?

While specific side effects of JSP191 are not detailed here, monoclonal antibodies can cause allergic reactions, infusion-related symptoms such as fever or chills, potential organ inflammation, and may affect immune system responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a donor who matches my HLA type.

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I have been diagnosed with a specific type of severe combined immunodeficiency.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any current severe infections.

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I am not currently on any experimental treatments or undergoing chemotherapy or radiation.

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I currently have an active cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years post donor cell transplant (28 days dose limiting toxicity period)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years post donor cell transplant (28 days dose limiting toxicity period)

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years post donor cell transplant (28 days dose limiting toxicity period) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events

Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Blood Stem Cell Transplant w/ anti-CD117 conditioningExperimental Treatment1 Intervention

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.

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Severe Combined Immunodeficiency (SCID) include hematopoietic stem cell transplantation (HSCT) and gene therapy. The antibody conditioning regimen, such as JSP191, works by targeting and depleting the patient's existing immune cells to create space for the new, healthy stem cells to engraft and proliferate. This is crucial for SCID patients because their immune systems are severely compromised, making them highly susceptible to infections. By effectively conditioning the patient, the new stem cells can develop into functional immune cells, thereby restoring the immune system and improving the patient's ability to fight infections.

Neonatal combination therapy improves some of the clinical manifestations in the Mucopolysaccharidosis type I murine model.