JSP191 for Severe Combined Immunodeficiency (SCID)

No longer recruiting at 11 trial locations

Overseen By Clinical Trials Jasper Therapeutics, Inc.

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Jasper Therapeutics, Inc.

Must not be taking: Investigational agents, Chemotherapy

Disqualifiers: Acute infections, Active malignancies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called JSP191 (also known as AMG 191) for individuals with Severe Combined Immune Deficiency (SCID), a condition where the immune system functions poorly. The goal is to determine if JSP191 is safe and effective when used before a blood stem cell transplant. The study includes two groups: one with individuals who have previously undergone a transplant but require additional support, and another with newly diagnosed patients. The trial seeks children 12 and under with SCID who have a matched donor available. As a Phase 1 trial, this research focuses on understanding how JSP191 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot participate if you are receiving other investigational agents, or concurrent biological, chemotherapy, or radiation therapy.

Is there any evidence suggesting that JSP191 is likely to be safe for humans?

Research has shown that JSP191 was tested in 14 patients with SCID, a serious immune system disorder, to assess its safety. The results indicated that JSP191 was generally well-tolerated by these patients. Early findings suggest it is a safe preparation for a stem cell transplant, as it helps ready the body without causing significant harm. Some studies highlight that JSP191 does not use harsh chemicals to remove old cells, offering a gentler option for patients. While these results are promising, further research is underway to confirm its safety. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for SCID?

Unlike the standard treatments for Severe Combined Immunodeficiency (SCID), which often involve traditional chemotherapy for conditioning, JSP191 uses a targeted anti-CD117 antibody approach. This innovative mechanism specifically targets and clears out faulty blood stem cells without the harsh side effects typically associated with chemotherapy. Researchers are excited because this method could potentially prepare patients for a stem cell transplant more safely and effectively, minimizing the risk of complications and enhancing overall patient outcomes.

What evidence suggests that JSP191 might be an effective treatment for SCID?

Research has shown that JSP191, which participants in this trial will receive, might help patients with Severe Combined Immunodeficiency (SCID). In a study with 14 SCID patients, JSP191 successfully prepared them for blood stem cell transplants. This treatment helped the new blood cells settle into the bone marrow, created a mix of donor and patient cells, and led to the production of new, healthy immune cells. Importantly, JSP191 was well tolerated and did not cause any treatment-related side effects. These early results are promising for improving outcomes for SCID patients. ^{1 2 3 6 7}

Who Is on the Research Team?

Christen Ebens, MD

Principal Investigator

University of Minnesota

Shanmuganathan Chandrakasan, MD

Principal Investigator

Children's Healthcare of Atlanta

Susan Prockop, MD

Principal Investigator

Boston Children's Hospital

Theodore Moore, MD

Principal Investigator

University of California, Los Angeles

Rajni Agarwal-Hashmi, M.D.

Principal Investigator

Lucile Packard Children's Hospital

Christopher C. Dvorak, M.D.

Principal Investigator

UCSF Benioff's Children's Hospital

Joseph H. Oved, M.D.

Principal Investigator

Memorial Sloan Kettering Cancer Center

Sharat Chandra, M.D.

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Harry Malech, MD

Principal Investigator

National Institutes of Health Clinical Center (CC)

Neena Kapoor, M.D.

Principal Investigator

Children's Hospital Los Angeles

Elizabeth D Hicks, M.D.

Principal Investigator

Children's National Research Institute

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have a donor who matches my HLA type.

My organs are functioning well enough for the study.

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

Exclusion Criteria

I do not have any current severe infections.

I have had graft-versus-host disease or been on treatment for it in the last 6 months.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants receive a single dose of intravenous JSP191 antibody followed by monitoring for antibody clearance

4 weeks

Transplantation

Participants receive stem cell transplant and are monitored for hematopoietic recovery

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

104 weeks

What Are the Treatments Tested in This Trial?

Interventions

• JSP191

Trial Overview 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.

How Is the Trial Designed?

Group I: Blood Stem Cell Transplant w/ anti-CD117 conditioning Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Jasper Therapeutics, Inc.

Lead Sponsor

Trials

7

Recruited

310+

Published Research Related to This Trial

Severe combined immunodeficiency (SCID) can be caused by mutations in at least seven different genes, with significant advances in understanding these causes since 1993.

Treatment options have improved dramatically, with over 95% success rates for related marrow transplants performed within the first 3.5 months of life, and recent successes in gene therapy for infants with X-linked SCID, highlighting the importance of early diagnosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advances in the understanding and treatment of human severe combined immunodeficiency. Buckley, RH.[2022]

The patient with B-cell-negative severe combined immunodeficiency (SCID) showed a complete absence of peripheral CD3(+) T cells and CD19(+) B cells, indicating a severe impairment in the immune system.

Analysis revealed that while common gamma chain expression was normal, there was a complete arrest in the development of B cells from pro-B to pre-B stages, suggesting that genes involved in V(D)J recombination, other than RAG, may contribute to the immunodeficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Complete arrest from pro- to pre-B cells in a case of B cell-negative severe combined immunodeficiency (SCID) without recombinase activating gene (RAG) mutations. Agematsu, K., Nagumo, H., Hokibara, S., et al.[2019]

In a study of 57 SCID patients from India, most presented within 6 months of age, with common symptoms including recurrent pneumonia (66%) and failure to thrive (60%), highlighting the severe impact of this immunodeficiency.

Hematopoietic Stem Cell Transplantation (HSCT) is the only curative treatment, but outcomes were poor in the four patients who underwent the procedure, indicating a need for improved therapeutic strategies and further research into genetic causes, with 32 novel variants identified in the cohort.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical, Immunological, and Molecular Findings in 57 Patients With Severe Combined Immunodeficiency (SCID) From India. Aluri, J., Desai, M., Gupta, M., et al.[2020]

In a study of 10 SCID patients with various JAK3 mutations, bone marrow transplantation effectively restored T-cell immunity, with all but one patient showing normal T-cell function after the procedure.

However, the restoration of B-cell and NK cell functions was less successful, as only 3 patients regained antibody function and only 2 showed normal NK activity post-transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Janus kinase 3 (JAK3) deficiency: clinical, immunologic, and molecular analyses of 10 patients and outcomes of stem cell transplantation. Roberts, JL., Lengi, A., Brown, SM., et al.[2022]

A 6-month-old girl with severe combined immunodeficiency (SCID) was diagnosed with compound heterozygous mutations in the JAK3 gene, which is linked to a severe form of immunodeficiency.

The patient successfully underwent bone marrow transplantation from a haploidentical donor, highlighting the potential for effective treatment in cases of JAK3-deficient SCID.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Novel compound heterozygous mutations in a Japanese girl with Janus kinase 3 deficiency. Sato, T., Okano, T., Tanaka-Kubota, M., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02963064

JSP191 Antibody Targeting Conditioning in SCID Patients 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 ...

2. ir.jaspertherapeutics.com ir.jaspertherapeutics.com/news-releases/news-release-details/jasper-therapeutics-announces-fda-fast-track-designation-jsp191

News Release Details To date, JSP191 has been studied in 14 SCID patients in an ongoing multicenter clinical trial with clinical outcome data presented at academic ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S0006497121025465

JSP191 As a Single-Agent Conditioning Regimen Results ... JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Naïve Lymphocytes in ...

4. ir.jaspertherapeutics.com ir.jaspertherapeutics.com/news-releases/news-release-details/jasper-therapeutics-present-data-jsp191-conditioning-scid

Jasper Therapeutics to Present Data on JSP191 ... JSP191 is well tolerated with no treatment-related adverse events in dose-escalation study; Single-agent conditioning with JSP191 is ...

5. alphaclinic.ucsf.edu alphaclinic.ucsf.edu/phase-12-study-evaluate-safety-tolerability-and-efficacy-jsp191-hematopoietic-cell-transplantation

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and ... The aim of this study is to evaluate the safety, tolerability and efficacy of JSP191 as a conditioning agent used in pediatric subjects undergoing hematopoietic ...

6. jaspertx.com jaspertx.com/wp-content/uploads/2022/04/2022-CIS-Poster-Final-Mar-21.pdf

Single-Agent Conditioning with Anti-CD117 Antibody ... Study endpoints include 1) safety and tolerability of JSP191, 2) donor chimerism of stringently flow-sorted CD15+ blood granulocytes, 3) lymphocyte ...

7. ashpublications.org ashpublications.org/blood/article/136/Supplement%201/10/471270/First-Report-of-Non-Genotoxic-Conditioning-with

First Report of Non-Genotoxic Conditioning with JSP191 (anti ... These first-in-class results provide proof of concept of the safety and efficacy of the use of JSP191 antibody to clear host marrow niche space ...