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Stem Cell Transplant

Stem Cell Transplant + JSP191 for Fanconi Anemia

Phase 1 & 2

Recruiting

Led By Rajni Agarwal, MD

Research Sponsored by Porteus, Matthew, MD

Eligibility Criteria Checklist

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

Must have

Organ function criteria: Serum Creatinine <2.0 mg/dL and corrected creatinine clearance/cystatin cL >60 mL/min/1.73m^2 without dialysis, Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and diffusing capacity of the lung for carbon monoxide (DLCO) corrected for hemoglobin and volume, >50% predicted by pulmonary function tests (PFTs), Shortening fraction of ≥29% or ejection fraction of ≥45% by echocardiogram, Serum total bilirubin of <4 x ULN, Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) < 5 x ULN, Prothrombin time international normalized ratio (PT INR) and partial thromboplastin time (PTT) <1.5 x ULN, Life expectancy of at least 2 years, Patients of childbearing potential must be willing to use an effective contraceptive method for the duration of the peri-transplant conditioning through hematopoietic recovery, Patients and/or parents or legal guardians must be able to provide written informed consent and authorize use and disclosure of personal health information in accordance with Health Insurance Portability and Accountability Act

Patients must have ≥5/10 HLA-matched related or unrelated donor available for apheresis

Must not have

Patients with any active malignancies, myelodysplastic syndrome or other concerns for high-risk bone marrow disease

Lansky/Karnofsky performance score <50%

Timeline

Screening 3 weeks

Treatment Varies

Follow Up prior to start of ratg infusion; 15 minutes after first, second, and third ratg infusion; day of cell infusion; and week +1, week +2 and week +12 post cell infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing an experimental cell therapy for Fanconi Anemia which may enable enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor after using an experimental treatment called JSP-191 as a part of conditioning.

Who is the study for?

This trial is for children and adults with Fanconi Anemia, confirmed by specific tests and genetic mutations. Participants must be over 2 years old, have certain organ function levels (kidney, lung, heart), not be pregnant or breastfeeding, willing to use contraception if of childbearing potential, and have a life expectancy of at least 2 years. They should not have active cancers or uncontrolled infections.

What is being tested?

The trial is testing a new cell therapy using depleted stem cells from donors after conditioning with JSP191 antibody treatment. This aims to rebuild the patient's blood and immune systems with less toxicity than chemotherapy. Patients will undergo this regimen before receiving the stem cell infusion and will be monitored for up to two years.

What are the potential side effects?

Potential side effects may include reactions related to the immune system due to JSP191 antibody treatment, complications from stem cell transplant like infection risks or graft-versus-host disease (where transplanted cells attack the body), as well as typical chemotherapy-related issues such as nausea or hair loss.

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 at least half of my HLA markers for a cell donation.

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I have Fanconi Anemia confirmed by specific genetic tests.

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I am at least 2 years old.

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 active cancers, myelodysplastic syndrome, or high-risk bone marrow diseases.

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I need considerable assistance and medical care.

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I have not taken androgens in the last 3 months.

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I do not have any current infections that are not under control.

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I have a sibling donor who matches me perfectly for the donation and agrees to donate.

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I am breastfeeding and do not want to stop.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ prior to start of ratg infusion; 15 minutes after first, second, and third ratg infusion; day of cell infusion; and week +1, week +2 and week +12 post cell infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~prior to start of ratg infusion; 15 minutes after first, second, and third ratg infusion; day of cell infusion; and week +1, week +2 and week +12 post cell infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and prior to start of ratg infusion; 15 minutes after first, second, and third ratg infusion; day of cell infusion; and week +1, week +2 and week +12 post cell infusion for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of participants able to achieve donor engraftment

Number of participants without grade 3 and 4 treatment-emergent adverse events (TEAEs) (infusion related reactions) following administration of JSP191

Secondary study objectives

Number of participants who achieve disease-free survival

Number of participants who achieve donor engraftment

Number of participants who achieve hematopoietic recovery

+6 more

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: Depleted Stem Cell Transplant with JSP-191 ConditioningExperimental Treatment7 Interventions

Participants will receive an infusion of donor stem cells which have been depleted of αβ+T cells using the CliniMACS System device. Before the stem cell transplant, they will receive a reduced-intensity preparative regimen containing JSP191 in combination with rATG, cyclophosphamide, fludarabine and rituximab.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

2010

Completed Phase 4

~2310

Fludarabine

2012

Completed Phase 4

~1860