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Stem Cell Transplant
Stem Cell Transplant + JSP191 for Fanconi Anemia
Phase 1 & 2
Recruiting
Led By Agnieszka Czechowicz, MD, PhD
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years post-cell infusion
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
I have a donor who matches at least half of my HLA markers for a cell donation.
Select...
I have Fanconi Anemia confirmed by specific genetic tests.
Select...
I am at least 2 years old.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 2 years post-cell infusion
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years post-cell infusion
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of participants able to achieve donor engraftment
Number of participants who are able to have donor engraftment persist at the same rate or better compared to alternative hematopoietic cell transplant regimens for this patient population
Number of participants without grade 3 and 4 treatment-emergent adverse events (TEAEs) (infusion related reactions) following administration of JSP191
+1 moreSecondary outcome measures
Number of participants who achieve disease-free survival
Number of participants who achieve donor engraftment
Number of participants who achieve hematopoietic recovery
+8 moreTrial 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
1995
Completed Phase 3
~3770
Rituximab
1999
Completed Phase 4
~1880
Fludarabine
2012
Completed Phase 3
~1100
Find a Location
Who is running the clinical trial?
Porteus, Matthew, MDLead Sponsor
1 Previous Clinical Trials
22 Total Patients Enrolled
Maria Grazia RoncaroloLead Sponsor
Rajni AgarwalLead Sponsor
1 Previous Clinical Trials
3 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I do not have any active cancers, myelodysplastic syndrome, or high-risk bone marrow diseases.I need considerable assistance and medical care.I haven't had any experimental treatments or other cancer therapies in the last 14 days.My blood tests show low counts in at least one type of blood cell on two different tests a month apart.I have not taken androgens in the last 3 months.I have a donor who matches at least half of my HLA markers for a cell donation.You have tested positive for HIV or HTLV.I have Fanconi Anemia confirmed by specific genetic tests.I do not have any current infections that are not under control.I have a sibling donor who matches me perfectly for the donation and agrees to donate.I am at least 2 years old.I am breastfeeding and do not want to stop.
Research Study Groups:
This trial has the following groups:- Group 1: Depleted Stem Cell Transplant with JSP-191 Conditioning
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Have there been any prior investigations involving JSP191?
"Currently, there are 1169 trials researching JSP191. Of these active clinical studies, 224 have entered into the final Phase 3 stage of development. Primarily based in Philadelphia, Pennsylvania, 36803 locations across the world are participating in research for this treatment."
Answered by AI
To what ailments is JSP191 typically prescribed?
"JSP191 is generally utilized as a therapy for dlbcl, but can also be beneficial in treating lung cancer, immunosuppressive treatment and multiple sclerosis."
Answered by AI
Is enrollment still open for this experiment?
"According to clinicaltrials.gov, the recruitment process for this medical trial is still ongoing since its initial announcement on December 7th 2021 and last edit on February 2nd 2022."
Answered by AI
How many participants are engaged in the current clinical experiment?
"Affirmative. Clinicaltrials.gov indicates that this research is actively enlisting participants, with the initial post dated December 7th 2021 and most recent edit being February 2nd 2022. The trial requires 12 individuals from one medical site to partake in the study."
Answered by AI
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