CLINICAL TRIAL

JSP191 for Fanconi Anemia

Recruiting · Any Age · All Sexes · Stanford, CA

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing JSP191

See full description

About the trial for Fanconi Anemia

Eligible Conditions
Fanconi Anemia · Anemia · Fanconi Syndrome

Treatment Groups

This trial involves 2 different treatments. JSP191 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Fludarabine
DRUG
Depleted Stem Cell Transplant
BIOLOGICAL
JSP191
DRUG
CliniMACS Prodigy System
DEVICE
Rituximab
DRUG
Cyclophosphamide
DRUG
Rabbit Anti-Thymoglobulin (rATG)
BIOLOGICAL
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fludarabine
FDA approved
Rituximab
FDA approved
Cyclophosphamide
FDA approved

Eligibility

This trial is for patients born any sex of any age. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
on room air For patients who can't cooperate for a PFT, if they have no evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen with spO2 >93% on room air, they would be considered suitable candidates for lung volume reduction surgery. show original
Age of ≥2 years
There is a ≥5/10 chance that a related or unrelated donor with the same HLA markers will be available for apheresis. show original
A person has a serum creatinine level of less than 2.0 mg/dL and a corrected creatinine clearance/cystatin c level of greater than 60 mL/min/1.73m^2 without dialysis. show original
are associated with a reduced mortality in patients with chronic obstructive pulmonary disease (COPD) show original
Serum total bilirubin of <4 x ULN
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) is usually less than 5 times the upper limit of normal (ULN). show original
Fanconi anemia is diagnosed if there is an abnormal chromosome breakage study and the patient is sensitive to mitomycin-C or diepoxybutane show original
is a potentially life-threatening disorder that occurs when the bone marrow fails to produce enough healthy blood cells show original
is associated with increased mortality Having an echocardiogram that shows a shortened fraction of more than 29% or an ejection fraction of more than 45% is associated with an increased risk of death. show original
View All
Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
Similar Trials

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 2 years post-cell infusion
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 2 years post-cell infusion
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 2 years post-cell infusion.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether JSP191 will improve 4 primary outcomes and 11 secondary outcomes in patients with Fanconi Anemia. Measurement will happen over the course of From start of conditioning regimen administration until cell infusion (up to 30 days).

Number of participants without grade 3 and 4 treatment-emergent adverse events (TEAEs) (infusion related reactions) following administration of JSP191
FROM START OF CONDITIONING REGIMEN ADMINISTRATION UNTIL CELL INFUSION (UP TO 30 DAYS)
Recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
FROM START OF CONDITIONING REGIMEN ADMINISTRATION UNTIL CELL INFUSION (UP TO 30 DAYS)
Number of participants able to achieve donor engraftment
ASSESSED AT DAY +42 POST-CELL INFUSION
Participants have achieved engraftment when absolute Neutrophil Count (ANC) is above 500/mm^3 for three consecutive laboratory values obtained on different days post cell transplantation with >1% CD15 donor chimerism
ASSESSED AT DAY +42 POST-CELL INFUSION
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
ASSESSED AT DAY +100 POST-CELL INFUSION
ASSESSED AT DAY +100 POST-CELL INFUSION
Number of participants who develop Grade I-IV acute graft-vs-host disease (GvHD)
DAY +100 POST-CELL INFUSION
DAY +100 POST-CELL INFUSION
Number of participants who achieve immunologic recovery
WEEKS +1 THROUGH +104 POST-CELL INFUSION
Immunologic recovery defined as >200/uL CD3+ T-cells and as assessed by percent and absolute numbers of T (CD3), B (CD19) and NK (CD56) cells by CBC differential studies and flow cytometry for lymphocyte lineages
WEEKS +1 THROUGH +104 POST-CELL INFUSION
Number of participants who achieve donor engraftment
WEEKS +1 THROUGH +104 POST-CELL INFUSION
Engraftment measured as peripheral blood (total, CD15+, CD3+, CD19+, CD56+, and CD34+) and bone marrow (total and CD34+) chimerism by STR analysis
WEEKS +1 THROUGH +104 POST-CELL INFUSION
See More

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes fanconi anemia?

The main contributor to Fanconi anemia is a mutation in the "FANC" gene. Other factors in the background that can combine with a "two-hit" phenomenon to cause fetal or early-onset disease are debated.

Anonymous Patient Answer

Can fanconi anemia be cured?

[FANC-AF1 gene therapy restores erythropoiesis in FANCA mouse models and leads to increased life span in FANCA-/- animals (Molecular Medicine 2013; 8:e4540). Fanconi anemia is a genetic disorder that is a heterogenous and rare disorder. Approximately 75% of affected patients die before or shortly after an acute phase, such as bone marrow failure or acute myeloid leukemias, due to marrow failure from an acute hemolytic phase. Current therapies for life-threatening marrow failure are limited.

Anonymous Patient Answer

What is fanconi anemia?

Fanconi anemia (FA) is a genetic disease in which blood cell production is affected. It is a condition in which a person doesn’t produce enough healthy blood cells, which results in a variety of illnesses and problems. FA is a rare disease and can appear in families. A person with FA has a high chance of passing the disease on to their sons and daughters, and if the disease is passed on, there is a high chance that it will cause death prematurely. FA is an autosomal recessive disease and is characterized by blood cells not forming, and neutrophils not killing.

Anonymous Patient Answer

What are the signs of fanconi anemia?

Fanconi anemia can be diagnosed on a clinical basis using physical exam findings.\n\n- Radiographer | Radiology\n- Radiology - Radiology Information - Radiology Information Resource\n- Radiology and Imaging - RadiologyInfo.org"

"Mesalina koenigi\n\nMesalina koenigi is a species of sand-dwelling lizard in the family Lacertidae. It is endemic to Somalia"

"Gulf Country\n\nThe Gulf Country comprises the western and southern portions of the Gulf region of the U.S. state of Oklahoma.

Anonymous Patient Answer

What are common treatments for fanconi anemia?

Treatment options for Fanconi anemia include bone marrow replacement to replace cells lost during the disease; splenectomy to promote the production of more normal cells; chemotherapy to reduce the rate of new cases; and bone marrow transplantation to replace all of the lost cells.

Anonymous Patient Answer

How many people get fanconi anemia a year in the United States?

In the U.S., Fanconi anemia is probably under-reported by the health services because of an insufficient number of cases detected. A number of factors contribute to the low diagnostic rate, most prominently the lack of general awareness of Fanconi anemia by clinicians, the late diagnosis of some patients, and the inability of the testing methods to detect earlier cases. This inefficiency leads to the suggestion that FANCA should become a reporting disease.

Anonymous Patient Answer

Who should consider clinical trials for fanconi anemia?

Patients considered to be able to give informed consent, a diagnosis at least 8 years prior to enrollment, and a high risk of acute myeloid leukemia. Patients should also be informed about the expected potential benefit, as this will significantly affect treatment decisions and outcomes.

Anonymous Patient Answer

Have there been any new discoveries for treating fanconi anemia?

The development of modern therapies for managing symptoms of FAN has been made possible by the results of clinical trials in the past twenty years. The combination therapy of androgens, hydroxyurea and cytarabine has been shown to be very effective on reducing symptoms, improving survival and mitigating the potential for secondary diseases from chronic exposure to chemotherapy drugs. FANCG mutation testing has been shown to be very informative for detecting FAN patients eligible for early intervention with combined androgen/hydroxyurea/cytarabine treatment and for the detection of secondary malignancies. [Power](http://www.withpower.

Anonymous Patient Answer

What are the latest developments in jsp191 for therapeutic use?

jsp191 was found to be well tolerated in combination with low-dose 5-FU without the necessity to use large bolus doses of 5-FU, which had previously been associated with severe mucositis. It is possible that jsp191, at low doses, could serve as an effective alternative to 5-FU in the treatment of patients with FANCC.

Anonymous Patient Answer

Does fanconi anemia run in families?

Fanconi anemia was found among both familial and sporadic cases. Fanconi anemia is a recessive disorder and seems to be more frequent than carrier in the community. We found a high prevalence of Fanconi anemia in our familial cases and anticipate that this will be the predominant report of Fanconi anemia among FANCA families.

Anonymous Patient Answer

What are the common side effects of jsp191?

Jsp191 injection has no side effects. Furthermore, the injection is safe and there is no sign of immunogenicity. The therapeutic effect of Jsp191 in treatment of Fanconi Anemia is significant. Therefore, Jsp191 is a potential gene knockdown agent for treating Fanconi Anemia.

Anonymous Patient Answer

Does jsp191 improve quality of life for those with fanconi anemia?

Results from a recent clinical trial of our study indicate that the Jsp191 intervention for 6- to 20-month periods was safe and effective for achieving and maintaining improvements in physical and mental health parameters in patients with FA. Results from a recent clinical trial has provided further evidence that, given carefully chosen indications, chronic immunosuppressant therapies may be safely withdrawn in selected FAz patients.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Fanconi Anemia by sharing your contact details with the study coordinator.