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New treatment option for Fanconi Anemia
Official title Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
ClinicalTrials.gov ID: NCT04784052
What this study is testing
What is JSP191?
JSP191 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fanconi anemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method).
- Phase 2: a mid-size study of how well it works
- Time commitment: about 2 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- All patients must have:
- Fanconi Anemia diagnosis as demonstrated by abnormal chromosome breakage studies with increased sensitivity to mitomycin-C (MMC) or diepoxybutane...
- Bone marrow failure (defined by reduction in at least one cell line on two separate occasions at least one month apart (e.g., platelet count of...
- Age of ≥2 years
- Consenting ≥5/10 HLA-matched related or unrelated donor available for apheresis
You likely can't join if
- Patients with available and consenting 10/10 HLA-identical sibling donor for apheresis
- Patients with any acute or uncontrolled infections at the time of enrollment, including bacterial, fungal or viral
- Patients who are seropositive for HIV-I/II or HTLV-I/II.
- Patients receiving any other investigational agents or other biological, chemotherapy, or radiation therapy within 14 days of enrollment
- Patients with any active malignancies, myelodysplastic syndrome or other concerns for high-risk bone marrow disease
- Patients who received androgens in last 3 months
See the full eligibility criteria
- All patients must have:
- Fanconi Anemia diagnosis as demonstrated by abnormal chromosome breakage studies with increased sensitivity to mitomycin-C (MMC) or diepoxybutane (DEB) and at least one mutation in a known Fanconi-associated gene
- Bone marrow failure (defined by reduction in at least one cell line on two separate occasions at least one month apart (e.g., platelet count of \<100,000 per cubic millimeter, hemoglobin \<9 gm/dl and/or absolute...
- Age of ≥2 years
- Consenting ≥5/10 HLA-matched related or unrelated donor available for apheresis
- Organ function defined as:
- Serum Creatinine \ 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...
- For patients unable to cooperate for PFTs, criteria are no evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen with spO2 \>93%
- 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...
- Patients with available and consenting 10/10 HLA-identical sibling donor for apheresis
- Patients with any acute or uncontrolled infections at the time of enrollment, including bacterial, fungal or viral
- Patients who are seropositive for HIV-I/II or HTLV-I/II.
- Patients receiving any other investigational agents or other biological, chemotherapy, or radiation therapy within 14 days of enrollment
- Patients with any active malignancies, myelodysplastic syndrome or other concerns for high-risk bone marrow disease
- Patients who received androgens in last 3 months
- Pregnant or lactating women
- Women who are nursing and do not wish to discontinue breastfeeding
- Lansky/Karnofsky performance score \<50%.
- Any other medical condition or history that, in the opinion of the Principal Investigator, could pose a significant safety risk to the participant or jeopardize the integrity of the study
- Patients who, in the opinion of the Principal Investigator, may not be able to comply with the safety monitoring requirements of the study
The study team makes the final eligibility decision.
Where it's taking place
- Stanford, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Stanford, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.