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

Recruiting now · Phase 1/Phase 2

Conditions studied: Fanconi Anemia

In brief

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). Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.

Key facts

Study ID: NCT04784052
Run by: Porteus, Matthew, MD
People needed: 18
Starts: 2021-12-07
Expected to finish: 2028-12-01
Last updated by the study team: 2026-01-27

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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 neutrophil count (ANC) of <1000/mm)
Age of ≥2 years
Consenting ≥5/10 HLA-matched related or unrelated donor available for apheresis
Organ function defined as:
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)
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 Accountability Act

You may not qualify 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
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

Where it is running

Stanford University — Stanford, California, United States (enrolling)

Full record on ClinicalTrials.gov

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