Is NCT04784052 recruiting in Stanford, CA?

Yes, NCT04784052 is currently recruiting participants at its Stanford, CA location. This fanconi anemia clinical trial is actively enrolling qualified participants.

How do I join the fanconi anemia clinical trial in Stanford?

To join this clinical trial in Stanford, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Stanford clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Fanconi Anemia Clinical Trial in Stanford, CA

About This Fanconi Anemia Study in Stanford

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.

Sponsor: Porteus, Matthew, MD

Who Can Participate

Inclusion 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 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

Exclusion Criteria

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

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Stanford?

Yes, this clinical trial (NCT04784052) has an active research site in Stanford, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Fanconi Anemia Treatment Options in Stanford, CA

If you're searching for fanconi anemia treatment options in Stanford, CA, this clinical trial (NCT04784052) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Stanford research site is actively enrolling participants for this clinical trial. You'll receive care from experienced fanconi anemia specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all fanconi anemia clinical trials near you to find additional studies recruiting in your area.

Fanconi Anemia Clinical Trial in Stanford, CA

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