Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab (NCT04784052)
TCRฮฑฮฒ+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 (Briquilimab) to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia
๐ฉบ Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Stanford University โ Stanford, California
Age range: 2 Years
๐งช Interventions in This Study
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria โ
๐ All Participating Trial Locations (1 Sites)
- Stanford University โ Stanford, California
๐ Contact the Study Coordinator
Phone: (650) 725-9250
Email: scgt_clinical_trials_office@lists.stanford.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Fanconi Anemia
- Eltrombopag for People With Fanconi Anemia โ Bethesda, Maryland
- Prenatal Transplantation for Fetuses With Fanconi Anemia โ San Francisco, California
- MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients โ Minneapolis, Minnesota
- HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy โ New York, New York
- Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders โ Minneapolis, Minnesota
Explore by condition: All Fanconi Anemia clinical trials
๐ก How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.