RECRUITING

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

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Fanconi AnemiaCell TransplantsGraftsStem Cells

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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 after 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.

Official Title

TCRαβ+ 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

Quick Facts

Study Start:2021-12-07
Study Completion:2028-04
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04784052

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. 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
  2. 2. 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)
  3. 3. Age of ≥2 years
  4. 4. Consenting ≥5/10 HLA-matched related or unrelated donor available for apheresis
  5. 5. Organ function defined as:
  6. 1. Serum Creatinine \<2.0 mg/dL and corrected creatinine clearance/cystatin cL \>60 mL/min/1.73m\^2 without dialysis
  7. 2. 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)
  8. 3. 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%
  9. 4. Shortening fraction of ≥29% or ejection fraction of ≥45% by echocardiogram
  10. 5. Serum total bilirubin of \<4 x ULN
  11. 6. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \< 5 x ULN
  12. 7. Prothrombin time international normalized ratio (PT INR) and partial thromboplastin time (PTT) \<1.5 x ULN
  13. 6. Life expectancy of at least 2 years
  14. 7. Patients of childbearing potential must be willing to use an effective contraceptive method for the duration of the peri-transplant conditioning through hematopoietic recovery
  15. 8. 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
  1. 1. Patients with available and consenting 10/10 HLA-identical sibling donor for apheresis
  2. 2. Patients with any acute or uncontrolled infections at the time of enrollment, including bacterial, fungal or viral
  3. 3. Patients who are seropositive for HIV-I/II or HTLV-I/II.
  4. 4. Patients receiving any other investigational agents or other biological, chemotherapy, or radiation therapy within 14 days of enrollment
  5. 5. Patients with any active malignancies, myelodysplastic syndrome or other concerns for high-risk bone marrow disease
  6. 6. Patients who received androgens in last 3 months
  7. 7. Pregnant or lactating women
  8. 8. Women who are nursing and do not wish to discontinue breastfeeding
  9. 9. Lansky/Karnofsky performance score \<50%.
  10. 10. 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
  11. 11. Patients who, in the opinion of the Principal Investigator, may not be able to comply with the safety monitoring requirements of the study

Contacts and Locations

Principal Investigator

Rajni Agarwal, MD
PRINCIPAL_INVESTIGATOR
Stanford University

Study Locations (Sites)

Stanford University
Stanford, California, 94305
United States

Collaborators and Investigators

Sponsor: Porteus, Matthew, MD

  • Rajni Agarwal, MD, PRINCIPAL_INVESTIGATOR, Stanford University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-12-07
Study Completion Date2028-04

Study Record Updates

Study Start Date2021-12-07
Study Completion Date2028-04

Terms related to this study

Keywords Provided by Researchers

  • Cell Transplants
  • Grafts
  • Stem Cells

Additional Relevant MeSH Terms

  • Fanconi Anemia