An observational study in Leukemia, Not Otherwise Specified and Leukemia, Other, sponsored by Wake Forest University Health Sciences. Recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-07-13.
Sponsored by Wake Forest University Health Sciences · Observational
This study will examine the influence of donor and recipient pharmacogenetics (PG), drug pharmacokinetics (PK), and T cell phenotypes and how it may permit a tailored dosing strategy to improve the therapeutic index of post-transplant cyclophosphamide (PTCy) and optimize the graft versus tumor effect, while minimizing acute and chronic graft versus host disease (GVHD).
The primary objective of this single-arm, pilot study is to determine whether pharmacogenetics (PG) of Cy-related candidate genes from the recipients and/or donors (haploidentical and matched related donor HCTs only) germ-line DNA is associated with incidence and severity of acute and chronic GVHD. Secondary objectives include determining whether pharmacogenetics (PG) of Cy-related candidate genes from the recipients and/or donors (haploidentical and matched related donor HCTs only) germ-line DNA is associated with Cy (and metabolites) exposure and toxicities; quantifying Cy (and related metabolites) exposure measured as the area under the concentration time curve (AUC) from zero to 24 hours both before (day -6) and after transplant (day +3), and correlate exposure with incidence of acute and chronic GVHD, and Adverse Events of Special Interest (AESIs); and determining whether immune activation or polarization prior to or following Cy GVHD prophylaxis is associated with grade of acute or chronic GVHD grade and AESIs. Safety objects include evaluating Cy administered, adverse events of special interest (including deaths while on study therapy), selected laboratory parameters (including time to neutrophil recovery), and immunosuppressant concomitant medications administration. Initially, 20 participants (HCT recipients and their respective haploidentical or matched related donors) will be enrolled with a subsequent 100 additional subjects enrolled.
5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.
This study's planned enrollment of 120 is close to the median of 120 across 744 observational studies indexed under Leukemia.
Browse Leukemia studies →Wake Forest University Health Sciences is the lead sponsor of 1,320 studies on the registry; 199 are open to participants now.
Of its 323 completed or terminated interventional studies of FDA-regulated products, 243 (75%) have results posted.
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Subjects who are scheduled as a recipient or respective donor for the following hematopoietic stem cell transplants (HCT): haplo-identical donor HCT, match related donor (MRD) HCT, matched unrelated donor (MUD) HCT.
Recipients and donors must meet all of the following applicable inclusion criteria to participate in this study:
Exclusion Criteria
Subjects meeting any of the criteria below may not participate in the study:
Cyclophosphamide
Drug: Cyclophosphamide · Other: Specimen collection
Specimen collection
Other: Specimen collection
Pharmacogenomics of candidate genes and pharmacokinetic analyses of cyclophosphamide administered as part of a reduced intensity conditioning (RIC) regimen and as post-transplant GVHD prophylaxis will be examined.
Buccal swabs will be obtained from donors for pharmacogenomics.
Comparison of Cy cMax Values
Evaluation and comparison of average Day 3 Cy cMax values between subjects who experience acute GVHD versus subjects who do not experience acute GVHD
Time frame: Approx. 24 mos
Incidence of chronic GVHD
Calculated for each subject as a binary variable indicating whether or not subject experienced chronic GVHD
Time frame: Approx. 24 mos
Cy exposure
Cy (and related metabolites) exposure will be calculated using area under the concentration curve (AUC). This will be accomplished using the trapezoidal approximation, and will be calculated over the 24 hour period following first pre-transplant dose of Cy and over the 24 hour period following post-transplant dose of Cy.
Time frame: Approx. 10 days
Toxicities
The incidence of adverse events of special interests will be collected according to CTCAE version 4.03
Time frame: Approx. 180 days
Pharmacogenetics of Cy-related genes
Evaluation of the prognostic value of pharmacogenetics (PG) of Cy-related candidate genes from the recipient's germ-line DNA, using logistic regression, on the incidence and severity of acute and chronic GVHD. PG of Cy-related candidate genes will be performed on 12 genes. Genes encoding CYP enzymes will be classified into metabolizer status and other genes will be classified as either wild type, heterozygous, or homozygous variants.
Time frame: Approx. 24 mos
Plan to share: No
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Wake Forest University Health Sciences