Neoplasms

Blood and Lymph Conditions

All Conditions

Immune System Diseases

Rare Diseases

Leukemia

Graft vs Host Disease

Neoplasms by Histologic Type

Hematologic Diseases

Homologous Wasting Disease

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Cyclophosphamide

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

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.

Specimen collection

Elizabeth Youngblade

Aleksander Chojecki, MD

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.

Factors Affecting Post-transplant Cyclophosphamide (PTCy) Efficacy

Elucidation of Factors Predicting Efficacy and Toxicity of Post Transplantation Cyclophosphamide (PTCy) as a Strategy for Graft Versus Host Disease Prevention in Haploidentical, Matched Related Donor and Matched Unrelated Donor Peripheral Blood Hematopoietic Cell Transplantation

Evaluation and comparison of average Day 3 Cy cMax values between subjects who experience acute GVHD versus subjects who do not experience acute GVHD

Calculated for each subject as a binary variable indicating whether or not subject experienced chronic GVHD

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.

The incidence of adverse events of special interests will be collected according to CTCAE version 4.03

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.

Atrium Health Levine Cancer Institute

Wake Forest University Health Sciences

Heart and Blood Diseases

Multiple Myeloma

Neoplasms, Plasma Cell

Patients will view the 23-minute "Guide to Stem Cell Transplantation" video depicting the HSCT experience

Patients will receive HSCT FAQ sheet developed by the NCI at the NIH

Video

FAQ Sheet

Robin K Matsuyama, PhD

Patients in the intervention arm will view the 23-minute video depicting the Hematopoietic stem cell transplantation (HSCT) experience. Patients in the control arm will receive HSCT frequently asked questions (FAQ) sheet developed by the National Cancer Institute (NCI) at the National Institutes of Health (NIH).

The study's overall goals are 1) to improve outcomes; and 2) to increase satisfaction with the physician consultation.

Patients will be randomized to intervention or control and data will be collected at three time points (before treatment, 30 days post-HSCT and 12 months post-HSCT).

Efficacy of a Patient Education Video

Data will be collected through review of medical and insurance billing records

The Physician consultation will be measured by the Patient Satisfaction Communication Questionnaire (PSCQA). Participants are asked to rate their experience with the physician consultation. There are 17 items, 8 positively worded and 9 negatively worded. Each item is rated on a 5 point likert scale with 1 being strongly agree to 5 being strongly disagree. The maximum score on the PSCQ is 85. The measure takes approximately 4 minutes to administer.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Factors Affecting Post-transplant Cyclophosphamide (PTCy) Efficacy	NCT03555851	Leukemia, Not O... Leukemia, Other	Cyclophosphamid... Specimen collec...	18 Years -	Wake Forest University Health Sciences
Efficacy of a Patient Education Video	NCT02114827	Leukemia, Not O... Malignant Neopl...	Video FAQ Sheet	18 Years -	Virginia Commonwealth University

Spots Global Cancer Trial Database for leukemia, not otherwise specified