Natural History Study to Determine Drug Metabolism Phenotype and Appropriate Germline Source DNA in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant
NCT ID: NCT06856226
Last Updated: 2025-11-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
88 participants
OBSERVATIONAL
2025-11-18
2028-12-01
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
After an allogeneic hematopoietic stem cell transplant (HSCT), the donor genome is found in the recipient s circulation and tissues.
Post-HSCT recipients may receive a medication in which the dosing needs to be adjusted based on genetic variation.
While genes in donor genome may influence dosing and administration of some agents, the majority of established gene-drug pairs in pharmacogenetics are related to expression of metabolic or transporting enzymes located in recipients tissues, often the liver.
Determining which genetic variants influence drug disposition in HSCT recipients is complicated by chimerism in samples that are routinely collected for determining genotype. However, chimerism in tissues is poorly studied in this patient population.
Objectives:
To determine the most reliable host genomic source for pharmacogenetic testing in participants that have received allogeneic HSCT.
Eligibility:
People ages 18 years and older who are enrolled on a clinical trial at the NIH Clinical Center under which they will donate or receive an allogeneic HSCT.
Design:
DNA is collected prior to HSCT and for two years after HSCT.
Blood will be collected and skin fibroblast cell lines will be established prior to HSCT to serve as a reference genome.
Blood, buccal cells, skin, and hair will be monitored for the development of mixed chimerism via detection of short tandem repeats. Liver biopsies will be collected from participants undergoing hepatic surgery.
Pharmacoscan arrays will be conducted to determine which samples are useful for pharmacogenetic testing in participants who receive allogeneic HSCT.
A probe drug cocktail will be administered pre- and post-HSCT to determine if transplantation alters the metabolic phenotype of liver enzymes.
...
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
* After an allogeneic hematopoietic stem cell transplant (HSCT), the donor genome is found in the recipient fs circulation and tissues.
* Post-HSCT recipients may receive a medication in which the dosing needs to be adjusted based on genetic variation.
* While genes in donor genome may influence dosing and administration of some agents, the majority of established gene-drug pairs in pharmacogenetics are related to expression of metabolic or transporting enzymes located in recipients f tissues, often the liver.
* Determining which genetic variants influence drug disposition in HSCT recipients is complicated by chimerism in samples that are routinely collected for determining genotype. However, chimerism in tissues is poorly studied in this patient population.
Primary Objective:
-To determine the most reliable host genomic source for pharmacogenetic testing in participants that have received allogeneic HSCT.
Eligibility:
* Age \>=18 years
* Must be enrolled on a clinical trial at the NIH Clinical Center under which they will donate or receive an allogeneic HSCT
Design:
* DNA is collected prior to HSCT and for two years after HSCT.
* Blood will be collected and skin fibroblast cell lines will be established prior to HSCT to serve as a reference genome.
* Blood, buccal cells, skin, and hair will be monitored for the development of mixed chimerism. Liver biopsies will be collected from participants undergoing hepatic surgery.
* Whole genome sequencing with Aldy analysis will be conducted to determine which samples are useful for pharmacogenetic testing in participants who receive allogeneic HSCT.
* A probe drug cocktail will be administered pre- and post-HSCT to determine if transplantation alters the metabolic phenotype of liver enzymes.
* Up to 88 participants will be enrolled.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Cohort 1
Participants undergoing allogeneic HSCT and donors
Arm 1
Short tandem repeat (STR) analysis from genomic DNA extracted from biospecimens.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Arm 1
Short tandem repeat (STR) analysis from genomic DNA extracted from biospecimens.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Participants must be enrolled on a clinical trial at the NIH Clinical Center (CC) under which they will donate or receive an allogeneic HSCT. The participant and their donor must enroll together to provide a complete set of samples for analysis.
* Ability of subject to understand and the willingness to sign a written informed consent document.
Exclusion Criteria
* Prior allogeneic HSCT
* History of psychiatric disorder which may compromise compliance with protocol requirements.
* Pregnant and lactating individuals
18 Years
120 Years
ALL
Yes
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
National Cancer Institute (NCI)
NIH
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Christopher G Kanakry, M.D.
Role: PRINCIPAL_INVESTIGATOR
National Cancer Institute (NCI)
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
National Institutes of Health Clinical Center
Bethesda, Maryland, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
NIH Clinical Center Office of Patient Recruitment (OPR)
Role: primary
Related Links
Access external resources that provide additional context or updates about the study.
NIH Clinical Center Detailed Web Page
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
001796-C
Identifier Type: -
Identifier Source: secondary_id
10001796
Identifier Type: -
Identifier Source: org_study_id