Leukemia, Not Otherwise SpecifiedNCT03555851
Factors Affecting Post-transplant Cyclophosphamide (PTCy) Efficacy
An observational study of Leukemia, Not Otherwise Specified and Leukemia, Other, run by Wake Forest University Health Sciences at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Not Otherwise Specified, Leukemia, Other
- What is being tested?
- Cyclophosphamide and Specimen collection
- How many people
- 120 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Jul 2018to Aug 2035
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
- Recipients and donors must meet all of the following applicable inclusion criteria to participate in this study:
- Informed consent and HIPAA authorization for release of personal health information signed by the subject.
- Age ≥ 18 years at the time of consent.
- Subject is scheduled as a recipient or respective donor (Donor consent/participation is not required for subjects undergoing matched unrelated donor HCT) for the following hematopoietic stem cell transplants (HCT) procedures using a non-myeloablative regimen at Levine Cancer Institute (LCI), and has been deemed a qualified candidate by his/her physician, per LCI medical standards: haplo-identical donor HCT, match related donor (MRD) HCT, matched unrelated donor (MUD) HCT.
- Recipient only: Planned post-transplant cyclophosphamide
- As determined by the enrolling physician, ability of the subject to understand and comply with study procedures for the entire length of the study
Who cannot join
- Subjects meeting any of the criteria below may not participate in the study:
- Recipient only (applies only to haplo-identical and MRD HCT recipients; not required for MUD HCT recipients): Does not have a respective donor who is willing to sign informed consent for participation in this study.
- Recipient only: Treatment with any investigational drug within 30 days prior to day -6 of treatment
- Donor only (applies only to haplo-identical and MRD HCTs; donor participation is not required for MUD HCTs): Does not have a respective recipient who is willing to sign informed consent for participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Levine Cancer Institute
Charlotte, North Carolina, 28204
Elizabeth Parke, 980-442-2011, elizabeth.parke@atriumhealth.org
About this study
Official title: 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
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).
Read the detailed description
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.
- Conditions
- Leukemia, Not Otherwise Specified, Leukemia, Other
- Being tested
- Cyclophosphamide, Specimen collection
- Medical subject headings
- Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
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.
Specimen collectionOther
Buccal swabs will be obtained from donors for pharmacogenomics.
| Study group | Role | What they receive |
|---|---|---|
| Recipient | Cyclophosphamide Receives: Drug: Cyclophosphamide; Other: Specimen collection | |
| Donor | Specimen collection Receives: Other: Specimen collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Approx. 24 mos
Secondary outcomes (4)
Incidence of chronic GVHD
Calculated for each subject as a binary variable indicating whether or not subject experienced chronic GVHD
Measured: 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.
Measured: Approx. 10 days
Toxicities
The incidence of adverse events of special interests will be collected according to CTCAE version 4.03
Measured: 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.
Measured: Approx. 24 mos
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2018
- Study start13 Jul 2018
- Primary completionOct 2033 (estimated)
- Study completionAug 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Not Otherwise SpecifiedStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- Atrium Health Levine Cancer Institute
- Responsible party
- Sponsor
- Investigators
- Aleksander Chojecki, MD, Principal investigator, Wake Forest University Health Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03555851
- Sponsor's protocol ID
- IRB00081375
- Other IDs
- LCI-HEM-HCT-PTCY-001, Pro00024582, NCI-2024-06503
- Status last verified
- Jul 2026
- First submitted
- 2 May 2018
- Last updated
- 13 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT03555851
- Acronym / sponsor ID
- IRB00081375
- Conditions
- Leukemia, Not Otherwise Specified, Leukemia, Other
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Collaborators
- Atrium Health Levine Cancer Institute
- Study start
- 13 Jul 2018 (actual)
- Primary completion
- Oct 2033 (estimated)
- Study completion
- Aug 2035 (estimated)
- First posted
- 14 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Recipient | – | Drug: Cyclophosphamide, Other: Specimen collectionCyclophosphamide |
| Donor | – | Other: Specimen collectionSpecimen collection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (6 listed)
- Recipients and donors must meet all of the following applicable inclusion criteria to participate in this study:
- Informed consent and HIPAA authorization for release of personal health information signed by the subject.
- Age ≥ 18 years at the time of consent.
- Subject is scheduled as a recipient or respective donor (Donor consent/participation is not required for subjects undergoing matched unrelated donor HCT) for the following hematopoietic stem cell transplants (HCT) procedures using a non-myeloablative regimen at Levine Cancer Institute (LCI), and has been deemed a qualified candidate by his/her physician, per LCI medical standards: haplo-identical donor HCT, match related donor (MRD) HCT, matched unrelated donor (MUD) HCT.
- Recipient only: Planned post-transplant cyclophosphamide
Key exclusion criteria (4 listed)
- Recipient only (applies only to haplo-identical and MRD HCT recipients; not required for MUD HCT recipients): Does not have a respective donor who is willing to sign informed consent for participation in this study.
- Recipient only: Treatment with any investigational drug within 30 days prior to day -6 of treatment
- Donor only (applies only to haplo-identical and MRD HCTs; donor participation is not required for MUD HCTs): Does not have a respective recipient who is willing to sign informed consent for participation in this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CyclophosphamidePharmacogenomics 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. | Drug | Recipient |
| Specimen collectionBuccal swabs will be obtained from donors for pharmacogenomics. | Other | Donor, Recipient |
Primary outcome
Comparison of Cy cMax Values
Time frame: Approx. 24 mos
Evaluation and comparison of average Day 3 Cy cMax values between subjects who experience acute GVHD versus subjects who do not experience acute GVHD
4 secondary outcomes
Incidence of chronic GVHD
Time frame: Approx. 24 mos
Cy exposure
Time frame: Approx. 10 days
Toxicities
Time frame: Approx. 180 days
Pharmacogenetics of Cy-related genes
Time frame: Approx. 24 mos
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Leukemia, Not Otherwise Specified
Active, not recruitingPhase 1
Cellular Immunotherapy Following Chemotherapy in Treating Patients With Recurrent Non-Hodgkin Lymphomas, Chronic Lymphocytic Leukemia, or B-Cell Prolymphocytic Leukemia
B-Cell Lymphoma, Unclassifiable, With Features Intermediate Between Diffuse Large B-Cell Lymphoma and Classic Hodgkin Lymphoma, B-Cell Prolymphocytic Leukemia
Active, not recruitingPhase 1/2
A Study to Assess the Safety, Tolerability, Efficacy, and Drug Levels of BMS-986369 (Golcadomide) in Participants With Relapsed or Refractory T-cell Lymphomas in Japan (GOLSEEK-3)
Relapsed or Refractory T-cell Lymphomas
RecruitingPhase 2
Decitabine With Ruxolitinib, Fedratinib or Pacritinib for the Treatment of Accelerated/Blast Phase Myeloproliferative Neoplasms
Acute Myeloid Leukemia, Essential Thrombocythemia
Active, not recruitingPhase 2
Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders
BCR-JAK2 Fusion Protein Expression, Blasts 20 Percent or Less of Peripheral Blood White Cells
Active, not recruitingPhase 2
Brentuximab Vedotin and Combination Chemotherapy in Treating Patients With CD30-Positive Peripheral T-cell Lymphoma
Adult T-Cell Leukemia/Lymphoma, Anaplastic Large Cell Lymphoma, ALK-Negative
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Aleksander Chojecki, MD principal investigator
Wake Forest University Health Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Not Otherwise Specified and Leukemia, Other, run by Wake Forest University Health Sciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Elizabeth Parke (980-442-2011, elizabeth.parke@atriumhealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparison of Cy cMax Values (time frame: Approx. 24 mos). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jul 2026.