Examining factors that affect the effectiveness of post-transplant cyclophosphamide
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 is trying to see how genetic factors and drug levels affect the success of a treatment called post-transplant cyclophosphamide in patients who have had a stem cell transplant.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 120 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Wake Forest University Health Sciences Academic / other |
| Drugs / interventions | cyclophosphamide |
| Locations | 1 site (Charlotte, North Carolina) |
| Trial ID | NCT03555851 on ClinicalTrials.gov |
What this trial studies
This observational study aims to investigate how donor and recipient pharmacogenetics, drug pharmacokinetics, and T cell phenotypes influence the efficacy of post-transplant cyclophosphamide (PTCy) in patients undergoing hematopoietic stem cell transplants. The primary goal is to identify associations between genetic factors and the incidence and severity of acute and chronic graft versus host disease (GVHD). Additionally, the study will measure cyclophosphamide exposure and its correlation with GVHD and adverse events. Initially, 20 participants will be enrolled, followed by an additional 100 subjects to enhance the study's findings.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older who are scheduled for hematopoietic stem cell transplants with haploidentical or matched related donors and are planned to receive post-transplant cyclophosphamide.
Not a fit: Patients who are not undergoing hematopoietic stem cell transplants or those receiving unrelated donor transplants may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a more personalized dosing strategy for post-transplant cyclophosphamide, improving patient outcomes and reducing the risk of GVHD.
How similar studies have performed: While this study explores a specific approach, similar studies have shown promise in tailoring treatments based on pharmacogenetics, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria Recipients and donors must meet all of the following applicable inclusion criteria to participate in this study: 1. Informed consent and HIPAA authorization for release of personal health information signed by the subject. 2. Age ≥ 18 years at the time of consent. 3. 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. 4. Recipient only: Planned post-transplant cyclophosphamide 5. As determined by the enrolling physician, ability of the subject to understand and comply with study procedures for the entire length of the study Exclusion Criteria Subjects meeting any of the criteria below may not participate in the study: 1. 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. 2. Recipient only: Treatment with any investigational drug within 30 days prior to day -6 of treatment 3. 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.
Where this trial is running
Charlotte, North Carolina
- Levine Cancer Institute — Charlotte, North Carolina, United States (Recruiting)
Study contacts
- Principal investigator: Aleksander Chojecki, MD — Wake Forest University Health Sciences
- Study coordinator: Elizabeth Parke
- Email: elizabeth.parke@atriumhealth.org
- Phone: 980-442-2011
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.