GVHDPhase 2NCT03945591
High-Dose Post-Transplant Cyclophosphamide and Bortezomib (CyBor) for the Prevention of Graft-versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
A Phase 2 study testing Bortezomib and Cyclophosphamide in people with GVHD, run by NYU Langone Health at 1 site in United States.
Trial summary
This is a single arm open label phase II clinical trial. Adult patients with hematological malignancies undergoing allogeneic HSCT from matched-related or unrelated donor are eligible for the study if they meet the standard criteria defined in the investigator's institutional standard operation procedures (SOPs), meet… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- GVHD
- What is being tested?
- Bortezomib and Cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Jun 2019to 4 Jan 2024
- Phase
- Phase 2
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
Who may be able to join
- Karnofsky score ≥ 70%
- No evidence of progressive bacterial, viral, or fungal infection
- Creatinine clearance > 50 mL/min/1.72m2
- Total bilirubin, ALT and AST < 2 x the upper limit of normal (except for Gilbert's syndrome)
- Alkaline phosphatase ≤ 250 IU/L
- Left Ventricular Ejection Fraction (LVEF) > 45%
Show all 9Show fewer
- Adjusted Carbon Monoxide Diffusing Capacity (DLCO) > 60%
- Negative HIV serology
- Negative pregnancy test: confirmation per negative serum β-human chorionic gonadotropin (β-hCG)
Who cannot join
- Pregnant or nursing females or women of reproductive capability who are unwilling to completely abstain from heterosexual sex or practice 2 effective methods of contraception from the first dose of bortezomib through 90 days after the last dose. A woman of reproductive capability is one who has not undergone a hysterectomy (removal of the womb), has not had both ovaries removed, or has not been post-menopausal (stopped menstrual periods) for more than 24 months in a row.
- Male subjects who refuse to practice effective barrier contraception during the entire study treatment period and through a minimum of 90 days after the last dose of study drug, or completely abstain from heterosexual intercourse. This must be done even if they are surgically sterilized (i.e., post-vasectomy).
- Inability to provide informed consent.
- Patient had myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure (see Appendix E), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening must be documented by the investigator as not medically relevant.
- Known allergies to any of the components of the investigational treatment regimen.
- Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
Show all 9Show fewer
- Diagnosed or treated for another malignancy within 3 years of enrollment, with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma, an in-situ malignancy, or low-risk prostate cancer after curative therapy.
- Participation in clinical trials with other investigational agents not included in this trial, within 14 days of the start of this trial and throughout the duration of this trial.
- Prisoners
Trial locations and contacts
1 site in 1 country.
United States1 site
New York University School of Medicine
New York, New York, 10016
About this study
Official title: A Phase II Study of High-Dose Post-Transplant Cyclophosphamide and Bortezomib (CyBor) for the Prevention of Graft-versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
This is a single arm open label phase II clinical trial. Adult patients with hematological malignancies undergoing allogeneic HSCT from matched-related or unrelated donor are eligible for the study if they meet the standard criteria defined in the investigator's institutional standard operation procedures (SOPs), meet all inclusion criteria, and do not satisfy any exclusion criteria. Patients will receive reduced-intensity or myeloablative conditioning regimen of fludarabine, busulfan, and rabbit anti-thymocyte globulin (rATG). Patients will receive PTCyBor as GvHD prophylaxis.
- Conditions
- GVHD
- Being tested
- Bortezomib, Cyclophosphamide
- Keywords
- Cyclophosphamide, Bortezomib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BortezomibDrug
1.3 mg/m2 IV 6 hours after graft infusion and 72 hours thereafter.
CyclophosphamideDrug
50 mg/kg IV over 2 hours on Day +3 and +4
| Study group | Role | What they receive |
|---|---|---|
| Cyclophosphamide and Bortezomib | Experimental | Not described Receives: Drug: Bortezomib; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Experience Acute GvHD
Rate of acute GvHD post-transplant. All participants that received a transplant and received any prophylactic treatment will be included in the analysis.
Measured: Day 120 Post-Transplant
Percentage of Participants Who Experience Moderate to Severe Chronic GvHD
Rate of chronic GvHD post-transplant. All participants that received a transplant and received any prophylactic treatment will be included in the analysis.
Measured: Day 365 Post-Transplant
Secondary outcomes (7)
Incidence of Primary Graft Failure
Incidence of graft failure will be calculated from date of transplant to failure for all patients who receive a transplant and any prophylactic treatment and from date of completion of prophylactic treatment for all participants that completed treatment. Graft failure is defined as failure to achieve neutrophil engraftment by day 28 post-transplant or lack of donor chimerism > 50% by day 45 post-transplant not due to the underlying malignancy.
Measured: Day 45 Post-Transplant
Incidence of Poor Graft Function
Incidence of poor graft function will be calculated from date of transplant to failure for all patients who receive a transplant and any prophylactic treatment and from date of completion of prophylactic treatment for all participants that completed treatment. Poor graft function is defined by at least 2 of the following 3 criteria: Hemoglobin < 8 g/dL, ANC < 0.5 109/L, and platelets < 20 109/L. The cytopenia must be unexplained (such as by disease relapse) and unresponsive to cytokines and must last at least 4 weeks.
Measured: Day 30 Post-Transplant
Incidence of Secondary Graft Failure
Incidence of secondary graft failure is evaluated after engraftment is achieved; this outcome is calculated from date of engraftment for all patients with engraftment. Secondary graft failure is defined as poor graft function associated with donor chimerism < 5%.
Measured: Day 730 Post-Transplant
Treatment Related Mortality (TRM)
Number of participant deaths not attributable to disease relapse or progression . This outcome is analyzed based on participants that who received a transplant with any prophylactic treatment and for all patients who received a transplant and completed prophylactic treatment.
Measured: Day 730 Post-Transplant
Relapse Rate (RR)
Percentage of participants in whom the disease for which transplant is performed is evident by methods of disease detection after transplant. This outcome is analyzed for all patients who received a transplant and for all transplanted patients that completed treatment.
Measured: Day 730 Post-Transplant
Graft Versus Host Disease Relapse Free Survival (GRFS)
Percentage of participants who are without reported GvHD III-IV acute GvHD, chronic GvHD requiring systemic therapy and have not experienced relapse or death after transplant.
Measured: Day 730 Post-Transplant
Overall Survival (OS)
Percentage of participants alive at the end of the study's evaluation period.
Measured: Day 730 Post-Transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2019
- Study start20 Jun 2019
- Primary completion29 Dec 2022
- Study completion4 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for GVHDStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ahmad Al-Homsi, MD, Principal investigator, New York Langone Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03945591
- Sponsor's protocol ID
- 18-01185
- Status last verified
- Apr 2026
- First submitted
- 8 May 2019
- Last updated
- 23 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices).Upon reasonable request is likely the most appropriate as well will report on incidental findings (i.e. infections) that may impact transplant.