Graft-versus-host DiseasePhase 2NCT05622318
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
A Phase 2 study testing Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil in people with Graft-versus-host Disease, run by Medical College of Wisconsin at 1 site in United States.
Trial summary
This is an open-label phase 2 study designed to explore the efficacy and safety of low-dose PTCy-ruxolitinib GVHD prophylaxis in older adults undergoing allogeneic HCT with a matched sibling or unrelated donor with a peripheral blood stem cell graft. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Graft-versus-host Disease
- What is being tested?
- Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Aug 2023to 6 Aug 2028
- Phase
- Phase 2
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- History of hematologic malignancy.
- Must be in remission:
- Acute Leukemia, chronic leukemia, or myelodysplasia/myeloproliferative neoplasm (excluding primary myelofibrosis): No circulating blasts and <5% blasts in the bone marrow.
- Hodgkin and non-Hodgkin lymphomas: Chemo-sensitive disease at time of transplant
- Patients must have a related or unrelated peripheral blood stem cell donor that is an 8/8 match at HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing. Unrelated donors must be willing to donate peripheral blood stem cells and meet NMDP criteria for donation.
- Planned reduced intensity conditioning therapy with fludarabine/melphalan, with total dose of melphalan of 100-140 mg/m^2 IV or fludarabine/busulfan with total dose of busulfan of 6.4 mg/kg IV.
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- Karnofsky Performance Scale of 60 or greater.
- Male participants must agree to abstinence or to use of barrier contraception during the entire study period.
- Female participants of childbearing potential will require a negative pregnancy test and should agree to practice two effective methods of contraception during the entire study period.
- Ability to understand a written informed consent document, and the willingness to sign it.
Who cannot join
- Prior allogeneic HCT or Chimeric antigen receptor (CAR) -T cell therapy.
- Patients with liver dysfunction evidenced by bilirubin ≥2x upper limit normal (ULN), except for a history of Gilbert syndrome.
- Patients with renal impairment defined by creatinine<2mg/dL.
- Patients with cardiac dysfunction defined by a left ventricular ejection fraction ≤45%.
- Patients with pulmonary dysfunction defined by a forced expiratory volume in the first second (FEV1) or diffusing capacity for carbon monoxide (DLCO) (corrected for hemoglobin) ≤50% of predicted.
- Patients with a chronic or active infection requiring systemic treatment during and after transplant.
- Presence of other active malignant disease diagnosed within 12 months, except for adequately treated non-melanoma skin cancer, adequately treated melanoma grade 2 or less, or cervical intraepithelial neoplasia. Active malignancy is malignancy receiving treatment.
- Pregnant or lactating subjects.
Trial locations and contacts
1 site in 1 country.
United States1 site
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: A Phase II Trial of De-escalated PTCy and Ruxolitinib for GVHD Prophylaxis in Patients Undergoing Reduced Intensity Conditioning Allogeneic HCT
This is an open-label phase 2 study designed to explore the efficacy and safety of low-dose PTCy-ruxolitinib GVHD prophylaxis in older adults undergoing allogeneic HCT with a matched sibling or unrelated donor with a peripheral blood stem cell graft.
Read the detailed description
Following reduced intensity conditioning and 8/8-matched peripheral blood transplant on Day 0, all patients will receive a GVHD prophylaxis post-transplant composed of the following: (i) cyclophosphamide administered at 25 mg/kg on Day +3 and +4, (ii) tacrolimus beginning on Day +5 and through Day +180 and administered with a trough target of 5-10 ng/ml through Day +90 and tapered thereafter; (iii) mycophenolate mofetil (MMF) administered at 15 mg/kg thrice daily beginning on Day +5 through Day +35; and (iv) ruxolitinib administered at 5 mg twice daily starting after engraftment (between Days +30 and +60) and continuing through one year post transplant.
- Conditions
- Graft-versus-host Disease
- Being tested
- Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Ruxolitinib
- Keywords
- Graft-versus-host disease, Ruxolitinib, Graft-versus-host disease prophylaxis
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called Cytoxan
25 mg/kg by IV on Days +3 and +4.
TacrolimusDrug
Also called Astagraf XL, Envarsus XR, Prograf
Target level 5-10 ng/mL (If the subject experiences nausea and vomiting that prevents the oral intake of tacrolimus anytime during treatment, tacrolimus is to be given by IV at the appropriate dose that was used to obtain the therapeutic level [IV:PO ratio = 1:4]). Administered Days +5 through +90. Taper after Day +90 and discontinue on Day +180.
Also called CellCept
15 mg/kg tablet thrice daily Days +5 through +35 every eight hours.
RuxolitinibDrug
Also called INC424, Jakafi
5 mg tablet twice daily after engraftment through Day +365. Taper after Day +365.
| Study group | Role | What they receive |
|---|---|---|
| Graft-versus-host disease prophylaxis | Experimental | Following reduced intensity conditioning and 8/8-matched peripheral blood transplant on Day 0, all patients will receive a GVHD prophylaxis post-transplant composed of the following: (i) cyclophosphamide administered at 25 mg/kg on Day +3 and +4, (ii) tacrolimus beginning on Day +5 and through Day +180 and administered with a trough target of 5-10 ng/ml through Day +90 and tapered thereafter; (iii) mycophenolate mofetil (MMF) administered at 15 mg/kg thrice daily beginning on Day +5 through Day +35; and (iv) ruxolitinib administered at 5 mg twice daily starting after engraftment (between Days +30 and +60) and continuing through one year post transplant. Receives: Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of subjects who experience GVHD-free survival.
This measure is defined as being alive without having experienced grade III/IV acute GVHD, or chronic GVHD requiring systemic immune suppression.
Measured: One year (365 Days) after hematopoietic cell transplantation (HCT)
Secondary outcomes (6)
The number of subjects with acute GVHD at Day +100.
The staging and grading of acute GVHD will be done according to Consensus GVHD grading criteria.
Measured: Day +100 after HCT
The number of subjects with acute GVHD at Day +180.
The staging and grading of acute GVHD will be done according to Consensus GVHD grading criteria.
Measured: Day +180 after HCT
The number of subjects with chronic GVHD at one year.
Chronic GVHD will be graded as mild, moderate, or severe according to the NIH consensus criteria.
Measured: One year after HCT
The number of subjects with non-relapse mortality at Day +100.
This is defined as death before day +100 after transplant that was not preceded by recurrent or progressive malignancy.
Measured: Day +100 after HCT
The number of subjects with non-relapse mortality at one year.
This is defined as death before day +100 after transplant that was not preceded by recurrent or progressive malignancy.
Measured: One year after HCT
Overall survival at one year.
This is defined as the number of subjects alive one year after HCT.
Measured: One year after HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2022
- Study start29 Aug 2023
- Primary completion8 Jul 2028 (estimated)
- Study completion6 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft-versus-host DiseaseStudies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe 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
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sameem M. Abedin, MD, Medical College of Wisconsin
- Investigators
- Sameem Abedin, MD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05622318
- Sponsor's protocol ID
- PRO00046962
- Status last verified
- Nov 2026
- First submitted
- 11 Nov 2022
- Last updated
- 6 Nov 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 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of De-escalated PTCy and Ruxolitinib for GVHD Prophylaxis in Patients Undergoing Reduced Intensity Conditioning Allogeneic HCT
- ClinicalTrials.gov ID
- NCT05622318
- Acronym / sponsor ID
- PRO00046962
- Conditions
- Graft-versus-host Disease
- Keywords
- Graft-versus-host disease, Ruxolitinib, Graft-versus-host disease prophylaxis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Study start
- 29 Aug 2023 (actual)
- Primary completion
- 8 Jul 2028 (estimated)
- Study completion
- 6 Aug 2028 (estimated)
- First posted
- 18 Nov 2022 (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 is an open-label phase 2 study designed to explore the efficacy and safety of low-dose PTCy-ruxolitinib GVHD prophylaxis in older adults undergoing allogeneic HCT with a matched sibling or unrelated donor with a peripheral blood stem cell graft.
Detailed description
Following reduced intensity conditioning and 8/8-matched peripheral blood transplant on Day 0, all patients will receive a GVHD prophylaxis post-transplant composed of the following: (i) cyclophosphamide administered at 25 mg/kg on Day +3 and +4, (ii) tacrolimus beginning on Day +5 and through Day +180 and administered with a trough target of 5-10 ng/ml through Day +90 and tapered thereafter; (iii) mycophenolate mofetil (MMF) administered at 15 mg/kg thrice daily beginning on Day +5 through Day +35; and (iv) ruxolitinib administered at 5 mg twice daily starting after engraftment (between Days +30 and +60) and continuing through one year post transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Graft-versus-host disease prophylaxis | Experimental | Drug: Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Drug: RuxolitinibFollowing reduced intensity conditioning and 8/8-matched peripheral blood transplant on Day 0, all patients will receive a GVHD prophylaxis post-transplant composed of the following: (i) cyclophosphamide administered at 25 mg/kg on Day +3 and +4, (ii) tacrolimus beginning on Day +5 and through Day +180 and administered with a trough target of 5-10 ng/ml through Day +90 and tapered thereafter; (iii) mycophenolate mofetil (MMF) administered at 15 mg/kg thrice daily beginning on Day +5 through Day +35; and (iv) ruxolitinib administered at 5 mg twice daily starting after engraftment (between Days +30 and +60) and continuing through one year post transplant. |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- History of hematologic malignancy.
- Must be in remission:
- Patients must have a related or unrelated peripheral blood stem cell donor that is an 8/8 match at HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing. Unrelated donors must be willing to donate peripheral blood stem cells and meet NMDP criteria for donation.
- Planned reduced intensity conditioning therapy with fludarabine/melphalan, with total dose of melphalan of 100-140 mg/m^2 IV or fludarabine/busulfan with total dose of busulfan of 6.4 mg/kg IV.
- Karnofsky Performance Scale of 60 or greater.
Key exclusion criteria (8 listed)
- Prior allogeneic HCT or Chimeric antigen receptor (CAR) -T cell therapy.
- Patients with liver dysfunction evidenced by bilirubin ≥2x upper limit normal (ULN), except for a history of Gilbert syndrome.
- Patients with renal impairment defined by creatinine<2mg/dL.
- Patients with cardiac dysfunction defined by a left ventricular ejection fraction ≤45%.
- Patients with pulmonary dysfunction defined by a forced expiratory volume in the first second (FEV1) or diffusing capacity for carbon monoxide (DLCO) (corrected for hemoglobin) ≤50% of predicted.
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 |
|---|---|---|
| CyclophosphamideAlso: Cytoxan25 mg/kg by IV on Days +3 and +4. | Drug | Graft-versus-host disease prophylaxis |
| TacrolimusAlso: Astagraf XL, Envarsus XR, PrografTarget level 5-10 ng/mL (If the subject experiences nausea and vomiting that prevents the oral intake of tacrolimus anytime during treatment, tacrolimus is to be given by IV at the appropriate dose that was used to obtain the therapeutic level [IV:PO ratio = 1:4]). Administered Days +5 through +90. Taper after Day +90 and discontinue on Day +180. | Drug | Graft-versus-host disease prophylaxis |
| Mycophenolate MofetilAlso: CellCept15 mg/kg tablet thrice daily Days +5 through +35 every eight hours. | Drug | Graft-versus-host disease prophylaxis |
| RuxolitinibAlso: INC424, Jakafi5 mg tablet twice daily after engraftment through Day +365. Taper after Day +365. | Drug | Graft-versus-host disease prophylaxis |
Primary outcome
The number of subjects who experience GVHD-free survival.
Time frame: One year (365 Days) after hematopoietic cell transplantation (HCT)
This measure is defined as being alive without having experienced grade III/IV acute GVHD, or chronic GVHD requiring systemic immune suppression.
6 secondary outcomes
The number of subjects with acute GVHD at Day +100.
Time frame: Day +100 after HCT
The number of subjects with acute GVHD at Day +180.
Time frame: Day +180 after HCT
The number of subjects with chronic GVHD at one year.
Time frame: One year after HCT
The number of subjects with non-relapse mortality at Day +100.
Time frame: Day +100 after HCT
The number of subjects with non-relapse mortality at one year.
Time frame: One year after HCT
Overall survival at one year.
Time frame: One year after HCT
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 6 Nov 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Sameem Abedin, MD principal investigator
Medical College of Wisconsin
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil in people with Graft-versus-host Disease, run by Medical College of Wisconsin 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 10 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 6 Nov 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number of subjects who experience GVHD-free survival. (time frame: One year (365 Days) after hematopoietic cell transplantation (HCT)). 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 6 Nov 2026; AzTrials last refreshed its copy on 7 Nov 2026.