Acute LeukemiaPhase 1/2NCT05120570
PTCy + Sirolimus/VIC-1911 as GVHD Prophylaxis in Myeloablative PBSC Transplantation
A Phase 1/2 study testing VIC- 1911 in people with Acute Leukemia and Myelodysplastic Syndromes, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a single-arm, phase I/II, study of PTCy/sirolimus plus VIC-1911 to prevent GVHD and relapse after Allogeneic Hematopoietic Cell Transplantation (alloHCT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm
- What is being tested?
- VIC- 1911
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Mar 2022to 30 Jun 2025
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Diagnosis of
- acute leukemia in complete remission, or
- myelodysplasia with <5% blasts, or
- myeloproliferative neoplasm/myelofibrosis with <5% marrow or circulating blasts
- chemosensitive Hodgkin or non-Hodgkin lymphoma
- Age 18 years or older
Show all 19Show fewer
- Performance status of ≥ 80% Karnofsky
- Adequate organ function within 28 days of study registration defined as:
- left ventricular ejection fraction ≥ 45%
- pulmonary function with FEV1, FVC, and DLCO ≥ 50% predicted
- AST and ALT < 2 times upper limit of normal
- Total bilirubin <1.5 times the upper limit of normal. If the patient is suspected of having Gilbert syndrome, they require prior approval of the medical monitor
- creatinine clearance ≥ 50cc/min
- no active/uncontrolled infection
- negative HIV, HBV and HCV
- ferritin < 2000 ng/ml
- Patients able to tolerate oral medication
- Women of childbearing potential and men with partners of child-bearing potential must agree to use of contraception for the duration of treatment through 60 days after the last treatment of VIC-1911 or sirolimus
- Able to provide written voluntary consent prior to the performance of any research related tests or procedures
Who cannot join
- HCT-CI > 4 or unable to receive myeloablative TBI
- Use of planned post-transplant maintenance therapy to begin prior to day +75. Patients may receive standard of care maintenance therapies starting at day
- +75 or later
- Patients with a history of hypersensitivity to any of the investigational products
- Pregnant or breastfeeding as agents used in this study are Pregnancy Category
- o C: Drugs which, owing to their pharmacological effects, have caused or may be suspected of causing, harmful effects on the human fetus or neonate without causing malformations, and Pregnancy category D: There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use of the drug in pregnant women despite potential risks. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 28 days of study registration.
- Women or men of childbearing potential unwilling to take adequate precautions to avoid unintended pregnancy from the start of protocol treatment through 60 days after the last treatment of VIC-1911 or sirolimus
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455
About this study
This is a single-arm, phase I/II, study of PTCy/sirolimus plus VIC-1911 to prevent GVHD and relapse after Allogeneic Hematopoietic Cell Transplantation (alloHCT).
Read the detailed description
Determination of the optimal dose during the Phase I trial is based on Dose Limiting Toxicity for safety and reduction of CD4+, pH3ser10+ T cells (phosphorylated histone 3 serine 10 is a biomarker of Aurora kinase A activity) for efficacy. Phase II will be powered to improve grade III-IV acute graft-versus-host disease and relapse after alloHCT, compared to historical estimates at the University of Minnesota.
Patients will receive myeloablative conditioning (MAC) with total body irradiation (TBI) followed by infusion of HLA-matched related or unrelated peripheral blood stem cells (PBSC) on day 0. Cyclophosphamide will be administered on days +3 and +4. Sirolimus targeting 8-12ng/ml will begin on day +5 until day +365. VIC-1911 will be administered as 25 mg, 50 mg, or 75 mg orally BID from day +5 to day +45 according to the rules of our phase I study. The lowest biologically active and safe dose of VIC-1911 will be identified as the recommended phase II dose.
- Being tested
- VIC- 1911
- Keywords
- VIC-1911, PTCy, Myeloablative, Allogeneic
- Medical subject headings
- Myelodysplastic Syndromes, Myeloproliferative Disorders, Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VIC- 1911Drug
25 mg, 50 mg, or 75 mg administered twice a day from day 5 post HCT to day 45, and the dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
| Study group | Role | What they receive |
|---|---|---|
| PTCy/sirolimus plus VIC-1911 | Experimental | Patients enrolled and treated with PTCy/sirolimus plus VIC-1911 Receives: Drug: VIC- 1911 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the Optimal Dose of VIC-1911 When Given in Combination With Standard Immunosuppressive Therapy in Adult Patients Undergoing Myeloablative Stem Cell Transplantation.
The optimal dose will be identified using the EffTox design. The proportion of patients with an average CD4+, pH3ser10+ T cell of <54%. The minimum desired biologic efficacy is 65% of patients by day 21 (+/- 3 days) with <30% of patients experiencing a DLT. Data only to reported from arm with maximum tolerated dose.
Measured: 21 days post treatment
Progression-free Survival
Participant progression-free survival assessed using aGVHD data.
Measured: 1 Year
Relapsed Assessment (Phase I)
Assessment to determine if patient has relapse in MTD arm. Data only to reported from arm with maximum tolerated dose.
Measured: 12 months
Secondary outcomes (6)
Overall Survival (OS)
Overall Survival for participants on MTD arm. Data only to reported from arm with maximum tolerated dose.
Measured: 1 year
To Determine the Cumulative Incidences of Acute GVHD
Assessment of aGVHD for MTD arm. Data only to reported from arm with maximum tolerated dose.
Measured: Day 100
To Determine the Cumulative Incidences of Chronic GVHD
Assessment of cGVHD
Measured: 12 months
Progression-free Survival Comparing Graft-Versus-Host Disease-Free (GRFS) to the Standard PTCY Plus Tacrolimus/Mycophenolate Mofetil Regimen From MT2015-29
Progression-free Survival assessed using GRFS defined as grade III-IV acute GVHD, chronic GVHD requiring immunosuppression, relapse, or death by 1 year
Measured: 12 months
Progression Free Survival
Percentage of participants with progression free survival at 1 year for MTD arm. Data only to reported from arm with maximum tolerated dose.
Measured: 1 Year
Frequency of CMV Reactivation and Disease
Analyze the frequency of CMV reactivation and disease for MTD arm. Data only to reported from arm with maximum tolerated dose.
Measured: Day 100
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2021
- Study start17 Mar 2022
- Primary completion19 Sep 2024
- Study completion30 Jun 2025
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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 1/2
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sherman Holtan, MD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05120570
- Sponsor's protocol ID
- 2021LS006
- Other IDs
- MT2021-01
- Status last verified
- Dec 2025
- First submitted
- 3 Nov 2021
- Last updated
- 22 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Holtan SG, Grover P, Walton K, El Jurdi N, Quinones V, Maakaron JE, Juckett MB, Bachanova V, Hou JH, Terezakis S, Myers TJ, Paradiso LJ, DeFor TE, Cao Q, Betts BC. Targeting Aurora kinase A to prevent GVHD and relapse after myeloablative allogeneic hematopoietic cell transplantation. Blood Adv. 2026 Apr 14;10(7):2190-2201. doi: 10.1182/bloodadvances.2025017360.
PubMed 41592279
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PTCy + Sirolimus/VIC-1911 as GVHD Prophylaxis in Myeloablative PBSC Transplantation
- ClinicalTrials.gov ID
- NCT05120570
- Acronym / sponsor ID
- 2021LS006
- Keywords
- VIC-1911, PTCy, Myeloablative, Allogeneic
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 17 Mar 2022 (actual)
- Primary completion
- 19 Sep 2024 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 15 Nov 2021 (actual)
- Results
- Posted 22 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single-arm, phase I/II, study of PTCy/sirolimus plus VIC-1911 to prevent GVHD and relapse after Allogeneic Hematopoietic Cell Transplantation (alloHCT).
Detailed description
Determination of the optimal dose during the Phase I trial is based on Dose Limiting Toxicity for safety and reduction of CD4+, pH3ser10+ T cells (phosphorylated histone 3 serine 10 is a biomarker of Aurora kinase A activity) for efficacy. Phase II will be powered to improve grade III-IV acute graft-versus-host disease and relapse after alloHCT, compared to historical estimates at the University of Minnesota.
Patients will receive myeloablative conditioning (MAC) with total body irradiation (TBI) followed by infusion of HLA-matched related or unrelated peripheral blood stem cells (PBSC) on day 0. Cyclophosphamide will be administered on days +3 and +4. Sirolimus targeting 8-12ng/ml will begin on day +5 until day +365. VIC-1911 will be administered as 25 mg, 50 mg, or 75 mg orally BID from day +5 to day +45 according to the rules of our phase I study. The lowest biologically active and safe dose of VIC-1911 will be identified as the recommended phase II dose.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTCy/sirolimus plus VIC-1911 | Experimental | Drug: VIC- 1911Patients enrolled and treated with PTCy/sirolimus plus VIC-1911 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Diagnosis of
- Age 18 years or older
- Performance status of ≥ 80% Karnofsky
- Adequate organ function within 28 days of study registration defined as:
- Patients able to tolerate oral medication
Key exclusion criteria (7 listed)
- HCT-CI > 4 or unable to receive myeloablative TBI
- Use of planned post-transplant maintenance therapy to begin prior to day +75. Patients may receive standard of care maintenance therapies starting at day
- +75 or later
- Patients with a history of hypersensitivity to any of the investigational products
- Pregnant or breastfeeding as agents used in this study are Pregnancy Category
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 |
|---|---|---|
| VIC- 191125 mg, 50 mg, or 75 mg administered twice a day from day 5 post HCT to day 45, and the dose escalation will stop once we identify the lowest biologically active and safe dose of VIC. | Drug | PTCy/sirolimus plus VIC-1911 |
Primary outcome
Determine the Optimal Dose of VIC-1911 When Given in Combination With Standard Immunosuppressive Therapy in Adult Patients Undergoing Myeloablative Stem Cell Transplantation.
Time frame: 21 days post treatment
The optimal dose will be identified using the EffTox design. The proportion of patients with an average CD4+, pH3ser10+ T cell of <54%. The minimum desired biologic efficacy is 65% of patients by day 21 (+/- 3 days) with <30% of patients experiencing a DLT. Data only to reported from arm with maximum tolerated dose.
Progression-free Survival
Time frame: 1 Year
Participant progression-free survival assessed using aGVHD data.
Relapsed Assessment (Phase I)
Time frame: 12 months
Assessment to determine if patient has relapse in MTD arm. Data only to reported from arm with maximum tolerated dose.
6 secondary outcomes
Overall Survival (OS)
Time frame: 1 year
To Determine the Cumulative Incidences of Acute GVHD
Time frame: Day 100
To Determine the Cumulative Incidences of Chronic GVHD
Time frame: 12 months
Progression-free Survival Comparing Graft-Versus-Host Disease-Free (GRFS) to the Standard PTCY Plus Tacrolimus/Mycophenolate Mofetil Regimen From MT2015-29
Time frame: 12 months
Progression Free Survival
Time frame: 1 Year
Frequency of CMV Reactivation and Disease
Time frame: Day 100
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41592279Holtan SG, Grover P, Walton K, El Jurdi N, Quinones V, Maakaron JE, Juckett MB, Bachanova V, Hou JH, Terezakis S, Myers TJ, Paradiso LJ, DeFor TE, Cao Q, Betts BC. Targeting Aurora kinase A to prevent GVHD and relapse after myeloablative allogeneic hematopoietic cell transplantation. Blood Adv. 2026 Apr 14;10(7):2190-2201. doi: 10.1182/bloodadvances.2025017360.
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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.
Sherman Holtan, MD principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VIC- 1911 in people with Acute Leukemia and Myelodysplastic Syndromes, run by Masonic Cancer Center, University of Minnesota 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 19 inclusion and 7 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: finished, as of the sponsor's last update on 22 Dec 2025. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Yes. Results are posted in the registry (first posted 22 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Determine the Optimal Dose of VIC-1911 When Given in Combination With Standard Immunosuppressive Therapy in Adult Patients Undergoing Myeloablative Stem Cell Transplantation. (time frame: 21 days post treatment). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05120570, which also lists 1 linked publication.
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 22 Dec 2025.