NCT05120570

PTCy + Sirolimus/VIC-1911 as GVHD Prophylaxis in Myeloablative PBSC Transplantation

Completed Phase 1, PHASE2 Results posted Last updated 22 December 2025

What this trial tests

Phase 1, PHASE2 trial testing VIC- 1911 in Acute Leukemia in 16 participants. Completed in 30 June 2025.

Timeline

17 March 2022

Primary endpoint
19 September 2024

30 June 2025

Quick facts

Lead sponsor	Masonic Cancer Center, University of Minnesota
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	supportive care
Enrollment	16
Start date	17 March 2022
Primary completion	19 September 2024
Estimated completion	30 June 2025
Sites	1 location across United States

Drugs / interventions tested

• VIC- 1911 — full drug profile →

Conditions studied

• Acute Leukemia — all drugs for Acute Leukemia →
• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →
• Myeloproliferative Neoplasm — all drugs for Myeloproliferative Neoplasm →
• Lymphoma — all drugs for Lymphoma →

Sponsor

Masonic Cancer Center, University of Minnesota

Who can join

18 and older, any sex, with Acute Leukemia or Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Determine the Optimal Dose of VIC-1911 When Given in Combination With Standard Immunosuppressive Therapy in Adult Patients Undergoing Myeloablative Stem Cell Transplantation. Primary · 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.

Group	Value	95% CI
PTCy/Sirolimus Plus VIC-1911	75

Progression-free Survival Primary · 1 Year

Participant progression-free survival assessed using aGVHD data.

Group	Value	95% CI
Dose Level A1	100
Dose Level A2	100
Dose Level A3	100

Relapsed Assessment (Phase I) Primary · 12 months

Assessment to determine if patient has relapse in MTD arm. Data only to reported from arm with maximum tolerated dose.

Group	Value	95% CI
Dose Level A3	0	0 – 0

Overall Survival (OS) Secondary · 1 year

Overall Survival for participants on MTD arm. Data only to reported from arm with maximum tolerated dose.

Group	Value	95% CI
Dose Level A3	100

To Determine the Cumulative Incidences of Acute GVHD Secondary · Day 100

Assessment of aGVHD for MTD arm. Data only to reported from arm with maximum tolerated dose.

Group	Value	95% CI
Dose Level A3	6	2 – 10

To Determine the Cumulative Incidences of Chronic GVHD Secondary · 12 months

Assessment of cGVHD

Group	Value	95% CI
Dose Level A1	0	0 – 0
Dose Level A2	0	0 – 0
Dose Level A3	33	4 – 63

Progression-free Survival Comparing Graft-Versus-Host Disease-Free (GRFS) to the Standard PTCY Plus Tacrolimus/Mycophenolate Mofetil Regimen From MT2015-29 Secondary · 12 months

Progression-free Survival assessed using GRFS defined as grade III-IV acute GVHD, chronic GVHD requiring immunosuppression, relapse, or death by 1 year

Group	Value	95% CI
Dose Level A1	100	0 – 100
Dose Level A2	100	0 – 100
Dose Level A3	67	28 – 88

Progression Free Survival Secondary · 1 Year

Percentage of participants with progression free survival at 1 year for MTD arm. Data only to reported from arm with maximum tolerated dose.

Group	Value	95% CI
Dose Level A3	100

Frequency of CMV Reactivation and Disease Secondary · Day 100

Analyze the frequency of CMV reactivation and disease for MTD arm. Data only to reported from arm with maximum tolerated dose.

Group	Value	95% CI
Dose Level A3	0	0 – 0

Adverse events — posted to ClinicalTrials.gov

Time frame: 1 year. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Dose Level A1

Serious: 1/4 (25%)

Deaths: 1/4

Dose Level A2

Serious: 2/3 (67%)

Deaths: 0/3

Dose Level A3

Serious: 3/9 (33%)

Deaths: 0/9

Serious adverse events (7 terms)

Reaction	System	Dose Level A1	Dose Level A2	Dose Level A3
Upper gastrointestinal hemorrhage	Gastrointestinal disorders	—	—	—
Skin and subcutaneous tissue disorders - Other, specify - Sweet's syndrome (acute febrile neutro	Skin and subcutaneous tissue disorders	—	—	—
Infections and infestations - Other, specify - Histoplasmosis	Infections and infestations	—	—	—
Immune system disorders - Other, specify - Immune Reconstitution Inflammatory Syndrome.	Immune system disorders	—	—	—
Adult respiratory distress syndrome	Respiratory, thoracic and mediastinal disorders	—	—	—
Vascular disorders, hematoma	Vascular disorders	—	—	—
Sepsis	Infections and infestations	—	—	—

Other adverse events (11 terms — click to expand)

Reaction	System	Dose Level A1	Dose Level A2	Dose Level A3
Keratitis	Eye disorders	—	—	—
Febrile Neutropenia	Blood and lymphatic system disorders	—	—	—
Pericarditis	Cardiac disorders	—	—	—
Sepsis	Infections and infestations	—	—	—
Upper gastrointestinal hemorrhage	Gastrointestinal disorders	—	—	—
Infections and infestations - Other, specify (Histoplasmosis)	Infections and infestations	—	—	—
Lung infection	Infections and infestations	—	—	—
Neutrophil count decreased	Investigations	—	—	—
Skin and subcutaneous tissue disorders - Other, specify (skin lesions)	Skin and subcutaneous tissue disorders	—	—	—
Skin and subcutaneous tissue disorders - Other, specify (Sweet's syndrome)	Skin and subcutaneous tissue disorders	—	—	—
Vascular disorders - Other, specify (hematoma)	Vascular disorders	—	—	—

Most-reported serious reactions: Upper gastrointestinal hemorrhage, Skin and subcutaneous tissue disorders - Other, specify - Sweet&#39;s syndrome (acute febrile neutro, Infections and infestations - Other, specify - Histoplasmosis, Immune system disorders - Other, specify - Immune Reconstitution Inflammatory Syndrome., Adult respiratory distress syndrome, Vascular disorders, hematoma, Sepsis.

Data from ClinicalTrials.gov NCT05120570 adverse events section.

Sponsor's own description

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).

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Phase II Study of Myeloablative 7-8/8-Matched Allotransplantation with Post-Transplantation Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil.
   Jurdi NE, Hoover A, O'Leary D, Cao Q, et al · · 2023 · cited 19× · PMID 37311510 · DOI 10.1016/j.jtct.2023.06.008
2. JAK2/mTOR inhibition fails to prevent acute GVHD despite reduced Th1/Th17 cells: final phase 2 trial results.
   Pidala J, Holtan SG, Walton K, Kim J, et al · · 2024 · cited 5× · PMID 39046783 · DOI 10.1182/blood.2024024789
3. Novel approaches to acute graft-versus-host disease prevention.
   Watkins B, Qayed M. · · 2023 · cited 2× · PMID 38066861 · DOI 10.1182/hematology.2023000426
4. Targeting Aurora kinase A to prevent GVHD and relapse after myeloablative allogeneic hematopoietic cell transplantation.
   Holtan SG, Grover P, Walton K, El Jurdi N, et al · · 2026 · PMID 41592279 · DOI 10.1182/bloodadvances.2025017360