Trial Outcomes & Findings for PTCy + Sirolimus/VIC-1911 as GVHD Prophylaxis in Myeloablative PBSC Transplantation (NCT NCT05120570)
NCT ID: NCT05120570
Last Updated: 2025-12-22
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
16 participants
Primary outcome timeframe
21 days post treatment
Results posted on
2025-12-22
Participant Flow
No participants were enrolled into the Phase II portion of the study. We concluded the trial when Phase Ia was completed, rather than proceeding to Phase Ib, due to funding constraints and the goal of focusing on developing a new randomized study.
Participant milestones
| Measure |
Dose Level A1
25 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
3
|
9
|
|
Overall Study
COMPLETED
|
3
|
3
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
PTCy + Sirolimus/VIC-1911 as GVHD Prophylaxis in Myeloablative PBSC Transplantation
Baseline characteristics by cohort
| Measure |
Dose Level A1
n=4 Participants
25 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A2
n=3 Participants
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
n=9 Participants
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=18 Participants
|
3 Participants
n=102 Participants
|
8 Participants
n=30 Participants
|
15 Participants
n=37 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
1 Participants
n=30 Participants
|
1 Participants
n=37 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=18 Participants
|
2 Participants
n=102 Participants
|
4 Participants
n=30 Participants
|
6 Participants
n=37 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=18 Participants
|
1 Participants
n=102 Participants
|
5 Participants
n=30 Participants
|
10 Participants
n=37 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=18 Participants
|
3 Participants
n=102 Participants
|
9 Participants
n=30 Participants
|
16 Participants
n=37 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=18 Participants
|
1 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
1 Participants
n=37 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=18 Participants
|
1 Participants
n=102 Participants
|
8 Participants
n=30 Participants
|
13 Participants
n=37 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=18 Participants
|
0 Participants
n=102 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=18 Participants
|
1 Participants
n=102 Participants
|
1 Participants
n=30 Participants
|
2 Participants
n=37 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=18 Participants
|
3 participants
n=102 Participants
|
9 participants
n=30 Participants
|
16 participants
n=37 Participants
|
PRIMARY outcome
Timeframe: 21 days post treatmentThe 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.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=15 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Determine the Optimal Dose of VIC-1911 When Given in Combination With Standard Immunosuppressive Therapy in Adult Patients Undergoing Myeloablative Stem Cell Transplantation.
|
75 mg of VIC
|
—
|
—
|
PRIMARY outcome
Timeframe: 1 YearParticipant progression-free survival assessed using aGVHD data.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=3 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
n=3 Participants
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
n=9 Participants
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Progression-free Survival
|
100 Percentage of participants
|
100 Percentage of participants
|
100 Percentage of participants
|
PRIMARY outcome
Timeframe: 12 monthsAssessment to determine if patient has relapse in MTD arm. Data only to reported from arm with maximum tolerated dose.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=9 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Relapsed Assessment (Phase I)
|
0 Percentage of participants
Interval 0.0 to 0.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 1 yearOverall Survival for participants on MTD arm. Data only to reported from arm with maximum tolerated dose.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=9 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Overall Survival (OS)
|
100 Percent of participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 100Assessment of aGVHD for MTD arm. Data only to reported from arm with maximum tolerated dose.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=9 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
To Determine the Cumulative Incidences of Acute GVHD
|
6 number of new cases per 900 person-days
Interval 2.0 to 10.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 monthsAssessment of cGVHD
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=3 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
n=3 Participants
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
n=9 Participants
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
To Determine the Cumulative Incidences of Chronic GVHD
|
0 Percentage of participants
Interval 0.0 to 0.0
|
0 Percentage of participants
Interval 0.0 to 0.0
|
33 Percentage of participants
Interval 4.0 to 63.0
|
SECONDARY outcome
Timeframe: 12 monthsProgression-free Survival assessed using GRFS defined as grade III-IV acute GVHD, chronic GVHD requiring immunosuppression, relapse, or death by 1 year
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=3 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
n=3 Participants
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
n=9 Participants
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Progression-free Survival Comparing Graft-Versus-Host Disease-Free (GRFS) to the Standard PTCY Plus Tacrolimus/Mycophenolate Mofetil Regimen From MT2015-29
|
100 Percentage of participants
Interval 0.0 to 100.0
|
100 Percentage of participants
Interval 0.0 to 100.0
|
67 Percentage of participants
Interval 28.0 to 88.0
|
SECONDARY outcome
Timeframe: 1 YearPercentage of participants with progression free survival at 1 year for MTD arm. Data only to reported from arm with maximum tolerated dose.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=9 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Progression Free Survival
|
100 Percent of participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 100Analyze the frequency of CMV reactivation and disease for MTD arm. Data only to reported from arm with maximum tolerated dose.
Outcome measures
| Measure |
PTCy/Sirolimus Plus VIC-1911
n=9 Participants
Patients enrolled and treated with PTCy/sirolimus plus VIC-1911
VIC- 1911: 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.
|
Dose Level A2
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Frequency of CMV Reactivation and Disease
|
0 Percentage of participants
Interval 0.0 to 0.0
|
—
|
—
|
Adverse Events
Dose Level A1
Serious events: 1 serious events
Other events: 1 other events
Deaths: 1 deaths
Dose Level A2
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Dose Level A3
Serious events: 3 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Dose Level A1
n=4 participants at risk
25 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A2
n=3 participants at risk
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
n=9 participants at risk
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
0.00%
0/4 • 1 year
|
0.00%
0/3 • 1 year
|
11.1%
1/9 • Number of events 4 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify - Sweet's syndrome (acute febrile neutro
|
0.00%
0/4 • 1 year
|
0.00%
0/3 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Infections and infestations
Infections and infestations - Other, specify - Histoplasmosis
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Immune system disorders
Immune system disorders - Other, specify - Immune Reconstitution Inflammatory Syndrome.
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
25.0%
1/4 • Number of events 2 • 1 year
|
0.00%
0/3 • 1 year
|
0.00%
0/9 • 1 year
|
|
Vascular disorders
Vascular disorders, hematoma
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Sepsis
|
0.00%
0/4 • 1 year
|
0.00%
0/3 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Dose Level A1
n=4 participants at risk
25 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A2
n=3 participants at risk
50 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
Dose Level A3
n=9 participants at risk
75 mg administered twice a day from day 5 post HCT to day 45. Dose escalation will stop once we identify the lowest biologically active and safe dose of VIC.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
0.00%
0/4 • 1 year
|
66.7%
2/3 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
Cardiac disorders
Pericarditis
|
0.00%
0/4 • 1 year
|
0.00%
0/3 • 1 year
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Eye disorders
Keratitis
|
0.00%
0/4 • 1 year
|
100.0%
3/3 • Number of events 3 • 1 year
|
0.00%
0/9 • 1 year
|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
0.00%
0/4 • 1 year
|
0.00%
0/3 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Infections and infestations
Infections and infestations - Other, specify (Histoplasmosis)
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Lung infection
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Sepsis
|
0.00%
0/4 • 1 year
|
66.7%
2/3 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify (skin lesions)
|
25.0%
1/4 • Number of events 1 • 1 year
|
0.00%
0/3 • 1 year
|
0.00%
0/9 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify (Sweet's syndrome)
|
0.00%
0/4 • 1 year
|
0.00%
0/3 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Vascular disorders
Vascular disorders - Other, specify (hematoma)
|
0.00%
0/4 • 1 year
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place