Trial Outcomes & Findings for Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haplo PBSCT (NCT NCT03018223)
NCT ID: NCT03018223
Last Updated: 2021-09-16
Results Overview
Cumulative incidence of grade II-IV acute GVHD by day 100 after HCT. Acute GVHD organ staging and assessment of overall grade will use standard consensus criteria. The cumulative incidence of acute and chronic GVHD will be estimated, considering malignancy relapse and non-relapse death as competing risk events.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
32 participants
Primary outcome timeframe
100 days post hematopoietic cell transplant (HCT)
Results posted on
2021-09-16
Participant Flow
Participant milestones
| Measure |
Conditioning/HCT/GVHD Prophylaxis
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Overall Study
STARTED
|
32
|
|
Overall Study
COMPLETED
|
32
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haplo PBSCT
Baseline characteristics by cohort
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 Participants
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
27 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
49.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
28 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
32 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 100 days post hematopoietic cell transplant (HCT)Cumulative incidence of grade II-IV acute GVHD by day 100 after HCT. Acute GVHD organ staging and assessment of overall grade will use standard consensus criteria. The cumulative incidence of acute and chronic GVHD will be estimated, considering malignancy relapse and non-relapse death as competing risk events.
Outcome measures
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 Participants
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Incidence of Grade II-IV Acute Graft vs. Host Disease (GVHD)
|
18.8 percentage of participants
Interval 7.5 to 34.0
|
SECONDARY outcome
Timeframe: 1 year post HCTCumulative incidence of chronic GVHD by 1 year. Chronic GVHD diagnosis follow National Institutes of Health (NIH) Consensus guidelines.
Outcome measures
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 Participants
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Incidence of Chronic GVHD
|
20.0 percentage of participants
Interval 7.9 to 36.0
|
SECONDARY outcome
Timeframe: Up to 1 year post HCTOverall survival is defined as time from transplant to death or last follow-up, and is reported as percentage of surviving participants.
Outcome measures
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 Participants
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Overall Survival (OS)
|
70.2 percentage of participants
Interval 55.5 to 88.6
|
SECONDARY outcome
Timeframe: Up to 1 year post HCTProgression-free survival defined by the time interval from transplant to relapse/recurrence, to death or to last follow-up. Reported as percentage of participants who are disease free one year post HCT.
Outcome measures
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 Participants
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Progression Free Survival (PFS)
|
56.6 percentage of participants
Interval 41.3 to 77.7
|
Adverse Events
Conditioning/HCT/GVHD Prophylaxis
Serious events: 17 serious events
Other events: 5 other events
Deaths: 5 deaths
Serious adverse events
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 participants at risk
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders -Other
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Cardiac disorders
Cardiac disorders - Other
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Gastrointestinal disorders
Colonic perforation
|
6.2%
2/32 • Number of events 2 • 2 years, 7 months
|
|
Gastrointestinal disorders
Nausea
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Gastrointestinal disorders
Vomiting
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Gastrointestinal disorders
Fever
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Renal and urinary disorders
Portal vein thrombosis
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Immune system disorders
Autoimmune disorder
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Immune system disorders
Immune system disorders - Other
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Infections and infestations
Enterocolitis infectious
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Infections and infestations
Infections and infestations -Other
|
9.4%
3/32 • Number of events 3 • 2 years, 7 months
|
|
Infections and infestations
Lung infection
|
9.4%
3/32 • Number of events 3 • 2 years, 7 months
|
|
Infections and infestations
Sepsis
|
6.2%
2/32 • Number of events 2 • 2 years, 7 months
|
|
Infections and infestations
Urinary tract infection
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Psychiatric disorders
Confusion
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Renal and urinary disorders
Cystitis noninfective
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Renal and urinary disorders
Hematuria
|
15.6%
5/32 • Number of events 5 • 2 years, 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
Vascular disorders
Thromboembolic event
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
Other adverse events
| Measure |
Conditioning/HCT/GVHD Prophylaxis
n=32 participants at risk
Pre-HCT Conditioning, HCT, GVHD Prophylaxis.
|
|---|---|
|
Infections and infestations
Infections and Infestations - Other
|
3.1%
1/32 • Number of events 1 • 2 years, 7 months
|
|
General disorders
Fever
|
12.5%
4/32 • Number of events 4 • 2 years, 7 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place