Trial Outcomes & Findings for Safety and Efficacy of Itacitinib in Combination With Corticosteroids for Treatment of Graft-Versus-Host Disease in Pediatric Subjects (NCT NCT03721965)
NCT ID: NCT03721965
Last Updated: 2025-11-04
Results Overview
A TEAE was defined as an AE that began or worsened from Baseline after the first administration of study drug.
TERMINATED
PHASE1/PHASE2
2 participants
up to 45 days
2025-11-04
Participant Flow
A study of itacitinib in combination with corticosteroids for the treatment of Grades II to IV acute graft-versus-host disease (aGVHD) in pediatric participants 28 days to \< 18 years old who were naïve to corticosteroids. The study was conducted from 31-Dec-2019 to 17-Feb-2020.
A total of 2 participants were screened and included in the study.
Participant milestones
| Measure |
Cohort 1 : Itacitinib + Corticosteroids
Itacitinib was administered once a day orally in combination with corticosteroids as per local treatment guidelines.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Cohort 1 : Itacitinib + Corticosteroids
Itacitinib was administered once a day orally in combination with corticosteroids as per local treatment guidelines.
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Study Terminated by Sponsor
|
1
|
Baseline Characteristics
Safety and Efficacy of Itacitinib in Combination With Corticosteroids for Treatment of Graft-Versus-Host Disease in Pediatric Subjects
Baseline characteristics by cohort
| Measure |
Cohort 1 : Itacitinib + Corticosteroids
n=2 Participants
Itacitinib was administered once a day orally in combination with corticosteroids as per local treatment guidelines.
|
|---|---|
|
Race/Ethnicity, Customized
White
|
2 Participants
n=15 Participants
|
|
Age, Categorical
<=18 years
|
2 Participants
n=15 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=15 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=15 Participants
|
|
Age, Continuous
|
13.5 years
STANDARD_DEVIATION 2.12 • n=15 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=15 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=15 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
2 Participants
n=15 Participants
|
PRIMARY outcome
Timeframe: up to 45 daysPopulation: All enrolled participants
A TEAE was defined as an AE that began or worsened from Baseline after the first administration of study drug.
Outcome measures
| Measure |
Cohort 1 : Itacitinib + Corticosteroids
n=2 Participants
Itacitinib was administered once a day orally in combination with corticosteroids as per local treatment guidelines.
|
|---|---|
|
Phase 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
2 Participants
|
PRIMARY outcome
Timeframe: Day 1: 1, 2, 4, and 6 hours post-dose. Day 7: predose; 1, 2, 4, and 6 hours post-dosePopulation: Due to the small sample size (n=2), data cannot be reported without risking participant re-identification. Data are not being reported in order to protect participants' privacy.
Cmax was defined as the maximum observed plasma concentration.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Day 1: 1, 2, 4, and 6 hours post-dose. Day 7: predose; 1, 2, 4, and 6 hours post-dosePopulation: Due to the small sample size (n=2), data cannot be reported without risking participant re-identification. Data are not being reported in order to protect participants' privacy.
Cmin was defined as the minimum observed plasma concentration.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Day 1: 1, 2, 4, and 6 hours post-dose. Day 7: predose; 1, 2, 4, and 6 hours post-dosePopulation: Due to the small sample size (n=2), data cannot be reported without risking participant re-identification. Data are not being reported in order to protect participants' privacy.
Tmax was defined as the time to the maximum concentration.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Day 1: 1, 2, 4, and 6 hours post-dose. Day 7: predose; 1, 2, 4, and 6 hours post-dosePopulation: Due to the small sample size (n=2), data cannot be reported without risking participant re-identification. Data are not being reported in order to protect participants' privacy.
AUC was defined as the area under the plasma concentration-time curve.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Day 1: 1, 2, 4, and 6 hours post-dose. Day 7: predose; 1, 2, 4, and 6 hours post-dosePopulation: Due to the small sample size (n=2), data cannot be reported without risking participant re-identification. Data are not being reported in order to protect participants' privacy.
Cl/F was defined as the apparent oral dose clearance.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Day 28Population: No participants enrolled in Phase 2 due to early termination of the study.
Overall response rate was defined as the number of participants demonstrating a complete response (CR), a very good partial response (VGPR), or a partial response (PR).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
A TEAE was defined as an AE that began or worsened from Baseline after the first administration of study drug.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 7: predose; 1, 2, and 4 hours post-dosePopulation: No participants enrolled in Phase 2 due to early termination of the study.
Cmax was defined as the maximum observed plasma concentration.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 7: predose; 1, 2, and 4 hours post-dosePopulation: No participants enrolled in Phase 2 due to early termination of the study.
Cmin was defined as the minimum observed plasma concentration.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 7: predose; 1, 2, and 4 hours post-dosePopulation: No participants enrolled in Phase 2 due to early termination of the study.
Tmax was defined as the time to the maximum concentration.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 7: predose; 1, 2, and 4 hours post-dosePopulation: No participants enrolled in Phase 2 due to early termination of the study.
AUC was defined as the area under the plasma concentration-time curve.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 7: predose; 1, 2, and 4 hours post-dosePopulation: No participants enrolled in Phase 2 due to early termination of the study.
Cl/F was defined as the apparent oral dose clearance.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 100 daysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Overall response rate was defined as the number of participants demonstrating a CR, a VGPR, or a PR.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to Day 28Population: The study was terminated before participants reached Day 28, the time point for analysis.
Overall response rate was defined as the number of participants demonstrating a CR, a VGPR, or a PR.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 24 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Non relapse mortality was defined as the number of participants who died due to causes other than underlying hematologic disorders relapse.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to approximately 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Duration of response was defined as the time of the onset of response to the loss of response.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to approximately 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Time to response was defined as the interval from treatment initiation to the first response.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to approximately 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Relapse rate was defined as the number of participants whose underlying disease relapsed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to approximately 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Mortality rate was defined as the number of participants whose underlying hematologic disorder relapsed and had a fatal outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 6 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Failure-free survival was defined as the number of participants who were still alive, had not relapsed, had not required additional therapy for acute graft-versus-host disease (aGVHD), and had not demonstrated signs or symptoms of chronic GVHD.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to approximately 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Overall survival was defined as the interval from study enrollment to death due to any cause.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to approximately 12 monthsPopulation: No participants enrolled in Phase 2 due to early termination of the study.
Analysis was to be conducted to assess the number of participants experiencing secondary graft failure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 180 daysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
The average number of participants who discontinued corticosteroids was to be assessed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 180 daysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
The number of participants with various cumulative corticosteroid doses was assessed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 100 daysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
The number of participants who discontinued corticosteroids was assessed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 100 daysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
The number of participants who discontinued immunosuppressive medication was assessed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 100 DaysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
The number of participants who experienced aGVHD flares requiring treatment was assessed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 365 daysPopulation: No participants enrolled in Phase 2 due to early termination of the study.
The number of participants with a diagnosis of any cGVHD, including mild, moderate, severe, was assessed.
Outcome measures
Outcome data not reported
Adverse Events
Cohort 1 : Itacitinib + Corticosteroids
Serious adverse events
| Measure |
Cohort 1 : Itacitinib + Corticosteroids
n=2 participants at risk
Itacitinib was administered once a day orally in combination with corticosteroids as per local treatment guidelines.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Cardiac disorders
Cardiac failure congestive
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Infections and infestations
Parvovirus infection
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Respiratory, thoracic and mediastinal disorders
Idiopathic pneumonia syndrome
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
Other adverse events
| Measure |
Cohort 1 : Itacitinib + Corticosteroids
n=2 participants at risk
Itacitinib was administered once a day orally in combination with corticosteroids as per local treatment guidelines.
|
|---|---|
|
Psychiatric disorders
Agitation
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Renal and urinary disorders
Dysuria
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Renal and urinary disorders
Micturition urgency
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Renal and urinary disorders
Pollakiuria
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Vascular disorders
Hypertension
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Blood and lymphatic system disorders
Neutropenia
|
50.0%
1/2 • Number of events 2 • up to 45 days
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
General disorders
Oedema peripheral
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Investigations
Ejection fraction decreased
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Investigations
Neutrophil count decreased
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Investigations
White blood cell count decreased
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
50.0%
1/2 • Number of events 2 • up to 45 days
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
50.0%
1/2 • Number of events 2 • up to 45 days
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
|
Nervous system disorders
Headache
|
50.0%
1/2 • Number of events 1 • up to 45 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Clinical Study Agreement
- Publication restrictions are in place
Restriction type: OTHER