Trial · NCT03774082

Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant

NCT03774082, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2025-04-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant. Trials, record NCT03774082. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03774082

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant
NCT number: NCT03774082
Status: Completed
Phases: Phase 2
Enrollment: 46 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Graft vs Host Disease
Interventions: INC424
First posted: 2018-12-12
Last updated: 2025-12-11
Other registries' IDs: eudract:2018-003296-35
Results first posted: 2025-04-17

Source ClinicalTrials.gov

Registered outcomes12

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Overall Response Rate (ORR) at Cycle 7 Day 1	At Cycle 7 Day 1 (Day 168); Cycle = 28 Days	ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response. The response is assessed per National Institute of Health (NIH) consensus criteria and scoring of response was relative to the organ stage at the start of study treatment.
Secondary	Ruxolitinib Concentrations by Timepoint	Cycle 1 Day 1: 0.5, 2 and 6 hours post-dose; Pre-dose on Cycle 1 Day 8, Cycle 1 Day 15, Cycle 1 Day 22, Cycle 3 Day 1, Cycle 5 Day 1 and Cycle 7 Day 1; Cycle = 28 Days	Pharmacokinetics (PK) of ruxolitinib by age groups (and formulation tablet vs liquid).
Secondary	Duration of Response (DOR)	From baseline up to 39 cycles; Cycle = 28 Days	Time from first response until chronic Graft vs. host disease (cGvHD) progression, death, or the date of addition of systemic therapies for cGvHD assessed for responders only based on BOR up to Cycle 7 Day 1. Presented as percentage of participants to still be in response at different time points in months (per Kaplan-Meier estimates). Participants without event will be censored at the date of their last response assessment prior to or at the analysis cut-off date if no events occurred on or before 12 weeks (84 days) after the last GvHD assessment. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
Secondary	Overall Response Rate (ORR) at Cycle 4 Day 1	At Cycle 4 Day 1 (Day 84); Cycle = 28 Days	ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response. The response is assessed per National Institute of Health (NIH) consensus criteria and scoring of response will be relative to the organ stage at the start of study treatment at Cycle 4 Day 1.
Secondary	Best Overall Response (BOR)	Until Cycle 7 Day 1 (Day 168) or the start of additional systemic therapy for cGvHD; Cycle = 28 Days	Percentage of participants who achieved overall response (complete response (CR) or partial response (PR)) at any time point until Cycle 7 Day 1 or the start of additional systemic therapy for chronic GvHD.
Secondary	Failure Free Survival (FFS)	From baseline up to 39 cycles; Cycle = 28 Days	Failure-free survival was defined as the time from date of treatment to any of the following events: i) relapse or recurrence of underlying disease or death due to underlying disease, ii) non-relapse mortality, or iii) addition or initiation of another systemic therapy for cGvHD per Kaplan-Meier estimates. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
Secondary	Cumulative Incidence of Malignancy Relapse/Recurrence (MR)	From baseline up to 39 cycles; Cycle = 28 Days	MR was defined as the time from date of treatment assignment to the date of hematologic malignancy relapse/recurrence. Calculated for subjects with underlying hematologic malignant disease. The cumulative incidence (CI) of malignancy relapse/recurrence at 1, 2, 6, 12, 18, 24, 30 and 36 months after start of treatment has been reported. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
Secondary	Non-relapse Mortality (NRM)	From baseline up to 39 cycles; Cycle = 28 Days	NRM is defined as the time from date of treatment assignment to date of death not preceded by underlying disease relapse/recurrence calculated for all participants. The cumulative incidence (CI) of non-relapse mortality at 1, 2, 6, 12, 18, 24, 30 and 36 months after start of treatment has been reported. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
Secondary	Overall Survival (OS)	From baseline up to 39 cycles; Cycle = 28 Days	OS is defined as the time from the date of treatment assignment to the date of death due to any cause. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
Secondary	Percentage of Participants With ≥50% Reduction From Baseline in Daily Corticosteroid Dose	Baseline to Cycle 7 Day 1 (Day 168); Cycle = 28 Days	Reduction of at least ≥50% from baseline in daily corticosteroid use by Cycle 7 Day 1 (regardless of reason). As planned in the SAP, this outcome measure is provided for all participants instead of per age groups, for those who received corticosteroids at baseline.

1 to 10 of 12Source ClinicalTrials.gov

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Outcomes12

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Overall Response Rate (ORR) at Cycle 7 Day 1	At Cycle 7 Day 1 (Day 168); Cycle = 28 Days	Full Analysis Set (FAS) comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.	Percentage of participants	NUMBER
SECONDARY	Ruxolitinib Concentrations by Timepoint	Cycle 1 Day 1: 0.5, 2 and 6 hours post-dose; Pre-dose on Cycle 1 Day 8, Cycle 1 Day 15, Cycle 1 Day 22, Cycle 3 Day 1, Cycle 5 Day 1 and Cycle 7 Day 1; Cycle = 28 Days	The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. Evaluable PK concentration: Participants took the dose of ruxolitinib prior to PK sample; For post-dose samples on Cycle 1 Day 1: participants did not vomit within 2 hours after dosing with ruxolitinib; For pre-dose samples: participant samples were collected before the next dose administration of ruxolitinib.	ug/ml	GEOMETRIC_MEAN
SECONDARY	Duration of Response (DOR)	From baseline up to 39 cycles; Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment. Duration of response (DOR) is assessed for responders only.	Percentage of participants	NUMBER
SECONDARY	Overall Response Rate (ORR) at Cycle 4 Day 1	At Cycle 4 Day 1 (Day 84); Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.	Percentage of participants	NUMBER
SECONDARY	Best Overall Response (BOR)	Until Cycle 7 Day 1 (Day 168) or the start of additional systemic therapy for cGvHD; Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.	Percentage of participants	NUMBER
SECONDARY	Failure Free Survival (FFS)	From baseline up to 39 cycles; Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.	Percentage of participants	NUMBER
SECONDARY	Cumulative Incidence of Malignancy Relapse/Recurrence (MR)	From baseline up to 39 cycles; Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment, assessed for subjects with malignancy relapse/occurrence only.	Percentage of participants	NUMBER
SECONDARY	Non-relapse Mortality (NRM)	From baseline up to 39 cycles; Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.	Percentage of participants	NUMBER
SECONDARY	Overall Survival (OS)	From baseline up to 39 cycles; Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.	survival probability in percentage	NUMBER
SECONDARY	Percentage of Participants With ≥50% Reduction From Baseline in Daily Corticosteroid Dose	Baseline to Cycle 7 Day 1 (Day 168); Cycle = 28 Days	FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment and received corticosteroids at baseline.	Percentage of participants	NUMBER

1 to 10 of 12Source ClinicalTrials.gov

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Results by group91

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Overall Response Rate (ORR) at Cycle 7 Day 1	≥ 12y - < 18y RUX 10mg BID (Group 1)		36.4	90% Confidence Interval 19.6 to 56.1	Percentage of participants
Overall Response Rate (ORR) at Cycle 7 Day 1	≥ 6y - < 12y RUX 5mg BID (Group 2)		50.0	90% Confidence Interval 27.9 to 72.1	Percentage of participants
Overall Response Rate (ORR) at Cycle 7 Day 1	≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)		28.6	90% Confidence Interval 5.3 to 65.9	Percentage of participants
Overall Response Rate (ORR) at Cycle 7 Day 1	All Participants		40.0	90% Confidence Interval 27.7 to 53.3	Percentage of participants
Ruxolitinib Concentrations by Timepoint	≥ 12y - < 18y RUX 10mg BID (Tablet)	Cycle 1 Day 1: 0.5 hour (hr) (post-dose)	60.0	Geometric Coefficient of Variation 350.7	ug/ml
Ruxolitinib Concentrations by Timepoint	≥ 6y - < 12y RUX 5mg BID (Tablet)	Cycle 1 Day 1: 0.5 hour (hr) (post-dose)	32.4	Geometric Coefficient of Variation 471.9	ug/ml
Ruxolitinib Concentrations by Timepoint	≥ 6y - < 12y RUX 5mg BID (Liquid)	Cycle 1 Day 1: 0.5 hour (hr) (post-dose)	78.6		ug/ml
Ruxolitinib Concentrations by Timepoint	≥ 2y - < 6y RUX 4mg/m2 BID (Liquid)	Cycle 1 Day 1: 0.5 hour (hr) (post-dose)	60.1	Geometric Coefficient of Variation 98.7	ug/ml
Ruxolitinib Concentrations by Timepoint	≥ 12y - < 18y RUX 10mg BID (Tablet)	Cycle 1 Day 1: 2 hr (post-dose)	112	Geometric Coefficient of Variation 51.8	ug/ml
Ruxolitinib Concentrations by Timepoint	≥ 6y - < 12y RUX 5mg BID (Tablet)	Cycle 1 Day 1: 2 hr (post-dose)	106	Geometric Coefficient of Variation 39.2	ug/ml

1 to 10 of 91Source ClinicalTrials.gov

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Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	≥ 12y - < 18y RUX 10mg BID (Group 1)	22
Overall Study	STARTED	≥ 6y - < 12y RUX 5mg BID (Group 2)	17
Overall Study	STARTED	≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)	7
Overall Study	Full Analysis Set	≥ 12y - < 18y RUX 10mg BID (Group 1)	22
Overall Study	Full Analysis Set	≥ 6y - < 12y RUX 5mg BID (Group 2)	16
Overall Study	Full Analysis Set	≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)	7
Overall Study	Completed Treatment	≥ 12y - < 18y RUX 10mg BID (Group 1)	3
Overall Study	Completed Treatment	≥ 6y - < 12y RUX 5mg BID (Group 2)	5
Overall Study	Completed Treatment	≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)	3
Overall Study	Discontinued From Treatment	≥ 12y - < 18y RUX 10mg BID (Group 1)	19

1 to 10 of 24Source ClinicalTrials.gov

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Adverse events1,448

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	On-Treatment >=12y - <18y RUX 10mg BID	7	22
other	Anaemia	Blood and lymphatic system disorders	On-Treatment >=6y - <12y RUX 5mg BID	2	16
other	Anaemia	Blood and lymphatic system disorders	On-Treatment >=2y - <6y RUX 4mg/m2 BID	2	7
other	Anaemia	Blood and lymphatic system disorders	On-Treatment - All Participants	11	45
other	Anaemia	Blood and lymphatic system disorders	Post-Treatment >=12y - <18y RUX 10mg BID	0	22
other	Anaemia	Blood and lymphatic system disorders	Post-Treatment >=6y - <12y RUX 5mg BID	0	16
other	Anaemia	Blood and lymphatic system disorders	Post-Treatment >=2y - <6y RUX 4mg/m2 BID	0	7
other	Anaemia	Blood and lymphatic system disorders	Post-Treatment All Participants	0	45
other	Coagulopathy	Blood and lymphatic system disorders	On-Treatment >=12y - <18y RUX 10mg BID	0	22
other	Coagulopathy	Blood and lymphatic system disorders	On-Treatment >=6y - <12y RUX 5mg BID	1	16

1 to 10 of 1,448Source ClinicalTrials.gov

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Adverse event totals24

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	On-Treatment >=12y - <18y RUX 10mg BID	0	22
Total, all-cause mortality	On-Treatment >=6y - <12y RUX 5mg BID	2	16
Total, all-cause mortality	On-Treatment >=2y - <6y RUX 4mg/m2 BID	1	7
Total, all-cause mortality	On-Treatment - All Participants	3	45
Total, all-cause mortality	Post-Treatment >=12y - <18y RUX 10mg BID	6	22
Total, all-cause mortality	Post-Treatment >=6y - <12y RUX 5mg BID	1	16
Total, all-cause mortality	Post-Treatment >=2y - <6y RUX 4mg/m2 BID	1	7
Total, all-cause mortality	Post-Treatment All Participants	8	45
Total, other adverse events	On-Treatment >=12y - <18y RUX 10mg BID	22	22
Total, other adverse events	On-Treatment >=6y - <12y RUX 5mg BID	15	16

1 to 10 of 24Source ClinicalTrials.gov

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Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
39002551	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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