Trial · NCT03491215

Study of Pharmacokinetics, Activity and Safety of Ruxolitinib in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease

NCT03491215, Phase 1, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2024-11-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Pharmacokinetics, Activity and Safety of Ruxolitinib in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease. Trials, record NCT03491215. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03491215

On this page

Trial

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

Title: Study of Pharmacokinetics, Activity and Safety of Ruxolitinib in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease
NCT number: NCT03491215
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 45 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Acute Graft Versus Host Disease
Interventions: Ruxolitinib
First posted: 2018-04-09
Last updated: 2025-05-04
Other registries' IDs: eudract:2018-000422-55
Results first posted: 2024-11-08

Source ClinicalTrials.gov

Registered outcomes32

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

List	Outcome	Time frame	Description
Primary	Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 sparse sampling in Group 4. AUClast: The AUC from time zero to the last measurable concentration sampling time (Tlast).
Primary	Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 and sparse sampling in Group 4. Cmax: The maximum (peak) observed plasma drug concentration
Primary	Phase I: Measurement of PK Parameter, T1/2, in aGvHD and SR-aGvHD Patients	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 and sparse sampling in Group 4. T1/2: The elimination half-life associated with the terminal slope (Lambda\_z ) of a semi logarithmic concentration-time curve
Primary	Phase I: Measurement of PK Parameter, Ctrough, in aGvHD and SR-aGvHD Patients	Day 7 at pre-dose	Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 and sparse sampling in Group 4. Ctrough: The minimum observed plasma concentration at the end of an administration interval (corresponding to the pre-dose concentration prior to the following administration).
Primary	Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using AUClast	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	Phase I: Age-based determination of RP2D in Groups 2 and 3 and was based on observed PK parameters in Groups 1-3 * Group 2: age ≥ 6 to \< 12 years * Group 3: age ≥ 2 to \< 6 years AUClast: The AUC from time zero to the last measurable concentration sampling time (Tlast).
Primary	Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using Cmax	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	Phase I: Age-based determination of RP2D in Groups 2 and 3 and was based on observed PK parameters in Groups 1-3 * Group 2: age ≥ 6 to \< 12 years * Group 3: age ≥ 2 to \< 6 years Cmax: The maximum (peak) observed plasma drug concentration.
Primary	Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using Ctrough	Day 7 at pre-dose	Phase I: Age-based determination of RP2D in Groups 2 and 3 and was based on observed PK parameters in Groups 1-3 * Group 2: age ≥ 6 to \< 12 years * Group 3: age ≥ 2 to \< 6 years Ctrough: The minimum observed plasma concentration at the end of an administration interval (corresponding to the pre-dose concentration prior to the following administration).
Primary	Phase II: Overall Response Rate (ORR)	Day 28	Phase II: ORR is defined as the percentage of patients demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, mixed response or non-response. Scoring of response was relative to the organ stage at the start of the study treatment. Complete response (CR) is defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of aGvHD in all evaluable organs without administration of additional systemic therapy for any earlier progression, mixed response or non-response of aGvHD. Partial response (PR) is defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapy for an earlier progression, mixed response or non-response of aGvHD.
Secondary	Percentage of All Patients Who Achieved a Complete Response (CR) or Partial Response (PR) (Durable Overall Response Rate (ORR))	Day 56	Durable ORR at Day 56 was defined as the percentage of all subjects who achieved a complete response (CR) or partial response (PR) at Day 28 and maintained a CR or PR at Day 56. Complete-response was defined as a score of 0 for the acute GvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of acute GvHD in all evaluable organs without administration of additional systemic therapies for any earlier progression, mixed response or non-response of acute GvHD. Partial response was defined as improvement of 1 stage in 1 or more organs involved with acute GvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapies for an earlier progression, mixed response or non-response of acute GvHD.
Secondary	Percentage of Patients Who Achieved OR (CR+PR) at Day 14	Day 14	ORR at Day 14 was defined as the percentage of participants with complete response (CR) or partial response (PR ) at Day 14 according to standard criteria. Complete response (CR) is defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of aGvHD in all evaluable organs without administration of additional systemic therapy for any earlier progression, mixed response or non-response of aGvHD. Partial response (PR) is defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapy for an earlier progression, mixed response or non-response of aGvHD.

1 to 10 of 32Source ClinicalTrials.gov

See all 32 registered outcomes

Outcomes33

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, to not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	ng*hr/mL	GEOMETRIC_MEAN
PRIMARY	Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, to not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	ng/ML	GEOMETRIC_MEAN
PRIMARY	Phase I: Measurement of PK Parameter, T1/2, in aGvHD and SR-aGvHD Patients	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, to not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	hour	GEOMETRIC_MEAN
PRIMARY	Phase I: Measurement of PK Parameter, Ctrough, in aGvHD and SR-aGvHD Patients	Day 7 at pre-dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	ng/ml	GEOMETRIC_MEAN
PRIMARY	Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using AUClast	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	h*ng/mL	GEOMETRIC_MEAN
PRIMARY	Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using Cmax	Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	ng/mL	GEOMETRIC_MEAN
PRIMARY	Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using Ctrough	Day 7 at pre-dose	PAS: The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. For a concentration to be evaluable, subjects were required to:~* Take the dose of ruxolitinib prior to PK sample.~* For pre-dose samples, not vomit within 2 hours after the previous dosing of ruxolitinib prior to sampling; for post-dose samples, to not vomit within 2 hours after the dosing of ruxolitinib.~No subjects were enrolled in Group 4.	ng/ml	GEOMETRIC_MEAN
PRIMARY	Phase II: Overall Response Rate (ORR)	Day 28	The Efficacy Evaluable Set (EES) comprised all subjects to whom study treatment was assigned at the recommended phase 2 dose (RP2D) of ruxolitinib and who received at least one dose of study treatment at that dose level. No subjects were enrolled in Group 4.	Percentage of participants	NUMBER
SECONDARY	Percentage of All Patients Who Achieved a Complete Response (CR) or Partial Response (PR) (Durable Overall Response Rate (ORR))	Day 56	The Efficacy Evaluable Set (EES) comprised all subjects to whom study treatment was assigned at the RP2D of ruxolitinib and who received at least one dose of study treatment at that dose level. No subjects were enrolled in Group 4.	Percentage of participants	NUMBER
SECONDARY	Percentage of Patients Who Achieved OR (CR+PR) at Day 14	Day 14	The Efficacy Evaluable Set (EES) comprised all subjects to whom study treatment was assigned at the RP2D of ruxolitinib and who received at least one dose of study treatment at that dose level. No subjects were enrolled in Group 4.	Percentage of participants	NUMBER

1 to 10 of 33Source ClinicalTrials.gov

See all 33 outcomes

Results by group340

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

Outcome	Group	Value	Spread	Units
Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg BID Tablet	252	Geometric Coefficient of Variation 186.6	ng*hr/mL
Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID Tablet	372	Geometric Coefficient of Variation 58.6	ng*hr/mL
Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID Capsule	154	Geometric Coefficient of Variation 58.1	ng*hr/mL
Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m^2 BID Capsule	239	Geometric Coefficient of Variation 65.3	ng*hr/mL
Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m^2 BID Liquid	259	Geometric Coefficient of Variation 53.6	ng*hr/mL
Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg BID Tablet	66.1	Geometric Coefficient of Variation 169.8	ng/ML
Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID Tablet	105	Geometric Coefficient of Variation 71.4	ng/ML
Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID Capsule	49.4	Geometric Coefficient of Variation 45.7	ng/ML
Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m^2 BID Capsule	61.2	Geometric Coefficient of Variation 81.1	ng/ML
Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m^2 BID Liquid	66.5	Geometric Coefficient of Variation 60.8	ng/ML

1 to 10 of 340Source ClinicalTrials.gov

See all 340 results by group

Participant flow24

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg BID	18
Overall Study	STARTED	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID	12
Overall Study	STARTED	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m^2 BID	15
Overall Study	STARTED	Group 4: Subjects >= 28 Days to < 2y	0
Overall Study	Rux Formulation - Received Tablet	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg BID	18
Overall Study	Rux Formulation - Received Tablet	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID	10
Overall Study	Rux Formulation - Received Tablet	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m^2 BID	0
Overall Study	Rux Formulation - Received Tablet	Group 4: Subjects >= 28 Days to < 2y	0
Overall Study	Rux Formulation - Received Capsule	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg BID	0
Overall Study	Rux Formulation - Received Capsule	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg BID	2

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events1,696

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	Group 1: Subjects >=12y - <18y - RUX 10mg (On-treatment)	6	18
other	Anaemia	Blood and lymphatic system disorders	Group 2: Subjects >=6y - <12y - RUX 5mg (On-treatment)	5	12
other	Anaemia	Blood and lymphatic system disorders	Group 3: Subjects >=2y - <6y - RUX 4mg/m2 (On-treatment)	9	15
other	Anaemia	Blood and lymphatic system disorders	All Subjects	20	45
other	Anaemia	Blood and lymphatic system disorders	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg (Post-treatment Survival Follow-up)		0
other	Anaemia	Blood and lymphatic system disorders	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg (Post-treatment Survival Follow-up)		0
other	Anaemia	Blood and lymphatic system disorders	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m2 (Post-treatment Survival Follow-up)		0
other	Anaemia	Blood and lymphatic system disorders	Group 4: Subjects >= 28 Days to < 2y		0
other	Coagulopathy	Blood and lymphatic system disorders	Group 1: Subjects >=12y - <18y - RUX 10mg (On-treatment)	1	18
other	Coagulopathy	Blood and lymphatic system disorders	Group 2: Subjects >=6y - <12y - RUX 5mg (On-treatment)	0	12

1 to 10 of 1,696Source ClinicalTrials.gov

See all 1,696 adverse events

Adverse event totals24

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Group 1: Subjects >=12y - <18y - RUX 10mg (On-treatment)	0	18
Total, all-cause mortality	Group 2: Subjects >=6y - <12y - RUX 5mg (On-treatment)	0	12
Total, all-cause mortality	Group 3: Subjects >=2y - <6y - RUX 4mg/m2 (On-treatment)	0	15
Total, all-cause mortality	All Subjects	0	45
Total, all-cause mortality	Group 1: Subjects ≥ 12y to < 18y - RUX 10mg (Post-treatment Survival Follow-up)	6	18
Total, all-cause mortality	Group 2: Subjects ≥ 6y to < 12y - RUX 5mg (Post-treatment Survival Follow-up)	2	12
Total, all-cause mortality	Group 3: Subjects ≥ 2y to < 6y - RUX 4mg/m2 (Post-treatment Survival Follow-up)	1	15
Total, all-cause mortality	Group 4: Subjects >= 28 Days to < 2y	0	0
Total, other adverse events	Group 1: Subjects >=12y - <18y - RUX 10mg (On-treatment)	18	18
Total, other adverse events	Group 2: Subjects >=6y - <12y - RUX 5mg (On-treatment)	12	12

1 to 10 of 24Source ClinicalTrials.gov

See all 24 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
39046767	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.

GET https://conductscience.org/api/trials/v1/NCT03491215

GET https://conductscience.org/api/trials/v1/NCT03491215/registered?page=2

API reference For AI assistants