Phase 2
Completed N=46
Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant
Graft vs Host Disease
Source: ClinicalTrials.gov NCT03774082 ↗
Enrolled (actual)
46
Serious AEs
32.2%
Results posted
Apr 2025
Primary outcomePrimary: Overall Response Rate (ORR) at Cycle 7 Day 1 — 36.4; 50.0; 28.6; 40.0 Percentage of participants
Summary
This open-label, single-arm, Phase II multi-center study enrolled 46 participants and investigated the activity, pharmacokinetics and safety of ruxolitinib added to the subject's immunosuppressive regimen among infants, children, and adolescents aged ≥28 days to <18 years old with either moderate to severe treatment-naive cGvHD or SR-cGvHD.
Although 46 participants were enrolled,1 participant (enrolled in the ≥6y to <12y age group) received study treatment beyond protocol requirements and was excluded from analyses.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Response Rate (ORR) at Cycle 7 Day 1 |
36.4; 50.0; 28.6; 40.0 | — |
| SECONDARY Ruxolitinib Concentrations by Timepoint |
60.0; 32.4; 78.6; 60.1; 112; 106 | — |
| SECONDARY Duration of Response (DOR) |
94.59; 86.23; 74.24; 63.10; 58.89; 58.89 | — |
| SECONDARY Overall Response Rate (ORR) at Cycle 4 Day 1 |
54.5; 62.5; 42.9; 55.6 | — |
| SECONDARY Best Overall Response (BOR) |
81.8; 81.3; 85.7; 82.2 | — |
| SECONDARY Failure Free Survival (FFS) |
91.11; 84.44; 68.89; 64.44; 57.78; 57.78 | — |
| SECONDARY Cumulative Incidence of Malignancy Relapse/Recurrence (MR) |
3.33; 10.00; 10.00; 10.00; 10.00; 10.00 | — |
| SECONDARY Non-relapse Mortality (NRM) |
2.22; 2.22; 6.67; 13.33; 17.78; 20.07 | — |
| SECONDARY Overall Survival (OS) |
97.78; 97.78; 91.06; 84.23; 79.67; 77.33 | — |
| SECONDARY Percentage of Participants With ≥50% Reduction From Baseline in Daily Corticosteroid Dose |
75.0 | — |
| SECONDARY Percentage of Participants With a Reduction to a Low Dose Corticosteriod |
67.5 | — |
| SECONDARY Graft Failure |
2 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female subjects age ≥28 days and 3 weeks from start of ruxolitinib.
- Failed prior alloSCT within the past 6 months
- Significant respiratory disease including subjects who are on mechanical ventilation or who have a resting oxygen saturation 1 mg/kg/daymethylprednisolone (or equivalent prednisone dose 1.25 mg/kg/day) within 7 days of the screening visit.
- History of progressive multifocal leuko-encephalopathy (PML).
- Presence of severely impaired renal function
Data sourced from ClinicalTrials.gov (NCT03774082). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.