Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis
Public ClinicalTrials.gov record NCT04551131. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone, and etoposide as Frontline therapy for patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH) or as Salvage therapy for patients with relapsed/refractory HLH. Primary Objective * To determine the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with newly diagnosed HLH. Secondary Objectives * To describe the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with relapsed/refractory HLH. * To describe the overall response and outcome for patients with newly diagnosed or relapsed/refractory HLH who are treated with this response-adapted ruxolitinib-containing regimen. Exploratory Objectives * To estimate the pharmacokinetic (PK) parameters of ruxolitinib, assess covariates of ruxolitinib pharmacokinetics, and test whether the drug's effectiveness is correlated with systemic drug exposure. * To query specific immunologic biomarkers and determine whether the levels of these biomarkers correlate with disease response and outcome.
Study identification
- NCT ID
- NCT04551131
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 10 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Weeks to 22 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 13, 2021 (Actual)
- Primary completion
- Nov 7, 2025 (Actual)
- Completion
- Aug 2026 (Estimated)
- Last update posted
- Apr 24, 2026
2021 – 2026
United States locations
- U.S. sites
- 13
- U.S. states
- 11
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| Children's Hospital of Orange County | Orange | California | 92868 | — |
| University of California San Francisco | San Francisco | California | 94158 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| John Hopkins University | Baltimore | Maryland | 21287 | — |
| Dana-Farber Cancer Institute | Boston | Massachusetts | 02215 | — |
| Cohen Children's Medical Center | New Hyde Park | New York | 11040 | — |
| Levine Children's Hospital | Charlotte | North Carolina | 28203 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75235 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| Children's Wisconsin/Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04551131, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 24, 2026 · Synced Oct 29, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04551131 live on ClinicalTrials.gov.