← Back to Search

Corticosteroid

Ruxolitinib-Based Regimen for Hemophagocytic Lymphohistiocytosis

Phase 1 & 2
Recruiting
Research Sponsored by St. Jude Children's Research Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patient or parent is willing to have the NG tube placed if patient is unable to take medications PO.
- Patient has known fHLH (e.g., patient has pathogenic/likely pathogenic germline variant(s) in genes such as PRF1, UNC13D, STX11, STXBP2, LYST, RAB27A, XIAP, SH2D1A, NLCR4) and meets ≥4 of the diagnostic HLH criteria listed below, OR
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 1 year
Awards & highlights

Study Summary

This trial is testing a new treatment for hemophagocytic lymphohistiocytosis (HLH) that combines a drug called ruxolitinib with dexamethasone and etoposide. The goal is to see if this new treatment is more effective and has fewer side effects than the standard treatment.

Who is the study for?
This trial is for patients aged 6 weeks to 22 years with newly diagnosed or relapsed/refractory Hemophagocytic Lymphohistiocytosis (HLH). They must weigh at least 3 kg and be able to take oral medication or agree to an NG tube. Participants should meet specific HLH diagnostic criteria but not have received certain prior treatments, except steroids or anakinra. Pregnant individuals, those with severe organ dysfunction, active malignancy, or on other investigational drugs are excluded.Check my eligibility
What is being tested?
The study tests a regimen combining Ruxolitinib with Dexamethasone and Etoposide in two scenarios: as initial treatment for new HLH cases (Frontline therapy) and as secondary treatment when previous therapies failed (Salvage therapy). It's a Phase Ib/II trial aiming to assess the efficacy and tolerability of this combination while also exploring pharmacokinetics and immunologic biomarkers.See study design
What are the potential side effects?
Potential side effects may include immune system suppression leading to increased infection risk, liver function abnormalities, blood cell count changes causing fatigue or bruising easily, digestive issues from dexamethasone like stomach ulcers or appetite changes, etoposide might cause hair loss or mouth sores.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am willing to use a feeding tube if I can't take medicine by mouth.
Select...
I have a genetic form of HLH and meet at least 4 of its diagnostic criteria.
Select...
I have worsening brain symptoms or new abnormal brain scan results, or my spinal fluid tests show signs of inflammation.
Select...
I have not had HLH treatment, except possibly steroids or anakinra.
Select...
I likely have fHLH based on specific test results and meet most of the HLH diagnostic criteria.
Select...
I have a history of HLH and meet the required diagnostic criteria.
Select...
My blood tests show low neutrophils or platelets, or I've needed platelet transfusions.
Select...
My previous cancer treatment did not work as expected.
Select...
I currently have a fever.
Select...
I currently have active HLH with at least 3 symptoms of relapsed/refractory HLH.
Select...
I am eligible for the initial treatment phase.
Select...
I have received at least one dose of ATG and it's been over a week since my last dose.
Select...
I have previously undergone treatment specifically for HLH.
Select...
I have a fever.
Select...
I am between 6 weeks and 22 years old.
Select...
I weigh more than 3 kilograms.
Select...
I can take medicine by mouth or am willing to use a tube if I can't.
Select...
I have been diagnosed with active HLH.
Select...
I am between 6 weeks and 22 years old.
Select...
I weigh more than 3 kilograms.
Select...
I am part of the salvage treatment group.
Select...
My spleen is enlarged and getting worse.
Select...
I have had an enlarged spleen before starting the study drug.
Select...
I've been treated with steroids and etoposide before starting ruxolitinib.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Adverse events (AEs) associated with the ruxolitinib-containing regimen
Complete Response (CR)/Complete Response with Incomplete Hematologic Recovery (CRi)
Secondary outcome measures
Overall Response (CR/CRi plus Partial Response [PR]))
Survival one year after HSCT
Survival to allogeneic hematopoietic stem cell transplantation (HSCT) in patients for whom an allogeneic HSCT is planned
+3 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Salvage ArmExperimental Treatment3 Interventions
Patients with relapsed/refractory HLH will receive ruxolitinib PO or NGT and dexamethasone PO or IV. Etoposide IV will be added based on disease response.
Group II: Frontline ArmExperimental Treatment3 Interventions
Safety Phase: Patients with newly diagnosed HLH will receive ruxolitinib PO or NGT and dexamethasone, PO or IV. Etoposide IV will be added based on disease response. Expansion Phase: Patients with newly diagnosed HLH treatment will begin with ruxolitinib PO or NGT at the MTD dose. Dexamethasone will be administered PO or IV. Etoposide IV will be added based on disease response.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ruxolitinib
2018
Completed Phase 3
~1140
Dexamethasone
2007
Completed Phase 4
~2590
Etoposide
2010
Completed Phase 3
~2440

Find a Location

Who is running the clinical trial?

North American Consortium for HistiocytosisOTHER
4 Previous Clinical Trials
1,715 Total Patients Enrolled
St. Jude Children's Research HospitalLead Sponsor
427 Previous Clinical Trials
5,306,515 Total Patients Enrolled
Cures Within ReachOTHER
19 Previous Clinical Trials
1,681 Total Patients Enrolled

Media Library

Dexamethasone (Corticosteroid) Clinical Trial Eligibility Overview. Trial Name: NCT04551131 — Phase 1 & 2
Hemophagocytic Lymphohistiocytosis Research Study Groups: Frontline Arm, Salvage Arm
Hemophagocytic Lymphohistiocytosis Clinical Trial 2023: Dexamethasone Highlights & Side Effects. Trial Name: NCT04551131 — Phase 1 & 2
Dexamethasone (Corticosteroid) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04551131 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What conditions is Ruxolitinib typically prescribed to treat?

"Ruxolitinib is a drug commonly prescribed to address synovitis, but it can also be useful in managing ophthalmia, sympathetic disorders, prostate cancer and branch retinal vein occlusion."

Answered by AI

Has there been additional research conducted involving Ruxolitinib?

"Currently, 915 clinical trials involving Ruxolitinib are in progress with 230 of these experiments having entered Phase 3. Seattle, Washington hosts most of the investigations; however, there is also a global reach that spans 34,485 locations."

Answered by AI

Is the age range for this research limited to those under 45 years old?

"To be accepted as a participant in this study, one must not exceed 22 years of age and should have been alive for at least 6 weeks. Across the board, 224 trials target pediatric patients while 757 clinical studies focus on adults aged 65 or over."

Answered by AI

Who meets the criteria to be included in this research endeavor?

"This clinical trial seeks 62 participants aged between 6 weeks and 22 years with hemophagocytic syndromes. In addition to these requirements, patients must possess low or absent NK-cell activity OR demonstrate decreased CD107a mobilization, be able to take medication orally or have a nasogastric tube placed if unable to do so, weigh at least 3 kgs."

Answered by AI

Are there numerous locations in the US that are conducting this research?

"There is a total of 11 sites offering this trial. Notable locations include Children's Wisconsin/Medical College of Wisconsin in Milwaukee, Johns Hopkins in Baltimore and University of California San Francisco in San Francisco with 8 other medical centres located throughout the country."

Answered by AI

How many participants are being recruited to this clinical research?

"Affirmative. Clinicaltrials.gov's data reveals that this trial is presently recruiting participants, having first been published on July 13th 2021 and lastly updated the same date in 2022. 62 individuals need to be signed up from 11 separate medical facilities."

Answered by AI

What positive impact is this research expected to have?

"The primary outcome of this 8-week study pertains to the Adverse Events (AEs) associated with ruxolitinib treatment. Secondary objectives include assessing patients' Overall Response rate, calculating mean Time to Response and estimating one year Survival after initiating therapy."

Answered by AI

Are there any opportunities for individuals to participate in this experiment?

"Affirmative. Based on the information posted at clinicaltrials.gov, this research endeavour is now searching for participants and has been since July 13th 2021. This trial requires 62 individuals from 11 different sites to be enrolled before completion."

Answered by AI
~21 spots leftby Aug 2025