Active — Not RecruitingPhase 1/2

Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis

United States10 participantsStarted 2021-07-13

Plain-language summary

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.

Who can participate

Age range

6 Weeks – 22 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Patient is ≥6 weeks and ≤22 years of age.
. Patient weighs ≥3 kg.
. Patient is able to take medication PO and/or patient or parent is willing to have NG tube placed if patient is unable to take medications PO.
. Patient has active HLH if:
. Patient has not received prior HLH therapy, except steroids (any dose or length of therapy is allowed) OR anakinra (any dose or length of therapy is allowed).
. Patient, parent, or legal authorized representative (LAR) must provide informed consent.
. Patient is ≥6 weeks and ≤22 years of age.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Complete Response (CR)/Complete Response with Incomplete Hematologic Recovery (CRi)

Timeframe: 8 weeks

Adverse events (AEs) associated with the ruxolitinib-containing regimen

Timeframe: up to 8 weeks

Adverse events (AEs) associated with the ruxolitinib-containing regimen

Timeframe: up to 1 year after diagnosis

Trial details

NCT IDNCT04551131

SponsorSt. Jude Children's Research Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-11-07

View on ClinicalTrials.gov More Hemophagocytic Lymphohistiocytosis trials

Plain-language summary

Who can participate

Age range

6 Weeks – 22 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Patient is ≥6 weeks and ≤22 years of age.
. Patient weighs ≥3 kg.
. Patient is able to take medication PO and/or patient or parent is willing to have NG tube placed if patient is unable to take medications PO.
. Patient has active HLH if:
. Patient has not received prior HLH therapy, except steroids (any dose or length of therapy is allowed) OR anakinra (any dose or length of therapy is allowed).
. Patient, parent, or legal authorized representative (LAR) must provide informed consent.
. Patient is ≥6 weeks and ≤22 years of age.

What they're measuring

Complete Response (CR)/Complete Response with Incomplete Hematologic Recovery (CRi)

Timeframe: 8 weeks

Adverse events (AEs) associated with the ruxolitinib-containing regimen

Timeframe: up to 8 weeks

Adverse events (AEs) associated with the ruxolitinib-containing regimen

Timeframe: up to 1 year after diagnosis

Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria