Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymp… (NCT04551131) | Clinical Trial Compass
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 range6 Weeks – 22 Years
SexALL
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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.
✓. Patient weighs ≥3 kg.
Exclusion criteria
✕. Patient is \<6 weeks or \>22 years of age.
✕. Patient weighs \<3 kg.
What they're measuring
1
Complete Response (CR)/Complete Response with Incomplete Hematologic Recovery (CRi)
Timeframe: 8 weeks
2
Adverse events (AEs) associated with the ruxolitinib-containing regimen
Timeframe: up to 8 weeks
3
Adverse events (AEs) associated with the ruxolitinib-containing regimen
✕. Patient is likely to require \<4 weeks of therapy (i.e., HSCT is imminent).
✕. Patients with creatinine clearance (CrCl) \<15 mL/min who are NOT receiving dialysis.
✕. Patient has evidence of severe organ dysfunction, defined as: Severe liver dysfunction (ALT \>1000 U/L), OR Cardiorespiratory failure requiring any ionotropic support OR extracorporeal life support, OR high frequency oscillatory ventilation, other forms of respiratory support or ventilation are allowed if the patient is not on vasopressors)
✕. Patient with pre-existing rheumatologic disorder.