All-Trans Retinoic Acid for the Treatment of Hemophagocytic Lymphohistiocytosis
Trial statusNot yet recruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18-65
SponsorBeijing Anzhen Hospital
About this trial
This study is designed to evaluate the safety and preliminary efficacy of all-trans retinoic acid (ATRA) as an initial treatment for patients with active hemophagocytic lymphohistiocytosis (HLH). HLH is a severe hyperinflammatory syndrome caused by excessive activation of immune cells and uncontrolled cytokine release. Current treatment often requires intensive immunosuppressive or cytotoxic therapy, which may be associated with significant toxicity.
ATRA is an orally available agent that has been widely used in other hematologic diseases and has immunomodulatory effects. Preclinical studies suggest that ATRA may help control HLH-related inflammation and improve immune dysregulation. In this study, patients with newly diagnosed or treatment-naïve active HLH will receive ATRA-based initial therapy. The study will assess clinical response, changes in HLH-related inflammatory markers, organ function, viral or disease-related parameters when applicable, and treatment-related adverse events.
The goal of this study is to determine whether ATRA can provide a safe and feasible initial therapeutic approach for active HLH and support further clinical development of ATRA-based treatment strategies in this disease.
Eligibility criteria
Qualifiers
1. Age ≥18 years.
Patients diagnosed with active hemophagocytic lymphohistiocytosis according to HLH-2004 diagnostic criteria or the investigator's clinical assessment.
Newly diagnosed or treatment-naïve active HLH requiring initial HLH-directed therapy.
Presence of active disease manifestations, including at least one of the following: persistent fever, cytopenia, hyperferritinemia, splenomegaly, hypofibrinogenemia and/or hypertriglyceridemia, elevated soluble CD25, hemophagocytosis, reduced or absent NK-cell activity, or other HLH-related organ involvement.
Disqualifiers
Prior systemic HLH-directed therapy for the current HLH episode, including etoposide-based therapy, ruxolitinib, emapalumab, alemtuzumab, PD-1 blockade, or other investigational HLH-directed treatment. Short-term corticosteroids, supportive care, anti-infective therapy, or emergency treatment before enrollment may be allowed at the investigator's discretion.
Known hypersensitivity to all-trans retinoic acid, tretinoin, retinoids, or any component of the study drug.
Pregnant or breastfeeding women.
Patients with acute promyelocytic leukemia or other diseases for which ATRA is being used as standard leukemia-directed therapy.
Trial design
Treatments tested in this trial
- ATRA
Treatment groups
30 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov