BridgeMD
New treatment option for Hemophagocytic Lymphohistiocytoses
Official title Ruxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)
ClinicalTrials.gov ID: NCT06160791
What this study is testing
What is Ruxolitinib?
Ruxolitinib is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophagocytic lymphohistiocytoses.
Also referred to as INCB18424, Oral JAK.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This phase II trial tests the effects of ruxolitinib in combination with a de-intensified HLH-94 drug regimen has on patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH), a disorder caused by dysregulated immune responses (that is, immune responses that are too strong and cause inflammatory damage to normal tissues). The therapy used for HLH decreases the activity of the immune system.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Ability to understand and the willingness to sign a written informed consent document.
- Males and females, 18 years of age or older at the time of enrollment.
- Participants must have active HLH and meet \>= 5 of 8 of the HLH-2004 diagnostic criteria, or have familial/primary HLH with pathogenic/likely...
- Fever \>= 38.5 degrees Celsius (C) (or \>= 38 degrees C if acetaminophen given in prior 6 hours).
- Splenomegaly.
You likely can't join if
- Participant is receiving or received any other investigational agent within 1 week of the first dose of treatment.
- Females who are pregnant or breastfeeding. Female participants of child-bearing potential must have a negative pregnancy test within 7 days of...
- Males who expect to conceive children, and/or who decline highly effective methods of contraception during the entire duration of the study.
- Patient cannot take medications orally or via a nasogastric/orogastric tube.
- Poor life expectancy \< 2 weeks.
- Clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute myocardial infarction, or stroke within 6 months, New...
See the full eligibility criteria
- Ability to understand and the willingness to sign a written informed consent document.
- Males and females, 18 years of age or older at the time of enrollment.
- Participants must have active HLH and meet \>= 5 of 8 of the HLH-2004 diagnostic criteria, or have familial/primary HLH with pathogenic/likely pathogenic germline variant(s) in genes known to cause HLH (e.g., PRF1...
- Fever \>= 38.5 degrees Celsius (C) (or \>= 38 degrees C if acetaminophen given in prior 6 hours).
- Splenomegaly.
- Peripheral cytopenias involving \>= 2 of 3 cell lines (absolute neutrophil count \< 1000/uL; hemoglobin \< 9 g/dL; platelets \< 100,000/uL).
- Hypertriglyceridemia (fasting triglycerides \>= 265 mg/dL) or Hypofibrinogenemia (fibrinogen =\< 150 g/dL).
- Hemophagocytosis on tissue biopsy, such as in the bone marrow, spleen, lymph node, or liver.
- Low/absent natural killer (NK)-cell activity/perforin and/or decreased CD107a mobilization.
- Ferritin \>= 500 ug/L.
- Soluble IL-2 receptor (sCD25) \> 2400 U/mL or two standard deviations above age-adjusted laboratory-specific norms.
- The effects of ruxolitinib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception for the duration of study participation and for...
- Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
- Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and two months after last administration of study treatment.
- Participant is receiving or received any other investigational agent within 1 week of the first dose of treatment.
- Females who are pregnant or breastfeeding. Female participants of child-bearing potential must have a negative pregnancy test within 7 days of treatment and lactating females must discontinue breast feeding during...
- Males who expect to conceive children, and/or who decline highly effective methods of contraception during the entire duration of the study.
- Patient cannot take medications orally or via a nasogastric/orogastric tube.
- Poor life expectancy \< 2 weeks.
- Clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute myocardial infarction, or stroke within 6 months, New York Heart Association class III or IV. congestive heart failure, and...
- Estimated creatine clearance (CrCl) \< 15 mL/min while not on dialysis.
- Known (biopsy-confirmed) liver cirrhosis or suspected cirrhosis with a Model for End- Stage Liver Disease (MELD) score of \> 20, or aspartate aminotransferase (AST) or alanine transaminase (ALT) values \> 1000 not...
- Severe organ dysfunction, such as cardiorespiratory failure requiring inotropic medications or extracorporeal life support. Respiratory support including intubation/ventilation is allowed. \ Vasopressors are allowed if...
- Newly diagnosed acute and clinically active tuberculosis, hepatitis B, and/or hepatitis C.
- Patients with active human immunodeficiency virus (HIV) are not excluded from this study but must be on antiretrovirals.
- Patients with hepatitis B or C viremia can be on study if the hepatitis is not considered clinically active and/or if it is chronic. These patients should be discussed with the principal investigator.
- Individuals with a prior malignancy whose natural history or treatment has the potential to interfere with the safety or how well it works assessment of the investigational regimen.
- Individuals with chimeric antigen receptor (CAR)-T-associated HLH.
- No prior HLH-directed therapy except corticosteroids for \< 2 consecutive weeks and anakinra.
- Adjunctive approaches such as rituximab for Epstein-Barr virus (EBV) viremia or IVIG for viral infection are permitted.
- Emapalumab, alemtuzumab, anti-thymocyte globulin (ATG), tocilizumab, siltuximab, or prior ruxolitinib are NOT permitted. Cyclosporine and tacrolimus are not permitted in the initial induction period.
- Hypersensitivity to ruxolitinib or any of its excipients
The study team makes the final eligibility decision.
Where it's taking place
- Irvine, California, United States
- Sacramento, California, United States
- San Diego, California, United States
- San Francisco, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Irvine, California, United States; Sacramento, California, United States; San Diego, California, United States; San Francisco, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.