ClinicalTrials.gov record NCT07085039
Ruxolitinib in Previously Treated Idiopathic Multicentric Castleman Disease
A Phase II, Single-Arm Open-Label Multi-Center Study of Ruxolitinib in Previously Treated Idiopathic Multicentric Castleman Disease
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase II, single-arm, open-label study gives ruxolitinib tablets to adults with idiopathic multicentric Castleman disease (iMCD) whose disease did not improve with, or who cannot take, siltuximab or tocilizumab. The study will follow participants for about a year to assess symptoms, laboratory measures, and lymph node size while taking the drug. The main goal is to evaluate tolerability and clinical benefit over 12 months.
What it is studying. The study is testing the effects and tolerability of ruxolitinib in adults with iMCD previously treated with or unable to use anti‑IL‑6 therapies. It will measure changes in symptoms, blood tests, and lymph node size while participants take the tablet daily.
What the researchers hope to learn. Scientifically, the study aims to learn how well ruxolitinib is tolerated in this patient group and how it affects iMCD signs and symptoms, blood markers, lymph node size, and standardized disease-activity scores.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The research study is being done to look at the effects of ruxolitinib in adults with idiopathic Multicentric Castleman Disease (iMCD) that has not gotten better from taking siltuximab or tocilizumab, or who cannot take those medications.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Ruxolitinib
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Male or female, age 18-80
- Documented disease history consistent with diagnostic criteria for iMCD
- Indication of clinico-histopathological features consistent with iMCD as determined by a licensed pathologist in a diagnostic pathology report, or a CAS grade of at least 3 in the companion registry study (ACCELERATE).
- Refractory (patient did not achieve sufficient disease control with anti-IL-6 therapy, as determined by the site investigator), relapsed (return of symptoms while on therapy), or inability to tolerate anti-IL-6 or anti-IL-6 receptor therapy
- Evidence of active disease, defined by at least two of: constitutional symptoms (fatigue, night sweats, fever, weight change), hemoglobin < lower limit of normal, C-reactive protein > upper limit of normal (or >10 mg/L), or albumin < lower limit of normal (<3.5 g/dL), at lease one lymph node meeting modified Cheson criteria
- Ability to consume oral medication in the form of a tablet
- Ability to provide informed consent prior to any study-specific activities
Exclusion criteria
- Subjects cannot be pregnant or nursing females
- Women of childbearing potential must have a negative pregnancy test documented at the Baseline Visit. Subjects of reproductive potential may not participate unless they have agreed, as part of the informed consent, to use an effective contraceptive method for the duration of the study and for at least 12 weeks after ending treatment
- Subjects cannot have received any systemic therapy(ies) intended to treat iMCD other than corticosteroids or anti-IL-6 therapy within 14 days of enrollment
- Corticosteroid treatment must have been initiated more than 28 days prior to enrollment and be maintained at a stable or tapering dose (prednisone or equivalent up to 1 mg/kg/day) prior to enrollment; dose cannot be elevated for the duration of the study but can be maintained or tapered
- For subjects who cannot or are unwilling to undergo a 14-day washout period from their anti-IL-6 therapy: i. Anti-IL-6 therapy may be permitted if the subject has been on therapy for at least 3 months, no toxicity has been documented, and sufficient disease control is not achieved ii. Anti-IL-6 therapy is recommended to be discontinued within the first 6 weeks after starting ruxolitinib
- Subjects cannot have previously received ruxolitinib monotherapy or combination therapy to treat iMCD
- Subjects cannot have uncontrolled infection or infectious disease(s) that is/are exclusionary for / mimickers of iMCD
- Fungal disease must be stable for at least two weeks before enrollment
- Subjects with history of recent bacteremia must have a documented negative blood culture before enrollment
- Subjects with past history of non-therapy related opportunistic infection should discuss eligibility and possible immunologic evaluation with the licensed site investigator prior to enrollment
- Subjects cannot have:
- ECOG >3
- eGFR <30mL/min/1.73 m2 or creatinine >3.0 mg/dL
- Absolute neutrophil count (ANC) < 1000 x 109/L
- Hemoglobin ≤ 6.5 g/dL (transfusion independent, defined as not receiving a red blood cell transfusion for ≥ 7 days prior)
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) laboratory values greater than three times the upper limit of normal
- Albumin < 2 g/dL (transfusion independent, defined as not receiving intravenous albumin for ≥ 7 days prior)
- Platelet count ≤ 40 x 109/L (transfusion independent, defined as not receiving platelet transfusion for ≥ 7 days prior)
- Pulmonary involvement or interstitial pneumonitis with dyspnea (adequate pulmonary function is defined as pulse oximetry > 94% on room air if there is clinical indication for determination (e.g. dyspnea at rest, history of interstitial pneumonitis, etc.)
- Fasting cholesterol > 300 mg/dL or fasting triglyceride > 400 mg/dL
- Subjects cannot have rheumatologic disease(s) that is/are exclusionary for / mimickers of iMCD
- Subjects cannot have a prior malignancy except for: (1) adequately treated basal cell or squamous cell skin cancer, (2) in situ cervical cancer, or (3) other cancer for which the subject has not received treatment within one year prior to enrollment
- Other cancers will only be acceptable if the patient's life expectancy exceeds five years and they are not exclusionary diagnoses for iMCD
- Subjects cannot have a documented history of human immunodeficiency virus (HIV) or HHV-8 infection, or severe combined immunodeficiency syndrome
- Subjects cannot have active infections of mycobacterium tuberculosis, hepatitis B, or hepatitis C due to the potential reactivation with ruxolitinib
- A positive hepatitis B serology indicative of previous immunization (i.e., hepatitis B surface antigen (HBsAb) positive and hepatitis B core antibody (HBcAb) negative), or a fully resolved acute hepatitis B infection is not an exclusion criterion.
- Subjects with an indolent chronic hepatitis B infection (normal ALT, AST, albumin and no radiographic or biopsy evidence of cirrhosis) may be eligible.
- Subjects with active hepatitis C are excluded. Subjects positive for hepatitis C virus (HCV) antibody are eligible only if polymerase chain reaction (PCR) is negative for HCV ribonucleic acid (RNA).
- Subjects cannot have had any major adverse cardiac events, including ischemic stroke and myocardial infarction, within 6 months of enrollment
- Subjects cannot have ongoing or planned participation in another clinical trial involving iMCD directed treatment or that involves immunomodulatory or anti-neoplastic treatment
- Subjects cannot have prior sensitivity / allergy to any formulation of ruxolitinib, its components, or its analogues
- Subjects cannot have serious medical illness, or psychiatric illness or disorders that could potentially interfere with the completion of treatment according to this protocol or participation in the trial
- Subjects cannot have psychiatric disorders that compromise their ability to provide informed consent
- Subjects cannot have any other condition or finding that, in the opinion of the investigator, would make participation in this trial inappropriate
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
There is currently no agreed-on recommended treatment for iMCD patients whose disease does not respond to siltuximab or tocilizumab, which is a major challenge for patients. The purpose of this study is to find out the effects of a study drug, ruxolitinib (also called Jakafi), in these patients. This study looks at how well the drug is tolerated by participants and its effects on participants' iMCD symptoms.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will take ruxolitinib tablets by mouth daily for up to about one year, continuing as long as it is helping their disease and not causing unacceptable side effects. They will attend regular clinic visits for physical exams, blood tests, symptom questionnaires, and imaging to measure lymph node size and other disease markers, with key assessments around 3, 6, 9 and 12 months and follow-up up to about 73 weeks. Women of childbearing potential will have a pregnancy test at baseline and must use effective contraception during the study and for 12 weeks after stopping treatment. Study staff will monitor safety and disease activity and may stop treatment if needed.
How the study is structured. Phase II interventional treatment study in which all enrolled participants receive oral ruxolitinib (no placebo or comparison group). The study is open-label (patients and researchers know the treatment) and follows participants with clinical and laboratory assessments over about 12 months, with additional follow-up.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Castleman's Disease (CD); Idiopathic Multicentric Castleman's Disease. Does it apply to my diagnosis?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Castleman disease
Important dates
- Study start
- Dec 18, 2025
- Primary completion
- Jul 31, 2028 (estimated)
- Study completion
- Dec 31, 2028 (estimated)
- Target enrollment
- 14 participants (estimated)
- Last updated on registry
- Apr 22, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- University of Pennsylvania
- Collaborators
- Incyte Corporation
- National Heart, Lung, and Blood Institute (NHLBI)
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Bridget Austin, MS 📞 267-586-9977 ✉ bridget.austin@pennmedicine.upenn.edu
Participating sites (1)
- University of PennsylvaniaRecruiting
Philadelphia, Pennsylvania, 19104 · United States
- Bridget Austin · 267-586-9977 · Bridget.Austin@Pennmedicine.upenn.edu
- Sunita Nasta, MD, FACP
- Adam Cohen, MD
- Joshua Brandstadter, MD, MS, PhD
Terms used on this page · explanations by MedicalUpdates.ai
- registry
- An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
- Refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- relapsed
- A condition that improved with treatment and has since returned or worsened.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Women of childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- Baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- corticosteroids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- washout period
- A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- eGFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- life expectancy
- Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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