Castleman's Disease (CD)Phase 2NCT07085039
Ruxolitinib in Previously Treated Idiopathic Multicentric Castleman Disease
A Phase 2 study testing Ruxolitinib in people with Castleman's Disease (CD) and Idiopathic Multicentric Castleman's Disease, run by University of Pennsylvania at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Castleman's Disease (CD), Idiopathic Multicentric Castleman's Disease
- What is being tested?
- Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2025to 31 Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 34Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Bridget Austin, 267-586-9977, Bridget.Austin@Pennmedicine.upenn.edu
Sunita Nasta, MD, FACP
About this study
Official title: A Phase II, Single-Arm Open-Label Multi-Center Study of Ruxolitinib in Previously Treated Idiopathic Multicentric Castleman Disease
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.
Read the detailed description
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.
- Being tested
- Ruxolitinib
- Medical subject headings
- Castleman Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Participants will take the study drug in a tablet form, by mouth, every day for the next year, as long as it is helping with their disease and not causing unacceptable side effects.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib | Other | This is an open-label study so all patients will be assigned to the same interventional arm and given ruxolitinib Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of participants achieving a positive Clinical Benefit Response (CBR) response at 12 months ± 1 month. Assessments at 12 months ± 1 month. will be compared to those at the baseline visit
Clinical Benefit Response (CBR):CBR is defined by improvements in clinical symptoms (fatigue, anorexia, fever and night sweats). Laboratory markers like Hemoglobin levels, weight change and lymph node size are included in the CBR. A CBR is considered positive if there is at least a 25% reduction in the size of the largest lymph node (measured by modified Cheson criteria), a significant improvement in at least one laboratory marker (e.g., hemoglobin), and improvement in at least one clinical symptom without worsening of others.
Positive response: Relative to baseline, improvement in at least one of the criterion without worsening of any single criterion other than hemoglobin on two consecutive study visits.
Negative response: Relative to baseline, worsening of any single criterion other than hemoglobin on two consecutive site visits or failure to achieve improvement for any criterion.
Measured: 12 months ± 1 month
Secondary outcomes (6)
The proportion of participants achieving a positive CBR response at 3, 6, 9 months compared to baseline
Clinical Benefit Response (CBR):CBR is defined by improvements in clinical symptoms (fatigue, anorexia, fever and night sweats). Laboratory markers like Hemoglobin levels, weight change and lymph node size are included in the CBR. A CBR is considered positive if there is at least a 25% reduction in the size of the largest lymph node (measured by modified Cheson criteria), a significant improvement in at least one laboratory marker (e.g., hemoglobin), and improvement in at least one clinical symptom without worsening of others
Measured: 3, 6, and 9 months compared to baseline
Disease activity, as measured by the CHAP scale, at 3, 6, 9, and 12 months ± 1 month. Assessments will be compared to those obtained at the Baseline Visit
The CHAP scale, which integrates C-reactive protein (CRP), hemoglobin, albumin levels, and Eastern Cooperative Oncology Group (ECOG) performance status, will be used to assess overall disease activity. CHAP is made up of four scores that are rated from 0-4. The sum of the four scores provides an objective scale for measuring a patient's disease activity and monitoring how it changes over time. The scale range is 0-16. A reduction in CHAP scores indicated decreased disease activity. A higher score indicates greater disease activity.
Measured: 3, 6, 9, and 12 months ± 1 month
Disease activity, as measured by MCD-related Overall Symptom Score as measured by 34 outcome measures at 3, 6, 9, and 12 months ± 1 month compared to those obtained at the Baseline Visit
MCD-related Overall Symptom Score is measured by 34 MCD-related outcome measures. The Overall Symptom Score, comprising 34 outcome measures (fatigue, weight change, night sweats, etc.), will be used to evaluate the broader impact of Ruxolitinib on disease symptoms.
Measured: 3, 6, 9, and 12 months ± 1 month
The proportion of participants achieving a lymph node response, following the modified Cheson response criteria compared to baseline
Radiological response will be assessed using the modified Cheson criteria, which quantifies changes in lymph node size. A lymph node response was defined as a 25% reduction in bi-dimensional measurements of the largest lymph node compared to baseline
Measured: Month 12
The proportion of participants that remain on study drug for the duration of the study
Number of patients who remained on the study drug for the entire study
Measured: Up to 73 weeks
The proportion of participants that indicate that they are currently receiving Ruxolitinib at the end of the Follow-Up Phase
Number of participants who continue to take the drug until the end of the follow up phase
Measured: Up to 73 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2025
- Study start18 Dec 2025
- Primary completion31 Jul 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Castleman's Disease (CD)Studies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Joshua Brandstadter, MD, PhD, MSc, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07085039
- Sponsor's protocol ID
- 858269
- Status last verified
- Apr 2026
- First submitted
- 20 May 2025
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Data sharing may be considered upon reasonable request to the Principal Investigator, with appropriate agreements in place
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Single-Arm Open-Label Multi-Center Study of Ruxolitinib in Previously Treated Idiopathic Multicentric Castleman Disease
- ClinicalTrials.gov ID
- NCT07085039
- Acronym / sponsor ID
- 858269
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- Incyte Corporation, National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 18 Dec 2025 (actual)
- Primary completion
- 31 Jul 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 25 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib | Other | Drug: RuxolitinibThis is an open-label study so all patients will be assigned to the same interventional arm and given ruxolitinib |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female, age 18-80
- Documented disease history consistent with diagnostic criteria for iMCD
- 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
Key exclusion criteria (34 listed)
- Subjects cannot be pregnant or nursing females
- 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
- 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
- Subjects cannot have:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RuxolitinibParticipants will take the study drug in a tablet form, by mouth, every day for the next year, as long as it is helping with their disease and not causing unacceptable side effects. | Drug | Ruxolitinib |
Primary outcome
The proportion of participants achieving a positive Clinical Benefit Response (CBR) response at 12 months ± 1 month. Assessments at 12 months ± 1 month. will be compared to those at the baseline visit
Time frame: 12 months ± 1 month
Clinical Benefit Response (CBR):CBR is defined by improvements in clinical symptoms (fatigue, anorexia, fever and night sweats). Laboratory markers like Hemoglobin levels, weight change and lymph node size are included in the CBR. A CBR is considered positive if there is at least a 25% reduction in the size of the largest lymph node (measured by modified Cheson criteria), a significant improvement in at least one laboratory marker (e.g., hemoglobin), and improvement in at least one clinical symptom without worsening of others. Positive response: Relative to baseline, improvement in at least one of the criterion without worsening of any single criterion other than hemoglobin on two consecutive study visits. Negative response: Relative to baseline, worsening of any single criterion other than hemoglobin on two consecutive site visits or failure to achieve improvement for any criterion.
6 secondary outcomes
The proportion of participants achieving a positive CBR response at 3, 6, 9 months compared to baseline
Time frame: 3, 6, and 9 months compared to baseline
Disease activity, as measured by the CHAP scale, at 3, 6, 9, and 12 months ± 1 month. Assessments will be compared to those obtained at the Baseline Visit
Time frame: 3, 6, 9, and 12 months ± 1 month
Disease activity, as measured by MCD-related Overall Symptom Score as measured by 34 outcome measures at 3, 6, 9, and 12 months ± 1 month compared to those obtained at the Baseline Visit
Time frame: 3, 6, 9, and 12 months ± 1 month
The proportion of participants achieving a lymph node response, following the modified Cheson response criteria compared to baseline
Time frame: Month 12
The proportion of participants that remain on study drug for the duration of the study
Time frame: Up to 73 weeks
The proportion of participants that indicate that they are currently receiving Ruxolitinib at the end of the Follow-Up Phase
Time frame: Up to 73 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
- Individual participant dataSharing planned: Undecided. Data sharing may be considered upon reasonable request to the Principal Investigator, with appropriate agreements in place
Related trials for Castleman's Disease (CD)
Active, not recruitingPhase 2
Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease
Castleman Disease, Castleman's Disease, Multicentric
RecruitingObservational study
International Registry for Patients With Castleman Disease
Castleman Disease, Castleman's Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Joshua Brandstadter, MD, PhD, MSc principal investigator
University of Pennsylvania
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib in people with Castleman's Disease (CD) and Idiopathic Multicentric Castleman's Disease, run by University of Pennsylvania at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 34 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Bridget Austin, MS (267-586-9977, bridget.austin@pennmedicine.upenn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of participants achieving a positive Clinical Benefit Response (CBR) response at 12 months ± 1 month. Assessments at 12 months ± 1 month. will be compared to those at the baseline visit (time frame: 12 months ± 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2026.