Adults 2 to 80, any sex, with Castleman Disease or Castleman's Disease, Multicentric. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR)Primary· 12 ± 1 months
Clinical Benefit Response (CBR): The CBR was defined by improvements in clinical symptoms such as fatigue, anorexia, fever, and night sweats.6 Laboratory markers such as hemoglobin levels and weight change were also included in the CBR criteria (Table 1). A CBR was considered positive if there was 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.
Group
Value
95% CI
Sirolimus
2
Sirolimus
2
Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 3Secondary· Month 3
Group
Value
95% CI
Sirolimus
1
Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 6Secondary· Month 6
Group
Value
95% CI
Sirolimus
2
Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 9Secondary· Month 9
Group
Value
95% CI
Sirolimus
2
Percentage of Patients That Remain on Study Drug for the Duration of the StudySecondary· Up to 73 weeks
Group
Value
95% CI
Sirolimus
4
Percentage of Patients That Indicate That They Are Currently Receiving Sirolimus at the End of the Follow Up PhaseSecondary· Up to 73 weeks
Group
Value
95% CI
Sirolimus
1
Disease Activity, as Measured by the CHAP ScaleSecondary· 12 months ± 2 weeks
The CHAP scale consists of C-reactive protein (CRP), hemoglobin, albumin, and Eastern Cooperative Oncology Group (ECOG) performance score, each with a subscale range of 0-4. Each criterion in the CHAP scoring system provides a graded measure for a patient's disease activity. The sum of the four scores provides an objective scale for measuring a patient's disease activity and monitoring how it changes over time (scale range 0-16). A higher score indicates greater disease activity.
Group
Value
95% CI
Sirolimus
3
± 0.433
Disease Activity, as Measured by the MCD-related Overall Symptom ScoreSecondary· Month 12
MCD-related Overall Symptom Score is measured by 34 MCD-related outcome measures. These scores addressed fatigue, weight change, night sweats, etc. The scores were evaluated and graded (as per CTCAE version 4.0, May, 2009), which was used to assess the efficacy of the study intervention. Each symptom score was measured on a numeric scale, ranging from 1 (no symptom) to 5 (very severe or disabling). Scores were combined to create a combined score per patient at each time point. Patients were then assessed as having no response, a symptomatic response, or a durable symptomatic response. A sympto
Group
Value
95% CI
Sirolimus
1
Sirolimus
1
Sirolimus
2
Proportion of Patients Achieving a Lymph Node Response, Following the Modified Cheson Response CriteriaSecondary· Month 12
Radiological response was assessed using the modified Cheson criteria, which quantify 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. Patients were then assessed according to the following responses:
Complete Response: All index lesion(s) must have regressed to normal size (≤1.0 cm in their greatest transverse diameter. No new sites of lymphadenopathy \>1.5 cm in longest dimension.
Partial Response: ≥50% decrease in sum of the products of the greatest diameters (SPD) of index lesio
Achieved Complete Response
Group
Value
95% CI
Sirolimus
1
Progressive Disease
Group
Value
95% CI
Sirolimus
2
Stable Disease
Group
Value
95% CI
Sirolimus
1
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events were collected from baseline to study completion (up to 73 weeks)..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Sirolimus
Serious: 1/7 (14%)
Deaths: 0/7
Serious adverse events (2 terms)
Reaction
System
Sirolimus
Pneumonia
Infections and infestations
—
Sepsis
Infections and infestations
—
Other adverse events (51 terms — click to expand)
Reaction
System
Sirolimus
Fatigue
General disorders
—
Headache
Nervous system disorders
—
Pain (whole body)
Musculoskeletal and connective tissue disorders
—
Pruritus
Skin and subcutaneous tissue disorders
—
Abdominal pain
Gastrointestinal disorders
—
Cough
Respiratory, thoracic and mediastinal disorders
—
Diarrhea
Gastrointestinal disorders
—
Hyperhidrosis
Skin and subcutaneous tissue disorders
—
Shortness of breath
Respiratory, thoracic and mediastinal disorders
—
Sore throat
Respiratory, thoracic and mediastinal disorders
—
Tremors
Nervous system disorders
—
Mucositis oral (mouth sores)
Gastrointestinal disorders
—
Ascites (worsening)
Gastrointestinal disorders
—
Bile duct stenosis
Hepatobiliary disorders
—
Blood and lymphatic system disorders-Other, specify
Blood and lymphatic system disorders
—
Conjunctivitis
Eye disorders
—
Covid-19 Infection
Infections and infestations
—
Dysphagia
Gastrointestinal disorders
—
Dyspnea
Respiratory, thoracic and mediastinal disorders
—
Eye pain
Eye disorders
—
Itchy on palms
Skin and subcutaneous tissue disorders
—
Lung infection
Infections and infestations
—
Muscle cramp
Musculoskeletal and connective tissue disorders
—
Nausea
Gastrointestinal disorders
—
Neck pain
Musculoskeletal and connective tissue disorders
—
Neuropathy
Nervous system disorders
—
Peripheral sensory neuropathy
Nervous system disorders
—
Pleural effusion
Respiratory, thoracic and mediastinal disorders
—
Rash acneiform
Skin and subcutaneous tissue disorders
—
Skin hyperpigmentation
Skin and subcutaneous tissue disorders
—
Urinary Tract Infection
Infections and infestations
—
Respiratory, thoracic and mediastinal disorders - Other
Respiratory, thoracic and mediastinal disorders
—
Skin and subcutaneous tissue disorders - Other, specify (ankle)
Skin and subcutaneous tissue disorders
—
Skin and subcutaneous tissue disorders - Other, specify (groin)
Skin and subcutaneous tissue disorders
—
Skin and subcutaneous tissue disorders - Other, specify (torso)
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by University of Pennsylvania
Last refreshed: 3 March 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03933904.