Trial · NCT03933904
Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease
NCT03933904, Phase 2, Active not recruiting, University of Pennsylvania; results first posted 2025-08-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease. Trials, record NCT03933904. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03933904
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease
- NCT number
- NCT03933904
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 7 participants, actual
- Lead sponsor
- University of Pennsylvania
- Conditions
- Castleman Disease, Castleman's Disease, Multicentric
- Interventions
- Sirolimus
- First posted
- 2019-05-01
- Last updated
- 2026-08-20
- Results first posted
- 2025-08-08
- Limitations and caveats
The study's single-arm, open-label design limited conclusions, as there was no control group and potential for bias. The small sample size, impacted by COVID-19 and off-label sirolimus use, reduced statistical power. A short follow-up (12 months plus 12 weeks) limited assessment of long-term efficacy and safety. Given iMCD's chronic nature, future studies should include larger cohorts, control arms, and extended follow-up to assess durability and safety.
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) | 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. |
| Secondary | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 3 | Month 3 | |
| Secondary | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 6 | Month 6 | |
| Secondary | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 9 | Month 9 | |
| Secondary | Percentage of Patients That Remain on Study Drug for the Duration of the Study | Up to 73 weeks | |
| Secondary | Percentage of Patients That Indicate That They Are Currently Receiving Sirolimus at the End of the Follow Up Phase | Up to 73 weeks | |
| Secondary | Disease Activity, as Measured by the CHAP Scale | 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. |
| Secondary | Disease Activity, as Measured by the MCD-related Overall Symptom Score | 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 symptomatic response was defined as a ≥50% decrease in the 34-point symptom score, a durable symptomatic response is a ≥50% decrease in the 34-point symptom score from baseline that was maintained for a minimum of 18 weeks. |
| Secondary | Proportion of Patients Achieving a Lymph Node Response, Following the Modified Cheson Response Criteria | 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 lesion(s), and no new sites of lymphadenopathy \>1.5 cm in longest dimension. Stable Disease: Failure to achieve a CR or PR (see above) without evidence of progressive disease. Progressive Disease: ≥50% increase from nadir in the SPD of any index lesion, or appearance of any new sites of lymphadenopathy that measure \>1.5 cm in longest dimension during or at the end of therapy. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) | 12 ± 1 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 3 | Month 3 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 6 | Month 6 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 9 | Month 9 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percentage of Patients That Remain on Study Drug for the Duration of the Study | Up to 73 weeks | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percentage of Patients That Indicate That They Are Currently Receiving Sirolimus at the End of the Follow Up Phase | Up to 73 weeks | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Disease Activity, as Measured by the CHAP Scale | 12 months ± 2 weeks | score on a scale | MEDIAN | |
| SECONDARY | Disease Activity, as Measured by the MCD-related Overall Symptom Score | Month 12 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Proportion of Patients Achieving a Lymph Node Response, Following the Modified Cheson Response Criteria | Month 12 | participants | NUMBER |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) | Sirolimus | Achieved CBR | 2 | Participants | |
| Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) | Sirolimus | Did Not Achieve CBR | 2 | Participants | |
| Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 3 | Sirolimus | 1 | Participants | ||
| Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 6 | Sirolimus | 2 | Participants | ||
| Percentage of Patients Achieving a Positive Clinical Benefit Response (CBR) Month 9 | Sirolimus | 2 | Participants | ||
| Percentage of Patients That Remain on Study Drug for the Duration of the Study | Sirolimus | 4 | Participants | ||
| Percentage of Patients That Indicate That They Are Currently Receiving Sirolimus at the End of the Follow Up Phase | Sirolimus | 1 | Participants | ||
| Disease Activity, as Measured by the CHAP Scale | Sirolimus | 3 | Standard Deviation 0.433 | score on a scale | |
| Disease Activity, as Measured by the MCD-related Overall Symptom Score | Sirolimus | Achieved a Symptomatic Response | 1 | Participants | |
| Disease Activity, as Measured by the MCD-related Overall Symptom Score | Sirolimus | Achieved a Durable Symptomatic Response | 1 | Participants |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sirolimus | 7 | |
| Overall Study | COMPLETED | Sirolimus | 4 | |
| Overall Study | NOT COMPLETED | Sirolimus | 3 |
Source ClinicalTrials.gov
Adverse events53
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pruritus | Skin and subcutaneous tissue disorders | Sirolimus | 2 | 7 | 3 |
| other | Abdominal pain | Gastrointestinal disorders | Sirolimus | 2 | 7 | 2 |
| other | Bile duct stenosis | Hepatobiliary disorders | Sirolimus | 1 | 7 | 1 |
| other | Blood and lymphatic system disorders-Other, specify | Blood and lymphatic system disorders | Sirolimus | 1 | 7 | 1 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Sirolimus | 2 | 7 | 2 |
| other | Conjunctivitis | Eye disorders | Sirolimus | 1 | 7 | 1 |
| other | Covid-19 Infection | Infections and infestations | Sirolimus | 1 | 7 | 1 |
| other | Diarrhea | Gastrointestinal disorders | Sirolimus | 2 | 7 | 2 |
| other | Dysphagia | Gastrointestinal disorders | Sirolimus | 1 | 7 | 1 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | Sirolimus | 1 | 7 | 1 |
1 to 10 of 53Source ClinicalTrials.gov
See all 53 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sirolimus | 0 | 7 |
| Total, other adverse events | Sirolimus | 7 | 7 |
| Total, serious adverse events | Sirolimus | 1 | 7 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03933904
GET https://conductscience.org/api/trials/v1/NCT03933904/events?page=2