Idiopathic Multicentric Castleman's DiseaseNCT05995834
Producing a Novel Symptom Burden Scale for People Living With Idiopathic Multicentric Castleman Disease (ISBUS)
An observational study of Idiopathic Multicentric Castleman's Disease, run by RECORDATI GROUP.
Trial summary
Development, validation of a novel symptom burden scale to assess and quantify the burden experienced by people living with Idiopathic Multicentric Castleman Disease (iMCD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Multicentric Castleman's Disease
- What is being tested?
- Research questions
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- Oct 2023to Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients diagnosed with idiopathic Multicentric Castleman Disease (iMCD).
Who may be able to join
- Diagnosis of idiopathic Multicentric Castleman Disease (iMCD)
- Adults (aged 18+ years)
- Fluent in English
Who cannot join
- No diagnosis of iMCD
- Children (aged < 18 years)
- Not able to understand or communicate in English
- People lacking the capacity to consent
Trial locations and contacts
No sites are listed yet.
About this study
Development, validation of a novel symptom burden scale to assess and quantify the burden experienced by people living with Idiopathic Multicentric Castleman Disease (iMCD).
Read the detailed description
Idiopathic Multicentric Castleman Disease (iMCD) is a rare lymphoproliferative disorder associated with systemic inflammation and organ dysfunction. The condition has an unknown aetiology and diagnostic criteria were established recently in 2017. It can be distinguished from other forms of Castleman Disease, including unicentric presentations and Multicentric Castleman Disease (MCD) that is associated with the Kaposi sarcoma herpesvirus. iMCD has an estimated prevalence of between 6.9 and 9.7 people per million. While clinical presentations vary, iMCD can be associated with a high degree of symptom burden, including constitutional, gastrointestinal, neuropsychiatric, dermatologic, respiratory, and hematologic or lymphoreticular problems. Both symptoms and the side-effects of treatment can impact the health-related quality of life (HRQoL) of people with iMCD
This international mixed-methods study will be conducted in four stages:
Stage 1: Item generation
- Drawing on the content, data, and analysis from a previous international patient online survey in iMCD with 65 patients, generate draft PROM content (including a longlist of candidate draft items under existing themes) to assess symptom burden in iMCD.
- Incorporate expert opinion and lived experience to achieve consensus on which items will be tested for inclusion in the symptom burden scale via an online multi-stakeholder workshop involving the funder, research team, patient and public involvement and engagement (PPIE) representative(s) (i.e., patients with iMCD and/or their carers), healthcare professionals, and any other relevant stakeholders.
Stage 2: Item testing and refinement
- Cognitive debriefing interviews with about 10 people living with iMCD to assess the content validity of the items (and associated PROM content) in terms of relevance, comprehensibility, and comprehensiveness. This will include assessing whether they are important/meaningful to patients.
- Analyse and summarise qualitative evidence on content validity, make recommendations on revisions to the draft PROM, and agree this with the multi-stakeholder advisory group (as described above) and a separate group of PPIE collaborators.
Stage 3: Final item selection
- Administer the refined PROM (from Stage 2) to as wide a sample as possible of people living with iMCD. The estimated sample size is 100 patients. The PROM will be administered alongside additional sociodemographic and clinical questions and measures of burden and/or health-related quality of life (HRQoL).
- Taking into account the sample size, conduct appropriate psychometric analyses in order to provide evidence for final item selection and to provide preliminary psychometric evidence on the reliability and validity of the final measure.
- Taking into account all of the available qualitative and quantitative evidence, and a consultation with PPIE collaborators, agree (with the multi-stakeholder advisory group) on final item selection, with the goal of obtaining an 8-10 item measure of symptom burden.
Stage 4: Preliminary measures of change
- Stage 4a: Re-administer the refined PROM (from Stage 2) to participants (from Stage 3) alongside measure(s) of perceived clinical change.
- Stage 4b: Conduct qualitative interviews with about 10 participants who have completed the PROM at both time points to understand what constitutes a meaningful difference from their perspective.
- Triangulate quantitative and qualitative data to estimate a minimally important clinical difference (MCID) for the new PROM, agreed with the multi-stakeholder advisory group.
Recruitment for Stages 2-4 will take place in USA, Canada, Brazil, Australia, New Zealand and the United Kingdom. Interviews will be conducted online for Stages 2 and 4 and the psychometric survey will be hosted and administered online.
Governance: The results of each stage will be discussed and final decisions will be made with a multi-stakeholder group consisting of the wider research team, clinicians and other healthcare professionals and PPIE.
- Being tested
- Research questions
- Medical subject headings
- Multi-centric Castleman's Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Research questionsOther
Stage 1: Secondary analysis of a previous international survey to identify items
Stage 2: Cognitive debriefing interviews with about 10 people living with iMCD to assess the content validity of the items
Stage 3: Completion of questionnaire to finalise scale
Stage 4a: Re-administering of follow-up questionnaire to Stage 3 patients to assess minimally important clinical difference. Test-retest will be calculated only in patients exhibiting no change.
Stage 4b: Qualitative interviews with a subset of participants from Stages and 4 to help with interpretation of meaningful difference from their perspective
What is being measured
The primary outcome is the main question the study is designed to answer.
Long list of candidate items for the scale (stage 1)
completion of data analysis from a previous survey
Measured: Sept 2023 - Nov 2023
Completion of interviews (stage 2)
completion of interviews by 10 patients from all recruiting sites
Measured: Within 6 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis)
Completion of questionnaire (stage 3)
completion of questionnaire which includes extra questions about treatments, time of diagnosis.
Completion of EQ-5D for external validity of PROM. NB: EQ-5D is the full name of the instrument and not an acronym.
Measured: Within 9 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis). NB: Advisory comments noted.
Completion of follow-up questionnaire (stage 4a)
completion of follow-up questionnaire from Stage 3 by same patients who participated in Stage 3. Note: The full name of the scale will be discussed and confirmed. At the moment, the working title is Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS).
Measured: About 8 weeks after Stage 3 questionnaire. Note: Advisory comments noted.
Completion of interviews (stage 4b)
completion of interviews by 10 patients from all recruiting sites
Measured: About 4 weeks after Stage 4a
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2023
- Study startOct 2023 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Multicentric Castleman's DiseaseStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- RECORDATI GROUP
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Sheffield (ScHARR), KMC Health Care, Castleman Disease Collaborative Network
- Responsible party
- Sponsor
- Investigators
- Anju Keetharuth, PhD, Principal investigator, University of SheffieldPhilip Powell, PhD, Principal investigator, University of Sheffield
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05995834
- Sponsor's protocol ID
- ISBUS
- Status last verified
- Aug 2023
- First submitted
- 26 Jul 2023
- Last updated
- 22 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Deanonymized transcriptions of stages 2 and 4 interviews will be securely stored in the University of Sheffield repository with access restricted to investigators only. Quantitative data from stages 3 and 4a will be processed, stored and accessed similarly in the same location.
Publications
Powell PA, Keetharuth A, Frances SM, Carlton J, Makarounas-Kirchmann K, Guerra Soares Brandao AA, Ramasamy K, Shupo F, Dacus KC, Casper C, Fajgenbaum DC, Goh Y, van Rhee F, Zinzani PL, Oksenhendler E, Zhang L, Mukherjee S. Development and Validation of the Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS): Protocol for an International Multistage Mixed Methods Study. JMIR Res Protoc. 2026 Aug 11;15:e96022. doi: 10.2196/96022.
PubMed 42580681
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Producing a Novel Symptom Burden Scale for People Living With Idiopathic Multicentric Castleman Disease (ISBUS)
- ClinicalTrials.gov ID
- NCT05995834
- Acronym / sponsor ID
- ISBUS
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- RECORDATI GROUPIndustry (pharma, biotech, devices)
- Collaborators
- University of Sheffield (ScHARR), KMC Health Care, Castleman Disease Collaborative Network
- Study start
- Oct 2023 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 16 Aug 2023 (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.
Development, validation of a novel symptom burden scale to assess and quantify the burden experienced by people living with Idiopathic Multicentric Castleman Disease (iMCD).
Detailed description
Idiopathic Multicentric Castleman Disease (iMCD) is a rare lymphoproliferative disorder associated with systemic inflammation and organ dysfunction. The condition has an unknown aetiology and diagnostic criteria were established recently in 2017. It can be distinguished from other forms of Castleman Disease, including unicentric presentations and Multicentric Castleman Disease (MCD) that is associated with the Kaposi sarcoma herpesvirus. iMCD has an estimated prevalence of between 6.9 and 9.7 people per million. While clinical presentations vary, iMCD can be associated with a high degree of symptom burden, including constitutional, gastrointestinal, neuropsychiatric, dermatologic, respiratory, and hematologic or lymphoreticular problems. Both symptoms and the side-effects of treatment can impact the health-related quality of life (HRQoL) of people with iMCD
This international mixed-methods study will be conducted in four stages:
Stage 1: Item generation
- Drawing on the content, data, and analysis from a previous international patient online survey in iMCD with 65 patients, generate draft PROM content (including a longlist of candidate draft items under existing themes) to assess symptom burden in iMCD.
- Incorporate expert opinion and lived experience to achieve consensus on which items will be tested for inclusion in the symptom burden scale via an online multi-stakeholder workshop involving the funder, research team, patient and public involvement and engagement (PPIE) representative(s) (i.e., patients with iMCD and/or their carers), healthcare professionals, and any other relevant stakeholders.
Stage 2: Item testing and refinement
- Cognitive debriefing interviews with about 10 people living with iMCD to assess the content validity of the items (and associated PROM content) in terms of relevance, comprehensibility, and comprehensiveness. This will include assessing whether they are important/meaningful to patients.
- Analyse and summarise qualitative evidence on content validity, make recommendations on revisions to the draft PROM, and agree this with the multi-stakeholder advisory group (as described above) and a separate group of PPIE collaborators.
Stage 3: Final item selection
- Administer the refined PROM (from Stage 2) to as wide a sample as possible of people living with iMCD. The estimated sample size is 100 patients. The PROM will be administered alongside additional sociodemographic and clinical questions and measures of burden and/or health-related quality of life (HRQoL).
- Taking into account the sample size, conduct appropriate psychometric analyses in order to provide evidence for final item selection and to provide preliminary psychometric evidence on the reliability and validity of the final measure.
- Taking into account all of the available qualitative and quantitative evidence, and a consultation with PPIE collaborators, agree (with the multi-stakeholder advisory group) on final item selection, with the goal of obtaining an 8-10 item measure of symptom burden.
Stage 4: Preliminary measures of change
- Stage 4a: Re-administer the refined PROM (from Stage 2) to participants (from Stage 3) alongside measure(s) of perceived clinical change.
- Stage 4b: Conduct qualitative interviews with about 10 participants who have completed the PROM at both time points to understand what constitutes a meaningful difference from their perspective.
- Triangulate quantitative and qualitative data to estimate a minimally important clinical difference (MCID) for the new PROM, agreed with the multi-stakeholder advisory group.
Recruitment for Stages 2-4 will take place in USA, Canada, Brazil, Australia, New Zealand and the United Kingdom. Interviews will be conducted online for Stages 2 and 4 and the psychometric survey will be hosted and administered online.
Governance: The results of each stage will be discussed and final decisions will be made with a multi-stakeholder group consisting of the wider research team, clinicians and other healthcare professionals and PPIE.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients diagnosed with idiopathic Multicentric Castleman Disease (iMCD).
Key inclusion criteria (3 listed)
- Diagnosis of idiopathic Multicentric Castleman Disease (iMCD)
- Adults (aged 18+ years)
- Fluent in English
Key exclusion criteria (4 listed)
- No diagnosis of iMCD
- Children (aged < 18 years)
- Not able to understand or communicate in English
- People lacking the capacity to consent
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 |
|---|---|---|
| Research questionsStage 1: Secondary analysis of a previous international survey to identify items Stage 2: Cognitive debriefing interviews with about 10 people living with iMCD to assess the content validity of the items Stage 3: Completion of questionnaire to finalise scale Stage 4a: Re-administering of follow-up questionnaire to Stage 3 patients to assess minimally important clinical difference. Test-retest will be calculated only in patients exhibiting no change. Stage 4b: Qualitative interviews with a subset of participants from Stages and 4 to help with interpretation of meaningful difference from their perspective | Other | – |
Primary outcome
Long list of candidate items for the scale (stage 1)
Time frame: Sept 2023 - Nov 2023
completion of data analysis from a previous survey
Completion of interviews (stage 2)
Time frame: Within 6 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis)
completion of interviews by 10 patients from all recruiting sites
Completion of questionnaire (stage 3)
Time frame: Within 9 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis). NB: Advisory comments noted.
completion of questionnaire which includes extra questions about treatments, time of diagnosis. Completion of EQ-5D for external validity of PROM. NB: EQ-5D is the full name of the instrument and not an acronym.
Completion of follow-up questionnaire (stage 4a)
Time frame: About 8 weeks after Stage 3 questionnaire. Note: Advisory comments noted.
completion of follow-up questionnaire from Stage 3 by same patients who participated in Stage 3. Note: The full name of the scale will be discussed and confirmed. At the moment, the working title is Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS).
Completion of interviews (stage 4b)
Time frame: About 4 weeks after Stage 4a
completion of interviews by 10 patients from all recruiting sites
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2023.
- Derived from this study: PubMed 42580681Powell PA, Keetharuth A, Frances SM, Carlton J, Makarounas-Kirchmann K, Guerra Soares Brandao AA, Ramasamy K, Shupo F, Dacus KC, Casper C, Fajgenbaum DC, Goh Y, van Rhee F, Zinzani PL, Oksenhendler E, Zhang L, Mukherjee S. Development and Validation of the Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS): Protocol for an International Multistage Mixed Methods Study. JMIR Res Protoc. 2026 Aug 11;15:e96022. doi: 10.2196/96022.
- example of registration of PROMsLink provided by the sponsor.
- example of registration of PROMsLink provided by the sponsor.
- example of registration of PROMsLink provided by the sponsor.
- Individual participant dataSharing planned: No. Deanonymized transcriptions of stages 2 and 4 interviews will be securely stored in the University of Sheffield repository with access restricted to investigators only. Quantitative data from stages 3 and 4a will be processed, stored and accessed similarly in the same location.
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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. This study is not recruiting at the moment.
Anju Keetharuth, PhD principal investigator
University of Sheffield
Philip Powell, PhD principal investigator
University of Sheffield
Questions about this trial
What is this clinical trial studying?
An observational study of Idiopathic Multicentric Castleman's Disease, run by RECORDATI GROUP.
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 3 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 22 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anju Keetharuth, PhD (+44 (0) 114 222 0884, d.keetharuth@sheffield.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long list of candidate items for the scale (stage 1) (time frame: Sept 2023 - Nov 2023). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05995834, which also lists 1 linked publication.
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 Aug 2023.