Myelodysplastic SyndromesNCT04973280
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
An observational study of Myelodysplastic Syndromes and Beta-thalassemia, run by Celgene at 4 sites in 4 countries.
Trial summary
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Myelodysplastic Syndromes, Beta-thalassemia
- How many people
- 187 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 26 Jul 2021to 21 Jul 2022
- 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 Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Healthcare Professionals (HCPs) who were part of the REBLOZYL aRMMs dissemination list and who practice in any of the participating EEA countries.
Who may be able to join
- Healthcare professionals (HCPs) experienced in treatment of haematological diseases who may intend to prescribe REBLOZYL in the participating European countries and were on the target group for dissemination of the additional risk minimization measures (aRMMs).
- The HCP provides permission to share their responses in aggregate with European Medicines Agency (EMA) or National Competent Authorities (NCAs), if requested.
Who cannot join
- HCPs who previously participated in the cognitive pre-testing of the survey questionnaires to be used for the study.
- HCPs who have been direct employees of the marketing authorization holder (MAH), the EMA, or the study vendor within the past 5 years.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: A Study to Evaluate the Effectiveness of the Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs) in the European Economic Area (EEA)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL. A survey will be used to measure the knowledge and comprehension of the REBLOZYL aRMMs among European Economic Area (EEA) based healthcare professionals (HCPs). The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available, potentially including Austria, Germany, Italy, Norway, Sweden, the Netherlands, Poland, and Spain. Additional EEA countries may be included, as needed, based on commercial availability and reimbursement status.
- Conditions
- Myelodysplastic Syndromes, Beta-thalassemia
- Keywords
- REBLOZYL, Myelodysplastic Syndromes, Healthcare Professionals, Post-authorization safety study, Additional educational measures, Beta-thalassemia, Luspatercept
- Medical subject headings
- Myelodysplastic Syndromes, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Healthcare Professionals (HCPs) | The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available. A sample of HCPs from EEA countries who manage care for patients with certain haematologic conditions and who may/do prescribe REBLOZYL will be recruited from the target population of HCPs who were sent the REBLOZYL aRMMs in these countries. The final list of countries to be included may include 1) only countries where reimbursement has been sought and gained, 2) a geographically representative sample (e.g., northern, southern, eastern, and western EU Members States to the degree possible based on the first criteria), 3) a mixture of countries with higher and lower REBLOZYL usage, and 4) other feasibility considerations such as the ability to conduct direct-to-HCP non-market research studies. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Knowledge that although there are no data from the use of REBLOZYL in pregnant women, studies in animals exposed to REBLOZYL have shown reproductive toxicity and embryo-fetal toxicity
Calculated as the percentage of healthcare professionals (HCPs) who answer "True" to question 1A
Measured: Up to 3 months
Knowledge that women of childbearing potential (WCBP) who become pregnant during treatment with REBLOZYL should not continue REBLOZYL treatment even if they receive appropriate monitoring
Calculated as the percentage of HCPs who answer "False" to question 1B
Measured: Up to 3 months
Knowledge that HCPs must counsel WCBP of the potential teratogenic risk of REBLOZYL
Calculated as the percentage of HCPs who answer "True" to question 1C
Measured: Up to 3 months
Knowledge that for WCBP who become pregnant during treatment with REBLOZYL, the pregnancy outcome should be evaluated
Calculated as the percentage of HCPs who answer "False" to question 1D
Measured: Up to 3 months
Knowledge that the use of REBLOZYL is contraindicated during pregnancy and in WCBP who are not using at least 1 highly effective method of contraception
Calculated as the percentage of HCPs who answer "True" to question 1E
Measured: Up to 3 months
Knowledge of what information about contraception methods should be provided when counselling WCBP
Calculated as the percentage of HCPs who answer "WCBP should use at least 1 highly effective method of contraception during treatment with REBLOZYL and for at least 3 months after stopping treatment" to question 2
Measured: Up to 3 months
Knowledge of how many pregnancy tests should be performed for WCBP before they start treatment with REBLOZYL
Calculated as the percentage of HCPs who answer "A single pregnancy test must be performed and medically verified before starting treatment with REBLOZYL" to question 3
Measured: Up to 3 months
Knowledge of the frequency pregnancy tests should be repeated for WCBP who are receiving treatment with REBLOZYL
Calculated as the percentage of HCPs who answer "At suitable intervals and medically verified as negative" to question 4
Measured: Up to 3 months
Knowledge that WCBP should be informed to report a pregnancy that occurs during treatment with REBLOZYL and should receive counselling if they become pregnant
Calculated as the percentage of HCPs who answer "True" to question 5
Measured: Up to 3 months
Knowledge of how long after stopping treatment with REBLOZYL should WCBP be informed to report a pregnancy that occurs and should they receive counselling if they become pregnant
Calculated as the percentage of HCPs who answer "Pregnancies that occur within 3 months after stopping REBLOZYL should be reported and participants should receive counselling" to question 6
Measured: Up to 3 months
Always providing WCBP with the REBLOZYL Patient Card
Calculated as the percentage of HCPs who answer "Yes, always" to providing the REBLOZYL Patient Card to WCBP (question 10-A). The denominator for this variable is the n who answer question 10 (not the n who answer 10-A)
Measured: Up to 3 months
Composite knowledge for all 5 items in the core question set
A composite endpoint calculated as the percentage of HCPs who answer: None of questions 1E, 2, 3, 4, and 10-A correctly; 1 of questions 1E, 2, 3, 4, and 10-A correctly; 2 of questions 1E, 2, 3, 4, and 10-A correctly; 3 of questions 1E, 2, 3, 4, and 10-A correctly; 4 of questions 1E, 2, 3, 4, and 10-A correctly; All 5 of questions 1E, 2, 3, 4, and 10-A correctly
Measured: Up to 3 months
Secondary outcomes (6)
Awareness of the HCP Checklist
Calculated as the percentage of HCPs who answer "Yes" to question 7
Measured: Up to 3 months
Read/reviewed the HCP Checklist
Calculated as the percentage of HCPs who answer "Yes, all of it" or "Yes, some of it" to question 8
Measured: Up to 3 months
Use of the HCP Checklist prior to initiating treatment
Calculated as percentages of HCPs who answer "Always" or "Sometimes" to question 9A
Measured: Up to 3 months
Use of the HCP Checklist at each subsequent administration
Calculated as percentages of HCPs who answer "Always" or "Sometimes" to question 9B
Measured: Up to 3 months
Use of the HCP Checklist for at least 3 months after stopping treatment
Calculated as percentages of HCPs who answer "Always" or "Sometimes" to question 9C
Measured: Up to 3 months
Primary source from which HCPs learned about the appropriate indication, contraindications and precautions for use, and potential risks for REBLOZYL
Calculated as the percentage of HCPs who tick each source in question 11 (for example, percentage of HCPs that ticked the REBLOZYL summary of product characteristics (SmPC), etc.)
Measured: Up to 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2021
- Study start26 Jul 2021
- Primary completion21 Jul 2022
- Study completion21 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by CelgeneThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04973280
- Sponsor's protocol ID
- ACE-536-MDS-005
- Status last verified
- Jul 2024
- First submitted
- 13 Jul 2021
- Last updated
- 12 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Evaluate the Effectiveness of the Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs) in the European Economic Area (EEA)
- ClinicalTrials.gov ID
- NCT04973280
- Acronym / sponsor ID
- ACE-536-MDS-005
- Conditions
- Myelodysplastic Syndromes, Beta-thalassemia
- Keywords
- REBLOZYL, Myelodysplastic Syndromes, Healthcare Professionals, Post-authorization safety study, Additional educational measures, Beta-thalassemia, Luspatercept
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 187 participants (actual)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 26 Jul 2021 (actual)
- Primary completion
- 21 Jul 2022 (actual)
- Study completion
- 21 Jul 2022 (actual)
- First posted
- 22 Jul 2021 (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.
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL. A survey will be used to measure the knowledge and comprehension of the REBLOZYL aRMMs among European Economic Area (EEA) based healthcare professionals (HCPs). The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available, potentially including Austria, Germany, Italy, Norway, Sweden, the Netherlands, Poland, and Spain. Additional EEA countries may be included, as needed, based on commercial availability and reimbursement status.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Healthcare Professionals (HCPs) | – | –The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available. A sample of HCPs from EEA countries who manage care for patients with certain haematologic conditions and who may/do prescribe REBLOZYL will be recruited from the target population of HCPs who were sent the REBLOZYL aRMMs in these countries. The final list of countries to be included may include 1) only countries where reimbursement has been sought and gained, 2) a geographically representative sample (e.g., northern, southern, eastern, and western EU Members States to the degree possible based on the first criteria), 3) a mixture of countries with higher and lower REBLOZYL usage, and 4) other feasibility considerations such as the ability to conduct direct-to-HCP non-market research studies. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Healthcare Professionals (HCPs) who were part of the REBLOZYL aRMMs dissemination list and who practice in any of the participating EEA countries.
Key inclusion criteria (2 listed)
- Healthcare professionals (HCPs) experienced in treatment of haematological diseases who may intend to prescribe REBLOZYL in the participating European countries and were on the target group for dissemination of the additional risk minimization measures (aRMMs).
- The HCP provides permission to share their responses in aggregate with European Medicines Agency (EMA) or National Competent Authorities (NCAs), if requested.
Key exclusion criteria (2 listed)
- HCPs who previously participated in the cognitive pre-testing of the survey questionnaires to be used for the study.
- HCPs who have been direct employees of the marketing authorization holder (MAH), the EMA, or the study vendor within the past 5 years.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Knowledge that although there are no data from the use of REBLOZYL in pregnant women, studies in animals exposed to REBLOZYL have shown reproductive toxicity and embryo-fetal toxicity
Time frame: Up to 3 months
Calculated as the percentage of healthcare professionals (HCPs) who answer "True" to question 1A
Knowledge that women of childbearing potential (WCBP) who become pregnant during treatment with REBLOZYL should not continue REBLOZYL treatment even if they receive appropriate monitoring
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "False" to question 1B
Knowledge that HCPs must counsel WCBP of the potential teratogenic risk of REBLOZYL
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "True" to question 1C
Knowledge that for WCBP who become pregnant during treatment with REBLOZYL, the pregnancy outcome should be evaluated
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "False" to question 1D
Knowledge that the use of REBLOZYL is contraindicated during pregnancy and in WCBP who are not using at least 1 highly effective method of contraception
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "True" to question 1E
Knowledge of what information about contraception methods should be provided when counselling WCBP
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "WCBP should use at least 1 highly effective method of contraception during treatment with REBLOZYL and for at least 3 months after stopping treatment" to question 2
Knowledge of how many pregnancy tests should be performed for WCBP before they start treatment with REBLOZYL
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "A single pregnancy test must be performed and medically verified before starting treatment with REBLOZYL" to question 3
Knowledge of the frequency pregnancy tests should be repeated for WCBP who are receiving treatment with REBLOZYL
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "At suitable intervals and medically verified as negative" to question 4
Knowledge that WCBP should be informed to report a pregnancy that occurs during treatment with REBLOZYL and should receive counselling if they become pregnant
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "True" to question 5
Knowledge of how long after stopping treatment with REBLOZYL should WCBP be informed to report a pregnancy that occurs and should they receive counselling if they become pregnant
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "Pregnancies that occur within 3 months after stopping REBLOZYL should be reported and participants should receive counselling" to question 6
Always providing WCBP with the REBLOZYL Patient Card
Time frame: Up to 3 months
Calculated as the percentage of HCPs who answer "Yes, always" to providing the REBLOZYL Patient Card to WCBP (question 10-A). The denominator for this variable is the n who answer question 10 (not the n who answer 10-A)
Composite knowledge for all 5 items in the core question set
Time frame: Up to 3 months
A composite endpoint calculated as the percentage of HCPs who answer: None of questions 1E, 2, 3, 4, and 10-A correctly; 1 of questions 1E, 2, 3, 4, and 10-A correctly; 2 of questions 1E, 2, 3, 4, and 10-A correctly; 3 of questions 1E, 2, 3, 4, and 10-A correctly; 4 of questions 1E, 2, 3, 4, and 10-A correctly; All 5 of questions 1E, 2, 3, 4, and 10-A correctly
6 secondary outcomes
Awareness of the HCP Checklist
Time frame: Up to 3 months
Read/reviewed the HCP Checklist
Time frame: Up to 3 months
Use of the HCP Checklist prior to initiating treatment
Time frame: Up to 3 months
Use of the HCP Checklist at each subsequent administration
Time frame: Up to 3 months
Use of the HCP Checklist for at least 3 months after stopping treatment
Time frame: Up to 3 months
Primary source from which HCPs learned about the appropriate indication, contraindications and precautions for use, and potential risks for REBLOZYL
Time frame: Up to 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2024.
- BMS Clinical Trial InformationLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html
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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.
Bristol-Myers Squibb study director
Bristol-Myers Squibb
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes and Beta-thalassemia, run by Celgene at 4 sites in 4 countries.
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 2 inclusion and 2 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: finished, as of the sponsor's last update on 12 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Austria, Belgium, Germany, Italy. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Knowledge that although there are no data from the use of REBLOZYL in pregnant women, studies in animals exposed to REBLOZYL have shown reproductive toxicity and embryo-fetal toxicity (time frame: Up to 3 months). The study lists 12 primary outcomes in all. 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 12 Jul 2024.