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Completed

NCT Number: NCT04973280

Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)

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.

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Key information

Conditions

Myelodysplastic Syndromes Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Beta-Thalassemia Bone Marrow Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Sex eligibility

All sexes

Study type

Observational

Primary location

Local Institutions, Vienna, Austria

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Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Primary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. 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

  11. 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)

  12. 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

Secondary outcomes

  1. Awareness of the HCP Checklist

    Time frame: Up to 3 months

    Calculated as the percentage of HCPs who answer "Yes" to question 7

  2. Read/reviewed the HCP Checklist

    Time frame: Up to 3 months

    Calculated as the percentage of HCPs who answer "Yes, all of it" or "Yes, some of it" to question 8

  3. Use of the HCP Checklist prior to initiating treatment

    Time frame: Up to 3 months

    Calculated as percentages of HCPs who answer "Always" or "Sometimes" to question 9A

  4. Use of the HCP Checklist at each subsequent administration

    Time frame: Up to 3 months

    Calculated as percentages of HCPs who answer "Always" or "Sometimes" to question 9B

  5. Use of the HCP Checklist for at least 3 months after stopping treatment

    Time frame: Up to 3 months

    Calculated as percentages of HCPs who answer "Always" or "Sometimes" to question 9C

  6. 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

    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.)

Sponsors and collaborators

Lead sponsor

Celgene

Industry

Registry information

Official study 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)

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Jul 22, 2021
Registry last updated
Jul 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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