CPX-351 Real-World Effectiveness and Safety Study
- Registration Number
- NCT05169307
- Lead Sponsor
- Jazz Pharmaceuticals
- Brief Summary
CPX-351 Real World Effectiveness and Safety Study (CREST UK) is a real-world evidence study designed to collect data on the potential benefits and/or risks of Vyxeos liposomal (liposomal daunorubicin/cytarabine; CPX-351) in routine clinical practice in the United Kingdom (UK).
- Detailed Description
This is a retrospective, non interventional, multi-centre, single arm, observational study designed to assess patients with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or acute myeloid leukaemia with myelodysplasia related changes (AML-MRC) who have been treated with Vyxeos liposomal (liposomal daunorubicin/cytarabine; CPX-351) in routine UK clinical practice and managed as per standard local practice.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 147
- Aged ≥18 years at the start of treatment of AML with Vyxeos liposomal
- Pathological diagnosis of t-AML or AML-MRC according to World Health Organization criteria (with at least 20% blasts in the peripheral blood or bone marrow)
- Patient has received at least one infusion of Vyxeos liposomal, prescribed as per the SmPC
- Patient signs an informed consent form or is included in accordance with an informed consent waiver
- Treatment with Vyxeos liposomal as part of a clinical trial or managed access program
- Prior treatment intended for induction therapy of AML; only hydroxyurea is permitted for control of blood counts
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description CPX-351 CPX-351 Adult patients with newly diagnosed, therapy related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) who were treated in routine practice with Vyxeos liposomal in the UK.
- Primary Outcome Measures
Name Time Method Overall Survival in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal First infusion of Vyxeos liposomal (post-August 2018 approval) to date of death, up to approximately 3 years 4 months Number of Participants Achieving Complete Response/Complete Response with Incomplete Platelet or Neutrophil Recovery in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months
- Secondary Outcome Measures
Name Time Method Mean Duration of Hospitalization Stay of Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months Number of Vyxeos Liposomal Induction and Consolidation Cycles Administered (Inpatient or Outpatient) in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months Percentage of Participants Transferred for Haematopoietic Stem Cell Transplant (HSCT) in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months Dose Per Cycle of Vyxeos Liposomal Given During Induction and Consolidation Cycles Administered in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months Overall Survival in Participants Post-HSCT With AML Who Were Treated in Routine Practice With Vyxeos Liposomal Date of HSCT to date of death, up to approximately 3 years 4 months
Trial Locations
- Locations (15)
University Hospitals Derby and Burton
🇬🇧Derby, United Kingdom
Royal Marsden
🇬🇧London, United Kingdom
Bristol Royal Infirmary
🇬🇧Bristol, United Kingdom
Belfast City Hospital
🇬🇧Belfast, United Kingdom
Western General Hospital (Lothian)
🇬🇧Edinburgh, United Kingdom
The Leeds Teaching Hospitals NHS Trust
🇬🇧Leeds, United Kingdom
Royal Devon and Exeter
🇬🇧Exeter, United Kingdom
University College London Hospitals
🇬🇧London, United Kingdom
Leicester Royal Infirmary
🇬🇧Leicester, United Kingdom
James Cook University Hospital
🇬🇧Middlesbrough, United Kingdom
Queen Alexandra Hospital, Portsmouth
🇬🇧Portsmouth, United Kingdom
Peterborough Hospital
🇬🇧Peterborough, United Kingdom
Torbay and South Devon
🇬🇧Torquay, United Kingdom
Royal Liverpool/Clatterbridge
🇬🇧Wirral, United Kingdom
New Cross Hospital - Wolverhampton
🇬🇧Wolverhampton, United Kingdom