Clinical trial NCT05169307: CPX-351 Real-World Effectiveness and Safety Study
Official title: CREST UK: CPX-351 Real-World Effectiveness and Safety Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Acute Myeloid Leukemia
Keywords: Acute Myeloid Leukemia
CPX-351
Vyxeos liposomal
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Iqvia Pty Ltd
First posted: 2021-12-23
Enrollment: 147
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with newly diagnosed, t-AML or AML-MRC who have been treated with Vyxeos liposomal in routine UK clinical practice and managed as per standard local practice will be enrolled in the study.

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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: 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
timeFrame: First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months
measure: Overall Survival in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal
timeFrame: First infusion of Vyxeos liposomal (post-August 2018 approval) to date of death, up to approximately 3 years 4 months
Secondary outcomes:
measure: 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
timeFrame: First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months
measure: 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
timeFrame: First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months
measure: Percentage of Participants Transferred for Haematopoietic Stem Cell Transplant (HSCT) in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal
timeFrame: First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months
measure: Overall Survival in Participants Post-HSCT With AML Who Were Treated in Routine Practice With Vyxeos Liposomal
timeFrame: Date of HSCT to date of death, up to approximately 3 years 4 months
measure: Mean Duration of Hospitalization Stay of Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal
timeFrame: First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months
Other outcomes:
Locations:
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
city: Bristol
country: United Kingdom
facility: Bristol Royal Infirmary
city: Derby
country: United Kingdom
facility: University Hospitals Derby and Burton
city: Edinburgh
country: United Kingdom
facility: Western General Hospital (Lothian)
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter
city: Leeds
country: United Kingdom
facility: The Leeds Teaching Hospitals NHS Trust
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary
city: London
country: United Kingdom
facility: University College London Hospitals
city: London
country: United Kingdom
facility: Royal Marsden
city: Middlesbrough
country: United Kingdom
facility: James Cook University Hospital
city: Peterborough
country: United Kingdom
facility: Peterborough Hospital
city: Portsmouth
country: United Kingdom
facility: Queen Alexandra Hospital, Portsmouth
city: Torquay
country: United Kingdom
facility: Torbay and South Devon
city: Wirral
country: United Kingdom
facility: Royal Liverpool/Clatterbridge
city: Wolverhampton
country: United Kingdom
facility: New Cross Hospital - Wolverhampton
Study officials:
References: