An Observational Post Authorisation Study to Evaluate Safety and Efficacy in Patients Receiving Azacitidine in Daily Clinical Practice in the Netherlands (OCEAN)

A Non-interventional Observational Post Authorization Study to Evaluate Safety and Efficacy in Patients Receiving Azacitidine in Daily Clinical Practice in the Netherlands (OCEAN)

The study design is a prospective, non-interventional, observational single arm study.

A minimum of 150 patients will be recruited from approximately 30 haematology/oncology sites in the Netherlands. In all cases, the decision to treat the patient with azacitidine was already made prior to the decision to enter the subject into the study.

Recruitment will continue until end of June 2015, provided a minimum of 150 patients have been included in the study. When this date is reached, all patients on azacitidine will continue to be followed until the last patient enrolled has been followed for 12 months.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic

• Study Type: Observational
• Enrollment (Actual): 209

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients over 18 years of age who are treated with azacitidine in accordance with registered indication and clinical practice. In all cases, the decision to treat the patient with azacitidine was already made prior to the decision to enter the subject into the study

Description

Inclusion Criteria:

• Patients over 18 years of age who understand and voluntarily sign an informed consent form.
• Patients who are treated with azacitidine in accordance with registered indication and clinical practice.

Exclusion Criteria:

• Refusal to participate in the study.
• Participation in an interventional clinical study.
• Patients previously treated with azacitidine except when given as induction therapy for a maximum of three courses.
• Women who are pregnant or breast-feeding.
• Hypersensitivity to the active substance or to any of the excipients.
• Advanced malignant hepatic tumors.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Patients receiving Azacitidine per daily clinical practice

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events (AEs)	Adverse events will be classified using the Medical Drug Regulatory Activities (MedDRA) classification system. The severity of the toxicities will be graded according to the NCI CTCAE VERSION 4.03 whenever possible	Up to approximately 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fact-Anemia Quality of life questionnaire	The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire was used to assess health-related quality of life (HRQoL). In addition to general HRQoL, the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning.	Up to approximately 4 years
Percentage of patients with a Haematological Response in daily clinical practice using the International Work Group Criteria in Myelodysplastic Syndrome Assessed by the Investigator	Hematologic Response according to the 2000 International Working Group (IWG) response criteria for Myelodysplastic Syndrome (MDS)	Up to approximately 4 years
Percentage of patients with a Hematologic Improvement Using International Working Group (IWG Criteria for Hematologic Improvement Cheson 2000) Criteria for Myelodysplastic Syndrome (MDS) and Assessed by the investigator in daily clinical practice	Overall hematological improvement (HI) was defined as any type (major or minor) of improvement of HI-E, HI-P, or HI-N. Criteria: Pretreatment=hemoglobin <100g/L or RBC transfusion-dependent, platelet count <100x10^9/L or platelet transfusion dependent, absolute neutrophil count <1.5x10^9/L. Sponsor's determination was derived using clinically relevant data. Denominator for progression/relapse after HI included participants who had achieved HI.	Up to approximately 4 years
Time to treatment Failure daily clinical practice	Time to Treatment Failure is defined as the time from randomization to treatment discontinuation for any reason, including disease progression, treatment toxicity, patient preference, or death.	Up to approximately 4 years
Overall Survival in daily clinical practice	Overall survival (OS) was assessed using the time between randomization and the date of death	Up to approximately 4 years

Collaborators and Investigators

• Sponsor: Celgene
• Investigators: Study Director: Jan Koedam, MSc, Celgene

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2012
• Primary Completion (Actual): June 30, 2016
• Study Completion (Actual): December 30, 2018

Study Registration Dates

• First Submitted: October 20, 2015
• First Submitted That Met QC Criteria: September 1, 2016
• First Posted (Estimate): September 7, 2016

Study Record Updates

• Last Update Posted (Actual): July 24, 2019
• Last Update Submitted That Met QC Criteria: July 23, 2019
• Last Verified: July 1, 2019

More Information

Terms related to this study

• Keywords: Observational; Leukemia; Chronic; Non-interventional; Acute; azacitidine; Netherlands; OCEAN
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Myelodysplastic-Myeloproliferative Diseases; Myelodysplastic Syndromes; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic
• Other Study ID Numbers: NIPMS-VZ-NL-001