A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia

NCT ID: NCT05883956

Last Updated: 2025-03-20

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-12-20
• Study Completion Date: 2025-05-31

Brief Summary

It is hypothesized that significantly more patients would prefer oral decitabine/cedazuridine to subcutaneous (SC) azacitidine (AZA) due to several factors, including improved treatment convenience, the reduced risk of nosocomial infections, and reduced treatment discomfort. However, this hypothesis has not been formally studied in a controlled setting. This study aims to address this evidence gap and evaluate patient, primary caregiver (carer), and clinician treatment preference between oral decitabine/cedazuridine and SC AZA in the treatment of adult patients with International Prognostic Scoring System-Revised (IPSS-R) intermediate, IPSS intermediate-2, or high-risk myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or low-blast (LB) acute myeloid leukemia (AML) and thereby lend further credibility to the clinical, economic, and patient value of oral decitabine/cedazuridine.

Conditions

Myelodysplastic Syndromes; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic; Patient Preference

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ABBA

Cycle 1: Oral decitabine/cedazuridine; Cycle 2: Subcutaneous azacitidine; Cycle 3: Subcutaneous azacitidine; Cycle 4: Oral decitabine/cedazuridine

Group Type: ACTIVE_COMPARATOR

Subcutaneous azacitidine

Intervention Type: DRUG

Subcutaneous azacitidine, 75mg/m2, 7 days

Oral decitabine/cedazuridine

Intervention Type: DRUG

Oral decitabine 35mg/cedazuridine 100mg, once daily, 5 days

BAAB

Cycle 1: Subcutaneous azacitidine; Cycle 2: Oral decitabine/cedazuridine; Cycle 3: Oral decitabine/cedazuridine; Cycle 4: Subcutaneous azacitidine

Group Type: ACTIVE_COMPARATOR

Subcutaneous azacitidine

Intervention Type: DRUG

Subcutaneous azacitidine, 75mg/m2, 7 days

Oral decitabine/cedazuridine

Intervention Type: DRUG

Oral decitabine 35mg/cedazuridine 100mg, once daily, 5 days

Interventions

Subcutaneous azacitidine

Subcutaneous azacitidine, 75mg/m2, 7 days

Intervention Type: DRUG

Oral decitabine/cedazuridine

Oral decitabine 35mg/cedazuridine 100mg, once daily, 5 days

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

For Patients:

Patients are eligible to be included in the study only if all of the following criteria apply at any time starting from Screening up to Day 1 prior to study treatment administration:

• Patients must be 18 years of age or older.
• IPSS-R defined intermediate MDS, IPSS defined intermediate 2 or high-risk MDS, LB AML or CMML (with symptoms), as confirmed by recent full blood examination, bone marrow biopsy, and cytogenetic testing. NOTE: IPSS-R defined intermediate MDS patients are limited to less than 50% of enrolled patients.
• Life expectancy of at least 6 months.
• Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2 inclusive.
• Patient must be able to co-operate and complete tasks (including tasks such as electronic questionnaires on digital devices) over the following 4 months.

For Patients:

Patients are eligible to be included in the study only if all of the following criteria apply at any time starting from Screening up to Day 1 prior to study treatment administration:

• Patients must be 18 years of age or older.
• IPSS-R defined intermediate MDS, IPSS defined intermediate 2 or high-risk MDS, LB AML or CMML (with symptoms), as confirmed by recent full blood examination, bone marrow biopsy, and cytogenetic testing. NOTE: IPSS-R defined intermediate MDS patients are limited to less than 50% of enrolled patients.
• Life expectancy of at least 6 months.
• Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2 inclusive.
• Patient must be able to co-operate and complete tasks (including tasks such as electronic questionnaires on digital devices) over the following 4 months.
• Patient must be able to identify a carer to participate in completing the cTPMQ.

For Carers:

For Clinicians:

Exclusion Criteria

For Patients:

Patients are excluded from the study if any of the following criteria apply:

• Patients with known hypersensitivity to the study treatments oral decitabine/cedazuridine or azacitidine.
• Patients with advanced malignant hepatic tumors.
• Patients with severe renal impairment (creatinine clearance <30 mL/min).
• Patients who have received hypomethylating agents (HMA) previously.
• Patients who are receiving lenalidomide or are receiving other therapies outside of standard of care (SOC).
• Receipt of any immunotherapy, any conventional or investigational systemic anti-cancer therapy within 5 half-lives of the drug, or within 4 weeks prior to the first dose of study treatment (whichever is longer).
• Any medical, psychological, social, or other condition which in the view of the Investigator is likely to interfere with the study, compliance, or put the patient at risk.
• Participants who are not fluent in English, or who cannot read or write in English will be excluded from the study.

For Carers:

Carers are excluded from the study if any of the following criteria apply:

• They are a relative of an employee of the investigational clinic or sponsor (e.g. Investigator, study coordinator)

For Clinicians:

• Clinicians will be excluded from participating in the study if they are a relative of an employee of the investigational clinic or sponsor (e.g. Investigator, Study Coordinator).

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Otsuka Australia Pharmaceutical Pty Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anoop Enjeti

Role: PRINCIPAL_INVESTIGATOR

Calvary Mater Newcastle, Edith Street, Waratah, NSW 2298 Australia

Locations

Calvary Mater Newcastle

Newcastle, New South Wales, Australia

Pindara Private Hospital

Benowa, Queensland, Australia

Townsville Hospital

Townsville, Queensland, Australia

Adelaide Oncology and Haematology

North Adelaide, South Australia, Australia

Grampian Health (Ballarat Base Hospital)

Ballarat Central, Victoria, Australia

Latrobe Regional Hospital

Traralgon, Victoria, Australia

Christchurch Hospital

Christchurch, , New Zealand

Dunedin Hospital

Dunedin, , New Zealand

Auckland City Hospital

Grafton, , New Zealand

Waikato Hospital

Hamilton, , New Zealand

North Shore Hospital (Waitemata District Health Board)

Takapuna, , New Zealand

Countries

Australia; New Zealand

Other Identifiers

393-419-00041

Identifier Type: -

Identifier Source: org_study_id