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

NCT05883956 | Active Not Recruiting Phase 3

• 1 other identifier: 393-419-00041
• Study Type: interventional
• Target: 13
• Locations: 2 countries
• Sites: 11

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

Outcome Measures

Primary Outcomes (2)

• Proportion of patients reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the patient treatment preference in myelodysplasia questionnaire (pTPMQ)

  Prior to initiation of Cycle 3 (each cycle is 28 days)

• Proportion of patients reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the pTPMQ

  Prior to initiation of Cycle 5 (each cycle is 28 days)

Secondary Outcomes (17)

• Proportion of carers reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the carer treatment preference in myelodysplasia questionnaire (cTPMQ)

  Prior to initiation of Cycle 3 (each cycle is 28 days)

• Proportion of carers reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the cTPMQ

  Prior to initiation of Cycle 5 (each cycle is 28 days)

• Proportion of clinicians reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the medical treatment preference in myelodysplasia questionnaire (mTPMQ)

  Prior to initiation of Cycle 4 (each cycle is 28 days)

• Proportion of clinicians reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the mTPMQ

  End of Study (EOS) Day 28

• Proportion of clinicians choosing oral decitabine/cedazuridine vs subcutaneous azacitidine for continuation of treatment and reasons for the treatment choice based on the mTPMQ

  Cycle 5, Day 1 (each cycle is 28 days)

Study Arms (2)

ABBA

ACTIVE COMPARATOR

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

Drug: Subcutaneous azacitidine; Drug: Oral decitabine/cedazuridine

BAAB

ACTIVE COMPARATOR

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

Drug: Subcutaneous azacitidine; Drug: Oral decitabine/cedazuridine

Interventions

Subcutaneous azacitidine DRUG

Subcutaneous azacitidine, 75mg/m2, 7 days

ABBA; BAAB

Oral decitabine/cedazuridine DRUG

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

ABBA; BAAB

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Otsuka Australia Pharmaceutical Pty Ltd: lead

Study Sites (11)

Calvary Mater Newcastle

Newcastle, New South Wales, Australia

Location

Pindara Private Hospital

Benowa, Queensland, Australia

Location

Townsville Hospital

Townsville, Queensland, Australia

Location

Adelaide Oncology and Haematology

North Adelaide, South Australia, Australia

Location

Grampian Health (Ballarat Base Hospital)

Ballarat Central, Victoria, Australia

Location

Latrobe Regional Hospital

Traralgon, Victoria, Australia

Location

Christchurch Hospital

Christchurch, New Zealand

Location

Dunedin Hospital

Dunedin, New Zealand

Location

Auckland City Hospital

Grafton, New Zealand

Location

Waikato Hospital

Hamilton, New Zealand

Location

North Shore Hospital (Waitemata District Health Board)

Takapuna, New Zealand

Location

MeSH Terms

Conditions

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

Interventions

decitabine and cedazuridine drug combination

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Patient Satisfaction; Treatment Adherence and Compliance; Health Behavior; Behavior

Study Officials

• Anoop Enjeti

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2023
• First Posted: June 1, 2023
• Study Start: December 20, 2023
• Primary Completion: December 6, 2024
• Study Completion: May 1, 2025
• Last Updated: March 20, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (5); AU (6)