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

Phase 3
Active, not recruiting
Conditions
Patient Preference
Leukemia, Myelomonocytic, Chronic
Myelodysplastic Syndromes
Leukemia, Myeloid, Acute
Interventions
Drug: Oral decitabine/cedazuridine
Registration Number
NCT05883956
Lead Sponsor
Otsuka Australia Pharmaceutical Pty Ltd
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
13
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.

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.
  • Patient must be able to identify a carer to participate in completing the cTPMQ.

For Carers:

• Primary carer of a patient meeting all of the inclusion criteria (ie, a patient who meets criteria defined above).

For Clinicians:

• Clinician treating patients meeting all of the inclusion criteria (ie, treats patients who meet criteria defined above).

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 carer of a patient who meets any of the exclusion criteria listed above.
  • 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).

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
ABBASubcutaneous azacitidineCycle 1: Oral decitabine/cedazuridine; Cycle 2: Subcutaneous azacitidine; Cycle 3: Subcutaneous azacitidine; Cycle 4: Oral decitabine/cedazuridine
ABBAOral decitabine/cedazuridineCycle 1: Oral decitabine/cedazuridine; Cycle 2: Subcutaneous azacitidine; Cycle 3: Subcutaneous azacitidine; Cycle 4: Oral decitabine/cedazuridine
BAABOral decitabine/cedazuridineCycle 1: Subcutaneous azacitidine; Cycle 2: Oral decitabine/cedazuridine; Cycle 3: Oral decitabine/cedazuridine; Cycle 4: Subcutaneous azacitidine
BAABSubcutaneous azacitidineCycle 1: Subcutaneous azacitidine; Cycle 2: Oral decitabine/cedazuridine; Cycle 3: Oral decitabine/cedazuridine; Cycle 4: Subcutaneous azacitidine
Primary Outcome Measures
NameTimeMethod
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 pTPMQPrior to initiation of Cycle 5 (each cycle is 28 days)
Secondary Outcome Measures
NameTimeMethod
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 cTPMQPrior to initiation of Cycle 5 (each cycle is 28 days)
Difference in quality of life between oral decitabine/cedazuridine and subcutaneous azacitidine as assessed using the EQ-5D-5L in Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myeloid Leukemia patientsCycle 6, Day 5 (each cycle is 28 days)
Difference in quality of life between oral decitabine/cedazuridine and subcutaneous azacitidine as assessed using the EORTC QLQ-C30 in Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myeloid Leukemia patientsCycle 6, Day 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 choosing oral decitabine/cedazuridine vs subcutaneous azacitidine for continuation of treatment and reasons for the treatment choice based on the mTPMQCycle 5, Day 1 (each cycle is 28 days)
Incidence and severity of adverse events upon study physician discretion.Baseline (pre-intervention) through to study completion (up to 6 cycles of treatment where each cycle is 28 days)
Proportion of clinicians reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the mTPMQEnd of Study (EOS) Day 28
Difference in quality of life between oral decitabine/cedazuridine and Subcutaneous azacitidine as assessed using the EORTC QLQ-C30 in Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myeloid Leukemia patientsCycle 4, Day 5 (each cycle is 28 days)
Difference in quality of life between oral decitabine/cedazuridine and Subcutaneous azacitidine as assessed using the EQ-5D-5L in Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myeloid Leukemia patientsCycle 4, Day 5 (each cycle is 28 days)
The difference in the incidence of treatment discontinuation and reasons for treatment discontinuationBaseline (pre-intervention) through to study completion (up to 6 cycles of treatment where each cycle is 28 days)

Trial Locations

Locations (11)

Calvary Mater Newcastle

🇦🇺

Newcastle, New South Wales, Australia

Pindara Private Hospital

🇦🇺

Benowa, Queensland, Australia

Townsville Hospital

🇦🇺

Townsville, Queensland, Australia

Grampian Health (Ballarat Base Hospital)

🇦🇺

Ballarat Central, Victoria, Australia

Adelaide Oncology and Haematology

🇦🇺

North Adelaide, South Australia, 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

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