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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05883956




Registration number
NCT05883956
Ethics application status
Date submitted
25/04/2023
Date registered
1/06/2023
Date last updated
6/06/2024

Titles & IDs
Public title
A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia
Scientific title
A Phase 3b, Randomized, Open-Label, Double Crossover Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Adult Patients With IPSS R Intermediate Myelodysplastic Syndrome, Low Blast Acute Myeloid Leukemia, IPSS Intermediate-2 or High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
Secondary ID [1] 0 0
393-419-00041
Universal Trial Number (UTN)
Trial acronym
PREFER-HMA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Leukemia, Myeloid, Acute 0 0
Leukemia, Myelomonocytic, Chronic 0 0
Patient Preference 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Subcutaneous azacitidine
Treatment: Drugs - Oral decitabine/cedazuridine

Active comparator: ABBA - Cycle 1: Oral decitabine/cedazuridine; Cycle 2: Subcutaneous azacitidine; Cycle 3: Subcutaneous azacitidine; Cycle 4: Oral decitabine/cedazuridine

Active comparator: BAAB - Cycle 1: Subcutaneous azacitidine; Cycle 2: Oral decitabine/cedazuridine; Cycle 3: Oral decitabine/cedazuridine; Cycle 4: Subcutaneous azacitidine


Treatment: Drugs: Subcutaneous azacitidine
Subcutaneous azacitidine, 75mg/m2, 7 days

Treatment: Drugs: Oral decitabine/cedazuridine
Oral decitabine 35mg/cedazuridine 100mg, once daily, 5 days
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the patient treatment preference in myelodysplasia questionnaire (pTPMQ)
Timepoint [1] 0 0
Prior to initiation of Cycle 3 (each cycle is 28 days)
Primary outcome [2] 0 0
Proportion of patients reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the pTPMQ
Timepoint [2] 0 0
Prior to initiation of Cycle 5 (each cycle is 28 days)
Secondary outcome [1] 0 0
Proportion of carers reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the carer treatment preference in myelodysplasia questionnaire (cTPMQ)
Timepoint [1] 0 0
Prior to initiation of Cycle 3 (each cycle is 28 days)
Secondary outcome [2] 0 0
Proportion of carers reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the cTPMQ
Timepoint [2] 0 0
Prior to initiation of Cycle 5 (each cycle is 28 days)
Secondary outcome [3] 0 0
Proportion of clinicians reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the medical treatment preference in myelodysplasia questionnaire (mTPMQ)
Timepoint [3] 0 0
Prior to initiation of Cycle 4 (each cycle is 28 days)
Secondary outcome [4] 0 0
Proportion of clinicians reporting preference for oral decitabine/cedazuridine vs subcutaneous azacitidine on the mTPMQ
Timepoint [4] 0 0
End of Study (EOS) Day 28
Secondary outcome [5] 0 0
Proportion of clinicians choosing oral decitabine/cedazuridine vs subcutaneous azacitidine for continuation of treatment and reasons for the treatment choice based on the mTPMQ
Timepoint [5] 0 0
Cycle 5, Day 1 (each cycle is 28 days)
Secondary outcome [6] 0 0
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 patients
Timepoint [6] 0 0
Cycle 1, Day 1 (each cycle is 28 days)
Secondary outcome [7] 0 0
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 patients
Timepoint [7] 0 0
Cycle 1, Day 1 (each cycle is 28 days)
Secondary outcome [8] 0 0
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 patients
Timepoint [8] 0 0
Cycle 3, Day 5 (each cycle is 28 days)
Secondary outcome [9] 0 0
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 patients
Timepoint [9] 0 0
Cycle 3, Day 5 (each cycle is 28 days)
Secondary outcome [10] 0 0
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 patients
Timepoint [10] 0 0
Cycle 4, Day 5 (each cycle is 28 days)
Secondary outcome [11] 0 0
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 patients
Timepoint [11] 0 0
Cycle 4, Day 5 (each cycle is 28 days)
Secondary outcome [12] 0 0
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 patients
Timepoint [12] 0 0
Cycle 5, Day 5 (each cycle is 28 days)
Secondary outcome [13] 0 0
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 patients
Timepoint [13] 0 0
Cycle 5, Day 5 (each cycle is 28 days)
Secondary outcome [14] 0 0
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 patients
Timepoint [14] 0 0
Cycle 6, Day 5 (each cycle is 28 days)
Secondary outcome [15] 0 0
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 patients
Timepoint [15] 0 0
Cycle 6, Day 5 (each cycle is 28 days)
Secondary outcome [16] 0 0
The difference in the incidence of treatment discontinuation and reasons for treatment discontinuation
Timepoint [16] 0 0
Baseline (pre-intervention) through to study completion (up to 6 cycles of treatment where each cycle is 28 days)
Secondary outcome [17] 0 0
Incidence and severity of adverse events upon study physician discretion.
Timepoint [17] 0 0
Baseline (pre-intervention) through to study completion (up to 6 cycles of treatment where each cycle is 28 days)

Eligibility
Key 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:

• 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).
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/12/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/05/2025
Actual
Sample size
Target
Accrual to date
Final
13
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Calvary Mater Newcastle - Newcastle
Recruitment hospital [2] 0 0
Pindara Private Hospital - Benowa
Recruitment hospital [3] 0 0
Townsville Hospital - Townsville
Recruitment hospital [4] 0 0
Adelaide Oncology and Haematology - North Adelaide
Recruitment hospital [5] 0 0
Grampian Health (Ballarat Base Hospital) - Ballarat Central
Recruitment hospital [6] 0 0
Latrobe Regional Hospital - Traralgon
Recruitment postcode(s) [1] 0 0
- Newcastle
Recruitment postcode(s) [2] 0 0
- Benowa
Recruitment postcode(s) [3] 0 0
- Townsville
Recruitment postcode(s) [4] 0 0
- North Adelaide
Recruitment postcode(s) [5] 0 0
- Ballarat Central
Recruitment postcode(s) [6] 0 0
- Traralgon
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Christchurch
Country [2] 0 0
New Zealand
State/province [2] 0 0
Dunedin
Country [3] 0 0
New Zealand
State/province [3] 0 0
Grafton
Country [4] 0 0
New Zealand
State/province [4] 0 0
Hamilton
Country [5] 0 0
New Zealand
State/province [5] 0 0
Takapuna

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Otsuka Australia Pharmaceutical Pty Ltd
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05883956
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Anoop Enjeti
Address 0 0
Calvary Mater Newcastle, Edith Street, Waratah, NSW 2298 Australia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05883956