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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05469737




Registration number
NCT05469737
Ethics application status
Date submitted
8/07/2022
Date registered
22/07/2022
Date last updated
28/08/2024

Titles & IDs
Public title
A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)
Scientific title
A Phase 2/3, Multicenter, Randomized, Dose Optimization (Part I), Double-blind (Part II) Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG®) Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With IPSS-R Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)
Secondary ID [1] 0 0
U1111-1276-5463
Secondary ID [2] 0 0
CA055-026
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Condition category
Condition code
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 - Oral Azacitidine
Treatment: Drugs - Placebo for Oral Azacitidine

Experimental: Part I - Oral-Aza (Dose 1) -

Experimental: Part I - Oral-Aza (Dose 2) -

Experimental: Part II - Oral-Aza (RP3D) - RP3D: Recommended Phase 3 Dose

Experimental: Part II - Placebo -


Treatment: Drugs: Oral Azacitidine
Specified dose on specified days

Treatment: Drugs: Placebo for Oral Azacitidine
Specified dose on specified days
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with Adverse Events (AEs) evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria v.5.0
Timepoint [1] 0 0
6 cycles plus 28 days (up to 24 weeks)
Primary outcome [2] 0 0
Number of participants who achieved complete remission (CR) per International Working Group (IWG) 2006 criteria within 6 cycles
Timepoint [2] 0 0
Up to 24 weeks
Secondary outcome [1] 0 0
Number of participants who achieved Overall Response (OR) per IWG 2006 criteria within 6 cycles
Timepoint [1] 0 0
Up to 24 weeks
Secondary outcome [2] 0 0
Number of participants who achieved 84-day packed red blood cells transfusion independence (pRBC-TI)
Timepoint [2] 0 0
Up to 32 weeks
Secondary outcome [3] 0 0
pRBC-TI duration
Timepoint [3] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [4] 0 0
Number of participants who achieve 84 day platelet transfusion independence (PLT-TI) within 6 cycles
Timepoint [4] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [5] 0 0
PLT-TI duration
Timepoint [5] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [6] 0 0
Number of participants who achieved pRBC transfusion reduction
Timepoint [6] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [7] 0 0
pRBC transfusion reduction duration
Timepoint [7] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [8] 0 0
CR duration
Timepoint [8] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [9] 0 0
Best OR
Timepoint [9] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [10] 0 0
OR duration
Timepoint [10] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [11] 0 0
Overall Survival (OS)
Timepoint [11] 0 0
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Secondary outcome [12] 0 0
Event-free Survival (EFS)
Timepoint [12] 0 0
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Secondary outcome [13] 0 0
Time to acute myeloid leukemia (AML)
Timepoint [13] 0 0
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Secondary outcome [14] 0 0
Time to subsequent therapy
Timepoint [14] 0 0
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Secondary outcome [15] 0 0
Iron parameters measured from blood
Timepoint [15] 0 0
Over the course of the study, an average of 1 year
Secondary outcome [16] 0 0
Number of participants with Adverse Events (AEs) evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria v.5.0
Timepoint [16] 0 0
Up to end of treatment/early termination, an average of 1 year
Secondary outcome [17] 0 0
Summary statistics for Functional Assessment of Cancer Therapy-Anemia (FACT-An) scales and subscales at each assessment point for each treatment arm
Timepoint [17] 0 0
Up to end of treatment/early termination, an average of 1 year
Secondary outcome [18] 0 0
Summary statistics for Quality of Life in Myelodysplasia Scale (QUALMS) scales and subscales at each assessment point for each treatment arm
Timepoint [18] 0 0
Up to end of treatment/early termination, an average of 1 year
Secondary outcome [19] 0 0
Summary statistics for the EuroQol 5 Dimension 5 Level (EQ-5D-5L) scales and subscales at each assessment point for each treatment arm
Timepoint [19] 0 0
Up to end of treatment/early termination, an average of 1 year
Secondary outcome [20] 0 0
Number of participants with healthcare resource use associated with the investigational product (IP)
Timepoint [20] 0 0
Over the course of the study, an average of 1 year

Eligibility
Key inclusion criteria
• Participant has a documented diagnosis of MDS according to WHO 2016 classification that meets International Prognostic Scoring System Revised (IPSS-R) classification of low- or intermediate-risk disease (IPSS-R score between 1.5 and 4.5).

MDS diagnosis, WHO classification, and IPSS-R risk classification will be prospectively determined by independent central pathology and cytogenetics review, and applicable central laboratory results.

• Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participants with prior malignancies must have an expected median life expectancy of at least 12 months at the time of inclusion and no active treatment of any sort for at least 24 weeks prior to randomization (including but not limited to immunotherapy or targeted therapy)
* Hypoplastic Myelodysplastic Syndrome (MDS) with a marrow cellularity of = 10%
* Participants diagnosed with MDS with excess blasts-2 (MDS-EB2)
* Prior treatment with azacitidine (any formulation), decitabine, or other hypomethylating agent

Other protocol-defined inclusion/exclusion criteria apply

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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
14/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
29/07/2026
Actual
Sample size
Target
230
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Local Institution - 0006 - Clayton
Recruitment hospital [2] 0 0
Local Institution - 0018 - Melbourne
Recruitment hospital [3] 0 0
Local Institution - 0004 - Melbourne
Recruitment hospital [4] 0 0
Local Institution - 0003 - Melbourne
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3000 - Melbourne
Recruitment postcode(s) [3] 0 0
3065 - Melbourne
Recruitment postcode(s) [4] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
United States of America
State/province [5] 0 0
Virginia
Country [6] 0 0
Argentina
State/province [6] 0 0
Buenos Aires
Country [7] 0 0
Argentina
State/province [7] 0 0
Ciudad Autónoma De Buenos Aires
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
China
State/province [10] 0 0
Hubei
Country [11] 0 0
Czechia
State/province [11] 0 0
Hradec Kralove
Country [12] 0 0
Denmark
State/province [12] 0 0
Midtjylland
Country [13] 0 0
Denmark
State/province [13] 0 0
Nordjylland
Country [14] 0 0
France
State/province [14] 0 0
Aquitaine
Country [15] 0 0
France
State/province [15] 0 0
Indre-et-Loire
Country [16] 0 0
France
State/province [16] 0 0
Maine-et-Loire
Country [17] 0 0
France
State/province [17] 0 0
Nord
Country [18] 0 0
France
State/province [18] 0 0
Val-de-Marne
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
Germany
State/province [20] 0 0
Nordrhein-Westfalen
Country [21] 0 0
Germany
State/province [21] 0 0
Sachsen
Country [22] 0 0
Germany
State/province [22] 0 0
Dresden
Country [23] 0 0
Germany
State/province [23] 0 0
Hamburg
Country [24] 0 0
Germany
State/province [24] 0 0
Mutlangen
Country [25] 0 0
Greece
State/province [25] 0 0
Attikí
Country [26] 0 0
Greece
State/province [26] 0 0
Thessaloníki
Country [27] 0 0
Greece
State/province [27] 0 0
Alexandroupolis
Country [28] 0 0
Hong Kong
State/province [28] 0 0
Hksar
Country [29] 0 0
Hong Kong
State/province [29] 0 0
Shatin
Country [30] 0 0
Italy
State/province [30] 0 0
Lazio
Country [31] 0 0
Italy
State/province [31] 0 0
Milano
Country [32] 0 0
Italy
State/province [32] 0 0
Toscana
Country [33] 0 0
Italy
State/province [33] 0 0
Bologna
Country [34] 0 0
Japan
State/province [34] 0 0
Fukuoka
Country [35] 0 0
Japan
State/province [35] 0 0
Hokkaido
Country [36] 0 0
Japan
State/province [36] 0 0
Hyogo
Country [37] 0 0
Japan
State/province [37] 0 0
Kanagawa
Country [38] 0 0
Japan
State/province [38] 0 0
Miyagi
Country [39] 0 0
Japan
State/province [39] 0 0
Tokyo
Country [40] 0 0
Japan
State/province [40] 0 0
Osaka
Country [41] 0 0
Korea, Republic of
State/province [41] 0 0
Jeonranamdo
Country [42] 0 0
Korea, Republic of
State/province [42] 0 0
Seoul Teugbyeolsi
Country [43] 0 0
Korea, Republic of
State/province [43] 0 0
Seoul-teukbyeolsi [Seoul]
Country [44] 0 0
Korea, Republic of
State/province [44] 0 0
Taegu-Kwangyokshi
Country [45] 0 0
Poland
State/province [45] 0 0
Warminsko-mazurskie
Country [46] 0 0
Spain
State/province [46] 0 0
Valenciana, Comunitat
Country [47] 0 0
Spain
State/province [47] 0 0
Granada
Country [48] 0 0
Spain
State/province [48] 0 0
Madrid
Country [49] 0 0
Spain
State/province [49] 0 0
Orense
Country [50] 0 0
Spain
State/province [50] 0 0
Oviedo
Country [51] 0 0
Spain
State/province [51] 0 0
Salamanca
Country [52] 0 0
Sweden
State/province [52] 0 0
Stockholms Län [se-01]
Country [53] 0 0
Sweden
State/province [53] 0 0
Örebro Län [se-18]

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bristol-Myers Squibb
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bristol-Myers Squibb
Address 0 0
Bristol-Myers Squibb
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05469737