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Trial record 12 of 12 for:    QUALMS

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)

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ClinicalTrials.gov Identifier: NCT05469737
Recruitment Status : Not yet recruiting
First Posted : July 22, 2022
Last Update Posted : September 16, 2022
Sponsor:
Information provided by (Responsible Party):
Bristol-Myers Squibb

Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of oral azacitidine in participants with low to intermediate International Prognostic Scoring System Revised (IPSS-R) myelodysplastic syndrome (MDS).

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Drug: Oral Azacitidine Drug: Placebo for Oral Azacitidine Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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)
Estimated Study Start Date : September 22, 2022
Estimated Primary Completion Date : December 23, 2025
Estimated Study Completion Date : December 24, 2030

Resource links provided by the National Library of Medicine

Drug Information available for: Azacitidine

Arm Intervention/treatment
Experimental: Part I - Oral-Aza (Dose 1) Drug: Oral Azacitidine
Specified dose on specified days
Other Names:
  • BMS-986345
  • Oral-Aza
  • ONUREG®

Experimental: Part I - Oral-Aza (Dose 2) Drug: Oral Azacitidine
Specified dose on specified days
Other Names:
  • BMS-986345
  • Oral-Aza
  • ONUREG®

Experimental: Part II - Oral-Aza (RP3D)
RP3D: Recommended Phase 3 Dose
Drug: Oral Azacitidine
Specified dose on specified days
Other Names:
  • BMS-986345
  • Oral-Aza
  • ONUREG®

Experimental: Part II - Placebo Drug: Placebo for Oral Azacitidine
Specified dose on specified days




Primary Outcome Measures :
  1. 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 [ Time Frame: 4 cycles plus 28 days (up to 20 weeks) ]
    Phase 2

  2. Number of participants who achieved First Overall Response (OR) [ Time Frame: Up to 16 weeks ]

    Phase 2

    First Overall Response is defined as modified complete response (modCR), partial remission (PR), marrow complete response (mCR), hematologic improvement-erythroid response (HI-E), hematologic improvement-platelet response (HI-P), or hematologic improvement-neutrophil response (HI-N) as per International Working Group (IWG) 2006 criteria


  3. Number of participants who achieved modified complete response (modCR) within 6 cycles [ Time Frame: Up to 24 weeks ]
    Phase 3


Secondary Outcome Measures :
  1. Number of participants who achieved modCR [ Time Frame: Up to 24 weeks ]
    Phase 2

  2. Number of participants who achieved 56-day packed red blood cells-transfusion independence (pRBC-TI) as per IWG 2006 criteria [ Time Frame: Up to 32 weeks ]
    Phase 2 and Phase 3

  3. pRBC-TI duration [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 2 and Phase 3

  4. Number of participants who achieved platelet-transfusion independence (PLT-TI) as per IWG 2006 criteria [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 2 and Phase 3

  5. PLT-TI duration [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 2 and Phase 3

  6. Number of participants who achieved pRBC transfusion reduction [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 3

  7. pRBC transfusion reduction duration [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 3

  8. modCR duration [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 2 and Phase 3

  9. Number of participants who achieved OR within 6 cycles, defined as modCR, PR, mCR, HI-E, HI-P, HI-N as per IWG 2006 criteria [ Time Frame: Up to 24 weeks ]
    Phase 3

  10. Best OR [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 2 and Phase 3

  11. OR duration [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 2 and Phase 3

  12. Overall Survival (OS) [ Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years ]
    Phase 3

  13. Event-free Survival (EFS) [ Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years ]
    Phase 3

  14. Time to acute myeloid leukemia (AML) [ Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years ]
    Phase 3

  15. Time to subsequent therapy [ Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years ]
    Phase 3

  16. Iron parameters measured from blood [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 3

  17. 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 [ Time Frame: Up to end of treatment/early termination, an average of 1 year ]
    Phase 3

  18. Summary statistics for Functional Assessment of Cancer Therapy-Anemia (FACT-An) scales and subscales at each assessment point for each treatment arm [ Time Frame: Up to end of treatment/early termination, an average of 1 year ]
    Phase 3

  19. Summary statistics for Quality of Life in Myelodysplasia Scale (QUALMS) scales and subscales at each assessment point for each treatment arm [ Time Frame: Up to end of treatment/early termination, an average of 1 year ]
    Phase 3

  20. Summary statistics for the EuroQol 5 Dimension 5 Level (EQ-5D-5L) scales and subscales at each assessment point for each treatment arm [ Time Frame: Up to end of treatment/early termination, an average of 1 year ]
    Phase 3

  21. Number of participants with healthcare resource use associated with the investigational product (IP) [ Time Frame: Over the course of the study, an average of 1 year ]
    Phase 3



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participant has a documented diagnosis of MDS according to WHO 2016 classification that meets International Prognostic Scoring System Revised (IPSS-R) classification 17 of low- or intermediate-risk disease (IPSS-R score between 1.5 and 4.5)
  • Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05469737


Contacts
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Contact: BMS Study Connect Contact Center http://www.bmsstudyconnect.com 855-907-3286 Clinical.Trials@bms.com
Contact: First line of the email MUST contain NCT # and Site #.

Locations
Show Show 24 study locations
Sponsors and Collaborators
Bristol-Myers Squibb
Investigators
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Study Director: Bristol-Myers Squibb Bristol-Myers Squibb
Additional Information:
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Responsible Party: Bristol-Myers Squibb
ClinicalTrials.gov Identifier: NCT05469737    
Other Study ID Numbers: CA055-026
U1111-1276-5463 ( Other Grant/Funding Number: WHO )
First Posted: July 22, 2022    Key Record Dates
Last Update Posted: September 16, 2022
Last Verified: September 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bristol-Myers Squibb:
Anemia
Thrombocytopenia
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Azacitidine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors