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Special Drug Use-results Surveillance of Scemblix Tablets

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ClinicalTrials.gov Identifier: NCT05421091
Recruitment Status : Recruiting
First Posted : June 16, 2022
Last Update Posted : January 26, 2023
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
Uncontrolled, central registration system, all-case, multicenter, special drug use-results surveillance.

Condition or disease Intervention/treatment
Chronic Myeloid Leukemia Other: Asciminib

Detailed Description:
The objective of this study is to collect data on the occurrence, severity, clinical courses of the safety specifications of asciminib, identify factors etc. involved in occurrence and assess its clinical safety inresistant/intolerant chronic myelogenous leukemia patients during an observational period of 48 weeks from the start of treatment with asciminib.
Study Design
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Study Type : Observational
Estimated Enrollment : 440 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Special Drug Use-results Surveillance of Scemblix Tablets (Resistant or Intolerant Chronic Myeloid Leukemia , CABL001A1401)
Actual Study Start Date : July 4, 2022
Estimated Primary Completion Date : July 15, 2028
Estimated Study Completion Date : July 15, 2028

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Asciminib
Patients prescribed with Asciminib
 Other: Asciminib
Prospective observational study. There is no treatment allocation. Patients prescribed with asciminib are eligible to enroll into this study.


Outcome Measures
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Primary Outcome Measures :
  1. Type, frequency, seriousness and severity of adverse event (AE)/treatment-related AE of the safety specifications [ Time Frame: Up to 48 Weeks ]
    For the safety specifications (myelosuppression, infections, QT interval prolongation, pancreatitis, vascular occlusive events, photosensitivity), type, frequency AE, seriousness, severity of adverse event (AE)/treatment-related AE will be collected

  2. AEs leading to interruption/discontinuation of the safety specifications [ Time Frame: Up to 48 Weeks ]
    For the safety specifications (myelosuppression, infections, QT interval prolongation, pancreatitis, vascular occlusive events, photosensitivity), AEs leading to interruption/discontinuation will be collected

  3. Number of patients with changes in relevant laboratory parameters for the safety specifications [ Time Frame: Up to 48 Weeks ]
    For the safety specifications (myelosuppression, infections, QT interval prolongation, pancreatitis, vascular occlusive events, photosensitivity), number of patients with changes in relevant laboratory parameters will be collected

  4. Frequency of AEs/Treatment-related AEs by patient characteristic factor [ Time Frame: Up to 48 Weeks ]
    Frequency of AEs/Treatment-related AEs by patient characteristic factor will be collected


Secondary Outcome Measures :
  1. Type, frequency, seriousness, severity of AEs/treatment-related AEs of the safety analysis set [ Time Frame: Up to 48 Weeks ]
    Type, frequency, seriousness, severity of AEs/treatment-related AEs of the safety analysis set will be collected

  2. AEs leading to interruption/discontinuation in the safety analysis set [ Time Frame: Up to 48 Weeks ]
    AEs leading to interruption/discontinuation in the safety analysis set will be collected

  3. Frequency of AEs/treatment-related AEs summarized by patient characteristic factor [ Time Frame: Up to 48 Weeks ]
    Frequency of AEs/treatment-related AEs summarized by patient characteristic factor will be collected

  4. Type, frequency, seriousness, severity of AEs/treatment-related AEs in patients with special characteristics [ Time Frame: Up to 48 Weeks ]
    Type, frequency, seriousness, severity of AEs/treatment-related AEs in patients with special characteristics (patients with concurrent renal impairment/hepatic impairment/cardiac impairment, elderly, children, pregnant/parturient women) will be collected

  5. AEs leading to interruption/discontinuation in patients with special characteristics [ Time Frame: Up to 48 Weeks ]
    AEs leading to interruption/discontinuation in patients with special characteristics (patients with concurrent renal impairment/hepatic impairment/cardiac impairment, elderly, children, pregnant/parturient women) will be collected

  6. Type, frequency, seriousness, severity and outcome of AEs/treatment-related AEs by treatment line [ Time Frame: Up to 48 Weeks ]
    Type, frequency, seriousness, severity and outcome of AEs/treatment-related AEs by treatment line will be collected

  7. Factors affecting occurrence of AEs by treatment line [ Time Frame: Up to 48 Weeks ]
    Factors affecting occurrence of AEs by treatment line will be collected

  8. AEs leading to interruption/discontinuation by treatment line [ Time Frame: Up to 48 Weeks ]
    AEs leading to interruption/discontinuation by treatment line will be collected

  9. Major molecular response (MMR) rates [ Time Frame: Week 12, Week 24, Week 48 ]

    Major molecular response is defined as BCR-ABL1 International Scale value ≤ 0.1%.

    BCR-ABL1: translocation-produced fusion gene


  10. MMR rates by Week 48 by patient characteristics factor [ Time Frame: Up to 48 Weeks ]

    Major molecular response (MMR) is defined as BCR-ABL1 International Scale value ≤ 0.1%.

    BCR-ABL1: translocation-produced fusion gene


  11. MR4.0 and MR4.5 rates [ Time Frame: Week 12, Week 24 and Week 48 ]

    MR4.0 and MR4.5 rates are defined as :

    • MR4.0: BCR-ABL1 International Scale value ≤ 0.01%
    • MR4.5: BCR-ABL1 International Scale value ≤ 0.0032%

    BCR-ABL1: translocation-produced fusion gene


  12. Complete cytogenetic response (CCyR) rates [ Time Frame: Week 12, Week 24 and Week 48 ]
    This study will collect complete cytogenetic response (CCyR), which is defined as a state of Ph+ metaphase cell disappearance, i.e. Ph+ cell = 0%.

  13. Complete hematological response (CHR) rates [ Time Frame: Week 12, Week 24 and Week 48 ]

    This study will collect complete hematological response (CHR), which is defined as meeting the following 6 criteria.

    1. White blood cell count < 10,000/µL
    2. Platelet count < 450,000/µL
    3. No blast cell and promyelocyte in peripheral blood
    4. Myelocyte + metamyelocyte in peripheral blood = 0%
    5. Basophil < 5%
    6. No spleen and liver swelling, and no extramedullary lesion

  14. Rate of patients with BCR-ABL1 gene mutations [ Time Frame: Up to 48 Weeks ]
    This study will collect the rate of patients with BCR-ABL1 gene mutations

  15. MMR rates by Week 48 in patients with special characteristics [ Time Frame: Week 48 ]
    This study will collect major molecular response (MMR) rates by Week 48 in patients with special characteristics (patients with concurrent renal impairment/hepatic impairment/cardiac impairment, elderly, children, pregnant/parturient women)

  16. MMR rates by treatment line [ Time Frame: Week 12, Week 24 and Week 48 ]
    This study will collect major molecular response (MMR) rates by treatment line

  17. MR4.0 and MR4.5 rates by treatment line [ Time Frame: Week 12, Week 24 and Week 48 ]

    MR4.0 and MR4.5 rates are defined as :

    • MR4.0: BCR-ABL1 International Scale value ≤ 0.01%
    • MR4.5: BCR-ABL1 International Scale value ≤ 0.0032%

    BCR-ABL1: translocation-produced fusion gene


  18. CCyR rates by treatment line [ Time Frame: Week 12, Week 24 and Week 48 ]
    This study will collect complete cytogenetic response (CCyR), which is defined as a state of Ph+ metaphase cell disappearance, i.e. Ph+ cell = 0%.

  19. CHR rates by treatment line [ Time Frame: Week 12, Week 24 and Week 48 ]

    This study will collect complete hematological response (CHR), which is defined as meeting the following 6 criteria.

    1. White blood cell count < 10,000/µL
    2. Platelet count < 450,000/µL
    3. No blast cell and promyelocyte in peripheral blood
    4. Myelocyte + metamyelocyte in peripheral blood = 0%
    5. Basophil < 5%
    6. No spleen and liver swelling, and no extramedullary lesion


Eligibility Criteria
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Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   up to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients treated with asciminib
Criteria

Inclusion Criteria:

  • patients treated with asciminib in Japan.

Exclusion Criteria:

NA

Contacts and Locations
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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): NCT05421091


Contacts
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Contact: Novartis Pharmaceuticals +81337978748 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Locations
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Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05421091    
Other Study ID Numbers: CABL001A1401
First Posted: June 16, 2022    Key Record Dates
Last Update Posted: January 26, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
chronic myeloid leukemia
CML
Scemblix Tablets
Asciminib
resistant or intolerant CML
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Neoplasms by Histologic Type
 Neoplasms
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases