Special Drug Use-results Surveillance of Scemblix Tablets

• Sponsor: Novartis Pharmaceuticals (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05421091
• Collaborator: (none)
• Enrollment: 440
• Locations: 25
• Anticipated Duration (Months): 72.5
• Patients Per Site: 17.6
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

Uncontrolled, central registration system, all-case, multicenter, special drug use-results surveillance.

Condition or Disease	Intervention/Treatment	Phase
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

• Study Type: Observational
• Anticipated 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: Jul 1, 2022
• Anticipated Primary Completion Date: Jul 15, 2028
• Anticipated Study Completion Date: Jul 15, 2028

Arms and Interventions

Arm	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

Primary Outcome Measures

1. Type, frequency, seriousness and severity of adverse event (AE)/treatment-related AE of the safety specifications [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [Up to 48 Weeks]

   Factors affecting occurrence of AEs by treatment line will be collected

8. AEs leading to interruption/discontinuation by treatment line [Up to 48 Weeks]

   AEs leading to interruption/discontinuation by treatment line will be collected

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

    This study will collect complete hematological response (CHR), which is defined as meeting the following 6 criteria. White blood cell count < 10,000/µL Platelet count < 450,000/µL No blast cell and promyelocyte in peripheral blood Myelocyte + metamyelocyte in peripheral blood = 0% Basophil < 5% No spleen and liver swelling, and no extramedullary lesion

14. Rate of patients with BCR-ABL1 gene mutations [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 [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 [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 [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 [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 [Week 12, Week 24 and Week 48]

    This study will collect complete hematological response (CHR), which is defined as meeting the following 6 criteria. White blood cell count < 10,000/µL Platelet count < 450,000/µL No blast cell and promyelocyte in peripheral blood Myelocyte + metamyelocyte in peripheral blood = 0% Basophil < 5% No spleen and liver swelling, and no extramedullary lesion

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 99 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• patients treated with asciminib in Japan.

Exclusion Criteria:

NA

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Toyohashi	Aichi	Japan	441-8111
2	Novartis Investigative Site	Matsuyama-city	Ehime	Japan	790-8524
3	Novartis Investigative Site	Muroran	Hokkaido	Japan	050-0076
4	Novartis Investigative Site	Sapporo	Hokkaido	Japan	060-0004
5	Novartis Investigative Site	Kobe-city	Hyogo	Japan	650-0047
6	Novartis Investigative Site	Kobe-city	Hyogo	Japan
7	Novartis Investigative Site	Kobe	Hyogo	Japan	657-0064
8	Novartis Investigative Site	Nishinomiya	Hyogo	Japan	663 8501
9	Novartis Investigative Site	Takarazuka	Hyogo	Japan	665-0827
10	Novartis Investigative Site	Tsukuba	Ibaraki	Japan	300-2622
11	Novartis Investigative Site	Suwa	Nagano	Japan	392-8510
12	Novartis Investigative Site	Nagasaki-shi	Nagasaki	Japan	852-8511
13	Novartis Investigative Site	Ikoma	Nara	Japan	630-0293
14	Novartis Investigative Site	Tenri	Nara	Japan	632-8552
15	Novartis Investigative Site	Maniwa	Okayama	Japan	719-3105
16	Novartis Investigative Site	Osaka Sayama	Osaka	Japan	589 8511
17	Novartis Investigative Site	Osaka-city	Osaka	Japan	541-8567
18	Novartis Investigative Site	Osaka-city	Osaka	Japan	543-8555
19	Novartis Investigative Site	Nagahama	Shiga	Japan	526-0831
20	Novartis Investigative Site	Matsue	Shimane	Japan	690-8506
21	Novartis Investigative Site	Wakayama-city	Wakayama	Japan	641-8510
22	Novartis Investigative Site	Nara		Japan	630-8305
23	Novartis Investigative Site	Okayama		Japan	700-8557
24	Novartis Investigative Site	Osaka		Japan	540-0008
25	Novartis Investigative Site	Osaka		Japan	542-0081

Sponsors and Collaborators

• Novartis Pharmaceuticals

Investigators

• Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT05421091
• Other Study ID Numbers: CABL001A1401
• First Posted: Jun 16, 2022
• Last Update Posted: Aug 11, 2022
• Last Verified: Aug 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Novartis Pharmaceuticals: chronic myeloid leukemia; CML; Scemblix Tablets; Asciminib; resistant or intolerant CML
• Additional relevant MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Study Results

No Results Posted as of Aug 11, 2022