Special Drug Use-results Surveillance of Scemblix Tablets

Completed

• Conditions: Chronic Myeloid Leukemia
• Interventions: Other: Asciminib

• Registration Number: NCT05421091
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

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 Timeline

• Study First Submitted: 2022-06-13
• Study First Submitted QC: 2022-06-13
• Study First Posted: 2022-06-16
• Study Start: 2022-07-04
• Primary Completion: 2024-02-29
• Study Completion: 2024-02-29
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-26
• Last Update Posted: 2024-03-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 550

Inclusion Criteria

• patients treated with asciminib in Japan.

Exclusion Criteria

NA

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Asciminib	Asciminib	Patients prescribed with Asciminib

Primary Outcome Measures

Name	Time	Method
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
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
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
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

Secondary Outcome Measures

Name	Time	Method
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
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
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
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
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
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.; 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
AEs leading to interruption/discontinuation by treatment line	Up to 48 Weeks	AEs leading to interruption/discontinuation by treatment line will be collected
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
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)
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
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
Factors affecting occurrence of AEs by treatment line	Up to 48 Weeks	Factors affecting occurrence of AEs by treatment line will be collected
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
MMR rates by treatment line	Week 12, Week 24 and Week 48	This study will collect major molecular response (MMR) rates by treatment line
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%.
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
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.; 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
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
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%.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇯🇵 Yamagata, Japan