Asthma
Conditions
Keywords
Exacerbation, Mepolizumab, NUCALA
Brief summary
This study is a special drug use investigation program of NUCALA (a brand name for Mepolizumab) administered subcutaneously (SC). In this study the information regarding the safety and effectiveness of long term use of NUCALA after subcutaneous injection will be collected from Asthma subjects in daily clinical practice. The observation period per subject will be 52 weeks from the initiation of NUCALA treatment with follow-up investigation for 2 years after the observation period. NUCALA is a registered trademark of the GlaxoSmithKline \[GSK\] group of companies
Interventions
DRUGNUCALA Injection
Single dose of NUCALA SC will be administered. Dose unit, daily dose frequency, date of administration will be at the investigator discretion.
Sponsors
GlaxoSmithKline
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Subjects receiving NUCALA for the first time for treatment of bronchial asthma (a refractory asthma whose symptoms are inadequately controlled despite receiving standard asthma medications)
Exclusion criteria
* No
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Whose Data Entered on Electronic Data Capture (EDC) System | Up to 14 days from the initiation of NUCALA treatment (Day 1) | The number of participants whose data was entered in the EDC system up to 14 days from the initiation of NUCALA treatment (Day 1) has been presented. |
| Percentage of Participants With Adverse Drug Reaction (ADR) | Up to 3 years | ADR is defined as a response to a drug which is noxious and unintended, and which occurred at doses normally used in man for the prophylaxis, diagnosis, or therapy of disease, or for the modifications of physiological function. Percentage of participants with ADR were calculated as the number of participants having a particular ADR divided by total number of participants on NUCALA treatment\*100. Percentage values are rounded-off. |
| Number of Participants Showing Response to the Treatment | Up to 1 year | The number of participants who showed response to the bronchial asthma treatment has been presented. |
| Number of Participants Excluded From Analysis Due to Exacerbation of Asthma | Up to 1 year | The number of participants excluded from analysis due to exacerbation of asthma has been presented. |
| Number of Participants With Adverse Drug Reactions (ADR) | Up to 3 years | ADR is defined as a response to a drug which is noxious and unintended, and which occurred at doses normally used in man for the prophylaxis, diagnosis, or therapy of disease, or for the modifications of physiological function. |
| Percentage of Participants With Occurrences of Safety Specifications and Priority Investigation Matters | Up to 3 years | Safety specifications and priority investigation matters included hypersensitivity reaction including anaphylaxis, infections, and malignant tumor. The percentage of participants with occurrences of safety specifications and priority investigation matters have been reported. Percentage values are rounded-off. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Response Rate Assessed by Global Assessment of Effectiveness | Up to 1 year | Response rate is the percentage of participants assessed as effective based on the course of subjective symptoms and clinical symptoms. Response rate was calculated as number of participants showing response to the NUCALA treatment divided by total number of participants on treatment\*100. Percentage values are rounded-off. Response rate and corresponding 95% confidence interval were reported. |
| Rate of Exacerbation of Asthma | 52 weeks before the initiation of NUCALA treatment (Day 1) and at Week 52 post-treatment | Asthma exacerbation was defined as new or increased asthma symptoms (wheezing, coughing, dyspnea, chest tightness, and/or nighttime awakenings due to these symptoms) that resulted in hospitalization, emergency room visit and usage of systemic steroids. Exacerbation rate was defined as number of asthma exacerbations divided by total person-year (52 weeks were converted to 1 year). Data for exacerbations requiring hospitalization, emergency room visit, and use of systemic corticosteroid have been presented. |
| Total Score of Asthma Control Test (ACT) at Indicated Time Points | Baseline, Weeks 12, 24 and 52 after the initiation of NUCALA treatment (Day 1) | The ACT is a validated self-completed questionnaire utilizing 5 questions to assess asthma control on a 5-point categorical scale ranging from 1 (not controlled at all) to 5 (completely controlled) with higher scores indicating better control. Total ACT score is calculated as the sum of the scores from the 5 questions and can range from 5 to 25, with higher scores indicating better control. An ACT total score of 5 to 19 suggests that the participant's asthma is unlikely to be well controlled, whilst a score of 20 to 25 suggests that the participant's asthma is likely to be well controlled. Baseline was defined as within 8 weeks prior to initiation of NUCALA treatment (Day 1). |
| Mean Peak Expiratory Flow (PEF) at Indicated Time Points | Baseline, Weeks 12, 24 and 52 after the initiation of NUCALA treatment (Day 1) | Peak Expiratory Flow is a person's maximum speed of expiration. PEF was measured using an electronic peak expiratory flow device (ePEF) at Baseline (Day 1), and at Weeks 12, 24, and 54 after the initiation of NUCALA treatment. Baseline was defined as within 8 weeks prior to initiation of NUCALA treatment (Day 1). |
Participant flow
Recruitment details
This non-interventional study aims to collect and assess information regarding the safety and effectiveness of long-term use of NUCALA in asthma participants in daily clinical practice.
Pre-assignment details
A total of 1061 participants were enrolled in the study (Enrolled Set included participants registered within the enrollment period specified in the protocol).
Participants by arm
| Arm | Count |
|---|---|
| Participants With Bronchial Asthma Participants receiving NUCALA for the first time for treatment of bronchial asthma (a refractory asthma whose symptoms were inadequately controlled despite receiving standard asthma medications) in clinical practice were enrolled in this study. No study treatment was administered during conduct of this study. | 1,027 |
| Total | 1,027 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Case report forms were not collected | 21 |
| Overall Study | Did not use the drug | 1 |
| Overall Study | No revisit after the first prescription date | 2 |
| Overall Study | Violation of the enrollment deadline | 10 |
Baseline characteristics
| Characteristic | Participants With Bronchial Asthma |
|---|---|
| Age, Continuous | 62.7 Years STANDARD_DEVIATION 16.1 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1027 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 641 Participants |
| Sex: Female, Male Male | 386 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 3 / 1,027 |
| other Total, other adverse events | 33 / 1,027 |
| serious Total, serious adverse events | 9 / 1,027 |
Outcome results
Primary
Number of Participants Excluded From Analysis Due to Exacerbation of Asthma
The number of participants excluded from analysis due to exacerbation of asthma has been presented.
Time frame: Up to 1 year
Population: Enrolled Set included participants registered within the enrollment period.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Participants With Bronchial Asthma | Number of Participants Excluded From Analysis Due to Exacerbation of Asthma | 16 Participants |
Primary
Number of Participants Showing Response to the Treatment
The number of participants who showed response to the bronchial asthma treatment has been presented.
Time frame: Up to 1 year
Population: Effectiveness Analysis Set consisted of the participants included in safety analysis, participants that do not fall under the participants excluded from efficacy analysis.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Participants With Bronchial Asthma | Number of Participants Showing Response to the Treatment | 866 Participants |
Primary
Number of Participants Whose Data Entered on Electronic Data Capture (EDC) System
The number of participants whose data was entered in the EDC system up to 14 days from the initiation of NUCALA treatment (Day 1) has been presented.
Time frame: Up to 14 days from the initiation of NUCALA treatment (Day 1)
Population: Enrolled Set included participants registered within the enrollment period specified in the protocol.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Participants With Bronchial Asthma | Number of Participants Whose Data Entered on Electronic Data Capture (EDC) System | 1040 Participants |
Primary
Number of Participants With Adverse Drug Reactions (ADR)
ADR is defined as a response to a drug which is noxious and unintended, and which occurred at doses normally used in man for the prophylaxis, diagnosis, or therapy of disease, or for the modifications of physiological function.
Time frame: Up to 3 years
Population: Safety Analysis Set consisted of the participants fixed in the case report form. It included participants that do not fall under the safety analysis exclusion criteria.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Participants With Bronchial Asthma | Number of Participants With Adverse Drug Reactions (ADR) | 42 Participants |
Primary
Percentage of Participants With Adverse Drug Reaction (ADR)
ADR is defined as a response to a drug which is noxious and unintended, and which occurred at doses normally used in man for the prophylaxis, diagnosis, or therapy of disease, or for the modifications of physiological function. Percentage of participants with ADR were calculated as the number of participants having a particular ADR divided by total number of participants on NUCALA treatment\*100. Percentage values are rounded-off.
Time frame: Up to 3 years
Population: Safety Analysis Set consisted of the participants fixed in the case report form (CRF). It included participants that do not fall under the safety analysis exclusion criteria.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Participants With Bronchial Asthma | Percentage of Participants With Adverse Drug Reaction (ADR) | 4.1 Percentage of participants |
Primary
Percentage of Participants With Occurrences of Safety Specifications and Priority Investigation Matters
Safety specifications and priority investigation matters included hypersensitivity reaction including anaphylaxis, infections, and malignant tumor. The percentage of participants with occurrences of safety specifications and priority investigation matters have been reported. Percentage values are rounded-off.
Time frame: Up to 3 years
Population: Safety Analysis Set consisted of the participants fixed in the case report form. It included participants that do not fall under the safety analysis exclusion criteria.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Participants With Bronchial Asthma | Percentage of Participants With Occurrences of Safety Specifications and Priority Investigation Matters | Hypersensitivity reaction including anaphylaxis | 1.2 Percentage of participants |
| Participants With Bronchial Asthma | Percentage of Participants With Occurrences of Safety Specifications and Priority Investigation Matters | Infections | 0.3 Percentage of participants |
| Participants With Bronchial Asthma | Percentage of Participants With Occurrences of Safety Specifications and Priority Investigation Matters | Malignant tumor | 0.2 Percentage of participants |
Secondary
Mean Peak Expiratory Flow (PEF) at Indicated Time Points
Peak Expiratory Flow is a person's maximum speed of expiration. PEF was measured using an electronic peak expiratory flow device (ePEF) at Baseline (Day 1), and at Weeks 12, 24, and 54 after the initiation of NUCALA treatment. Baseline was defined as within 8 weeks prior to initiation of NUCALA treatment (Day 1).
Time frame: Baseline, Weeks 12, 24 and 52 after the initiation of NUCALA treatment (Day 1)
Population: Effectiveness Analysis Set consisted of the participants included in safety analysis, participants that do not fall under the participants excluded from efficacy analysis. Only those participants who were measured and analyzed (i.e., contributed data reported in the table) were included in the Overall Number of Participants Analyzed field. 'Number Analyzed' signifies participants evaluable for the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants With Bronchial Asthma | Mean Peak Expiratory Flow (PEF) at Indicated Time Points | Baseline | 304.4 Liters/minute | Standard Deviation 146.8 |
| Participants With Bronchial Asthma | Mean Peak Expiratory Flow (PEF) at Indicated Time Points | 12 weeks after the initiation of NUCALA treatment | 333.7 Liters/minute | Standard Deviation 150.5 |
| Participants With Bronchial Asthma | Mean Peak Expiratory Flow (PEF) at Indicated Time Points | 24 weeks after the initiation of NUCALA treatment | 334.2 Liters/minute | Standard Deviation 138.7 |
| Participants With Bronchial Asthma | Mean Peak Expiratory Flow (PEF) at Indicated Time Points | 52 weeks after the initiation of NUCALA treatment | 358.9 Liters/minute | Standard Deviation 129.8 |
Secondary
Rate of Exacerbation of Asthma
Asthma exacerbation was defined as new or increased asthma symptoms (wheezing, coughing, dyspnea, chest tightness, and/or nighttime awakenings due to these symptoms) that resulted in hospitalization, emergency room visit and usage of systemic steroids. Exacerbation rate was defined as number of asthma exacerbations divided by total person-year (52 weeks were converted to 1 year). Data for exacerbations requiring hospitalization, emergency room visit, and use of systemic corticosteroid have been presented.
Time frame: 52 weeks before the initiation of NUCALA treatment (Day 1) and at Week 52 post-treatment
Population: Effectiveness Analysis Set consisted of the participants included in safety analysis, participants that do not fall under the participants excluded from efficacy analysis. Only those participants who were measured and analyzed (i.e., contributed data reported in the table) were included in the Overall Number of Participants Analyzed field. 'Number Analyzed' signifies participants evaluable for the specified time points.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Participants With Bronchial Asthma | Rate of Exacerbation of Asthma | Exacerbations requiring hospitalization: 52 weeks before the initiation of NUCALA treatment | 0.4 Asthma exacerbations per person-year |
| Participants With Bronchial Asthma | Rate of Exacerbation of Asthma | Exacerbations requiring hospitalization: at Week 52 post-treatment | 0.1 Asthma exacerbations per person-year |
| Participants With Bronchial Asthma | Rate of Exacerbation of Asthma | Exacerbations requiring emergency room visits: 52 weeks before the initiation of NUCALA treatment | 0.9 Asthma exacerbations per person-year |
| Participants With Bronchial Asthma | Rate of Exacerbation of Asthma | Exacerbations requiring emergency room visits: at Week 52 post-treatment | 0.2 Asthma exacerbations per person-year |
| Participants With Bronchial Asthma | Rate of Exacerbation of Asthma | Exacerbations requiring use of systemic corticosteroid:52weeks before initiation of NUCALA treatment | 3.2 Asthma exacerbations per person-year |
| Participants With Bronchial Asthma | Rate of Exacerbation of Asthma | Exacerbations requiring the use of systemic corticosteroid: at Week 52 post-treatment | 0.7 Asthma exacerbations per person-year |
Secondary
Response Rate Assessed by Global Assessment of Effectiveness
Response rate is the percentage of participants assessed as effective based on the course of subjective symptoms and clinical symptoms. Response rate was calculated as number of participants showing response to the NUCALA treatment divided by total number of participants on treatment\*100. Percentage values are rounded-off. Response rate and corresponding 95% confidence interval were reported.
Time frame: Up to 1 year
Population: Effectiveness Analysis Set consisted of the participants included in safety analysis, participants that do not fall under the participants excluded from efficacy analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Participants With Bronchial Asthma | Response Rate Assessed by Global Assessment of Effectiveness | 90.3 Percentage of participants |
Secondary
Total Score of Asthma Control Test (ACT) at Indicated Time Points
The ACT is a validated self-completed questionnaire utilizing 5 questions to assess asthma control on a 5-point categorical scale ranging from 1 (not controlled at all) to 5 (completely controlled) with higher scores indicating better control. Total ACT score is calculated as the sum of the scores from the 5 questions and can range from 5 to 25, with higher scores indicating better control. An ACT total score of 5 to 19 suggests that the participant's asthma is unlikely to be well controlled, whilst a score of 20 to 25 suggests that the participant's asthma is likely to be well controlled. Baseline was defined as within 8 weeks prior to initiation of NUCALA treatment (Day 1).
Time frame: Baseline, Weeks 12, 24 and 52 after the initiation of NUCALA treatment (Day 1)
Population: Effectiveness Analysis Set consisted of the participants included in safety analysis, participants that do not fall under the participants excluded from efficacy analysis. Only those participants who were measured and analyzed (i.e., contributed data reported in the table) were included in the Overall Number of Participants Analyzed field. 'Number Analyzed' signifies participants evaluable for the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants With Bronchial Asthma | Total Score of Asthma Control Test (ACT) at Indicated Time Points | Baseline | 16.2 Scores on a scale | Standard Deviation 4.9 |
| Participants With Bronchial Asthma | Total Score of Asthma Control Test (ACT) at Indicated Time Points | 12 weeks after the initiation of NUCALA treatment | 20.5 Scores on a scale | Standard Deviation 4.3 |
| Participants With Bronchial Asthma | Total Score of Asthma Control Test (ACT) at Indicated Time Points | 24 weeks after the initiation of NUCALA treatment | 20.9 Scores on a scale | Standard Deviation 4.2 |
| Participants With Bronchial Asthma | Total Score of Asthma Control Test (ACT) at Indicated Time Points | 52 weeks after the initiation of NUCALA treatment | 21.4 Scores on a scale | Standard Deviation 4 |