Dwarfism, Growth Hormone Deficiency
Conditions
Brief summary
Post marketing drug use investigation of Genotropin for GHD-ADULTS.
Detailed description
All the patients whom an investigator prescribes the first Somatropin should be registered consecutively until the number of subjects reaches target number.
Interventions
DRUGSomatropin
Genotropin® 12mg for Injection, Genotropin® MiniQuick for s.c. injection 0.6mg, Genotropin® MiniQuick for s.c. injection 1.0mg, Genotropin® MiniQuick for s.c. injection 1.4mg, Genotropin® 5.3mg. Dosage, Frequency: According to Japanese LPD, The initial dosage is 0.021mg/kg/week as somatropin (genetical recombination) in 6-7 divided doses by s.c. route. The dosage is titrated by 0.084mg/kg/week at a maximum according to the patient's clinical symptoms, and administered in 6-7 divided doses in a week by s.c. route. The dosage may be adjusted according to patient's clinical symptoms and laboratory test results such as serum Insulin-like Growth Factor-I (IGF-I) concentrations. However, the maximum daily dosage shouldn't be higher than 1mg/day. Duration: According to the protocol of A6281286, the duration of the investigation for findings regarding safety and efficacy of a patient is from the first drug administration to the 6 month after the first administration.
Sponsors
Pfizer
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
The patients who were administered Somatropin to treat Adult growth hormone deficiency (limited to severe type).
Exclusion criteria
Patients not administered Somatropin.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Related Adverse Events of Somatropin by Gender. | 6 month | To determine whether gender is a significant risk factor in the frequency of treatment related adverse events. |
| Number of Participants With Treatment Related Adverse Events. | 6 month | Adverse events mean all unfavorable events that occur in participants after administration of somatropin, irrespective of causal relationship to somatropin (including clinically problematic abnormal changes in laboratory test values). Treatment related adverse events were evaluated in company with the causal relationship to somatropin. |
| Number of Unlisted Treatment Related Adverse Events According to Japanese Package Insert. | 6 month | Adverse events mean all unfavorable events that occur in participants after administration of somatropin, irrespective of causal relationship to somatropin (including clinically problematic abnormal changes in laboratory test values). Treatment related adverse events were evaluated in company with the causal relationship to somatropin. Unlisted treatment related adverse events were confirmed with listed adverse drug reaction according to Japanese package insert. |
| Number of Participants With Treatment Related Adverse Events of Somatropin: <65 Years of Age vs. >=65 Years of Age. | 6 month | To determine whether age is a significant risk factor in the frequency of treatment related adverse events. |
| Number of Participants With Treatment Related Adverse Events of Somatropin: With Thyroid Stimulating Hormone (TSH) Deficiency vs. Without TSH Deficiency. | 6 month | To determine whether TSH deficiency is a significant risk factor in the frequency of treatment related adverse events. |
| Number of Participants With Treatment Related Adverse Events of Somatropin: With Past History of Any Disease vs. Without Past History of Any Disease. | 6 month | To determine whether past history of any disease is a significant risk factor in the frequency of treatment related adverse events. |
| Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin. | 6 month | To determine whether initial dose of somatropin is a significant risk factor in the frequency of treatment related adverse events. |
| Proportion of Participants Achieving Clinical Efficacy. | 6 month | Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables. |
| Proportion of Participants Achieving Clinical Efficacy: <65 Years of Age vs. >=65 Years of Age. | 6 month | Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables. |
| Proportion of Participants Achieving Clinical Efficacy by Gender. | 6 month | Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables. |
| Proportion of Participants Achieving Clinical Efficacy: With ACTH Deficiency vs. Without ACTH Deficiency. | 6 month | Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Somatropin Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert. | 226 |
| Total | 226 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 3 |
| Overall Study | Missed Visits | 1 |
Baseline characteristics
| Characteristic | Somatropin |
|---|---|
| Age, Customized <65 years | 196 participants |
| Age, Customized >=65 years | 30 participants |
| Sex: Female, Male Female | 120 Participants |
| Sex: Female, Male Male | 106 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 19 / 226 |
| serious Total, serious adverse events | 10 / 226 |
Outcome results
Primary
Number of Participants With Treatment Related Adverse Events.
Adverse events mean all unfavorable events that occur in participants after administration of somatropin, irrespective of causal relationship to somatropin (including clinically problematic abnormal changes in laboratory test values). Treatment related adverse events were evaluated in company with the causal relationship to somatropin.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Participants With Treatment Related Adverse Events. | 29 participants |
Primary
Number of Participants With Treatment Related Adverse Events of Somatropin: <65 Years of Age vs. >=65 Years of Age.
To determine whether age is a significant risk factor in the frequency of treatment related adverse events.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Participants With Treatment Related Adverse Events of Somatropin: <65 Years of Age vs. >=65 Years of Age. | 24 participants |
| >=65 Years | Number of Participants With Treatment Related Adverse Events of Somatropin: <65 Years of Age vs. >=65 Years of Age. | 5 participants |
Comparison: The risk factor tested was Age. The null hypothesis is that there is no difference between \<65 years and \>=65 years in the frequency of treatment related adverse events.p-value: =0.556Fisher Exact
Primary
Number of Participants With Treatment Related Adverse Events of Somatropin by Gender.
To determine whether gender is a significant risk factor in the frequency of treatment related adverse events.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Participants With Treatment Related Adverse Events of Somatropin by Gender. | 13 participants |
| >=65 Years | Number of Participants With Treatment Related Adverse Events of Somatropin by Gender. | 16 participants |
Comparison: The risk factor tested was Gender. The null hypothesis is that there is no difference between Male and Female in the frequency of treatment related adverse events.p-value: =0.845Fisher Exact
Primary
Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin.
To determine whether initial dose of somatropin is a significant risk factor in the frequency of treatment related adverse events.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin. | 23 participants |
| >=65 Years | Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin. | 4 participants |
| >=0.042 mg/kg/Week and <=0.084 mg/kg/Week | Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin. | 0 participants |
| >0.084 mg/kg/Week | Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin. | 1 participants |
Comparison: The risk factor tested was Initial Dose. The null hypothesis is that there is no association between Initial Dose and the frequency of treatment related adverse events.p-value: =0.037Fisher Exact
Comparison: The risk factor tested was Initial Dose. The null hypothesis is that there is no linear trend in the frequency of treatment related adverse events across increasing levels of initial dose.p-value: =0.063Cochran-Armitage Exact
Primary
Number of Participants With Treatment Related Adverse Events of Somatropin: With Past History of Any Disease vs. Without Past History of Any Disease.
To determine whether past history of any disease is a significant risk factor in the frequency of treatment related adverse events.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Participants With Treatment Related Adverse Events of Somatropin: With Past History of Any Disease vs. Without Past History of Any Disease. | 17 participants |
| >=65 Years | Number of Participants With Treatment Related Adverse Events of Somatropin: With Past History of Any Disease vs. Without Past History of Any Disease. | 12 participants |
Comparison: The risk factor tested was Past history. The null hypothesis is that there is no difference between With past history and Without past history in the frequency of treatment related adverse events.p-value: =0.013Fisher Exact
Primary
Number of Participants With Treatment Related Adverse Events of Somatropin: With Thyroid Stimulating Hormone (TSH) Deficiency vs. Without TSH Deficiency.
To determine whether TSH deficiency is a significant risk factor in the frequency of treatment related adverse events.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Participants With Treatment Related Adverse Events of Somatropin: With Thyroid Stimulating Hormone (TSH) Deficiency vs. Without TSH Deficiency. | 17 participants |
| >=65 Years | Number of Participants With Treatment Related Adverse Events of Somatropin: With Thyroid Stimulating Hormone (TSH) Deficiency vs. Without TSH Deficiency. | 12 participants |
Comparison: The null hypothesis is that there is no difference between With TSH deficiency and Without TSH deficiency in the frequency of treatment related adverse events.p-value: =0.038Fisher Exact
Primary
Number of Unlisted Treatment Related Adverse Events According to Japanese Package Insert.
Adverse events mean all unfavorable events that occur in participants after administration of somatropin, irrespective of causal relationship to somatropin (including clinically problematic abnormal changes in laboratory test values). Treatment related adverse events were evaluated in company with the causal relationship to somatropin. Unlisted treatment related adverse events were confirmed with listed adverse drug reaction according to Japanese package insert.
Time frame: 6 month
Population: The safety analysis population consists of the participants who satisfy the case conditions and in whom administration of this drug was confirmed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Number of Unlisted Treatment Related Adverse Events According to Japanese Package Insert. | 8 events |
Primary
Proportion of Participants Achieving Clinical Efficacy.
Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables.
Time frame: 6 month
Population: The efficacy analysis population basically consists of the evaluable participants in whom clinical responses were assessed comprehensively based on the time profile of variables including IGF-I, blood pressures, pulse rate, lipid metabolism, body composition, QOL, and degree of participant's satisfaction.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Somatropin | Proportion of Participants Achieving Clinical Efficacy. | Effective | 185 participants |
| Somatropin | Proportion of Participants Achieving Clinical Efficacy. | Not effective | 14 participants |
| Somatropin | Proportion of Participants Achieving Clinical Efficacy. | Not evaluable | 0 participants |
Primary
Proportion of Participants Achieving Clinical Efficacy: <65 Years of Age vs. >=65 Years of Age.
Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables.
Time frame: 6 month
Population: The efficacy analysis population basically consists of the evaluable participants in whom clinical responses were assessed comprehensively based on the time profile of variables including IGF-I, blood pressures, pulse rate, lipid metabolism, body composition, QOL, and degree of participant's satisfaction.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Proportion of Participants Achieving Clinical Efficacy: <65 Years of Age vs. >=65 Years of Age. | 165 participants |
| >=65 Years | Proportion of Participants Achieving Clinical Efficacy: <65 Years of Age vs. >=65 Years of Age. | 20 participants |
Comparison: The risk factor tested was Age. The null hypothesis is that there is no difference between \<65 years of age and \>=65 years of age in the efficacy of somatropin.p-value: =0.019Fisher Exact
Primary
Proportion of Participants Achieving Clinical Efficacy by Gender.
Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables.
Time frame: 6 month
Population: The efficacy analysis population basically consists of the evaluable participants in whom clinical responses were assessed comprehensively based on the time profile of variables including IGF-I, blood pressures, pulse rate, lipid metabolism, body composition, QOL, and degree of participant's satisfaction.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Proportion of Participants Achieving Clinical Efficacy by Gender. | 86 participants |
| >=65 Years | Proportion of Participants Achieving Clinical Efficacy by Gender. | 99 participants |
Comparison: The risk factor tested was Gender. The null hypothesis is that there is no difference between male and female in the efficacy of somatropin.p-value: =1Fisher Exact
Primary
Proportion of Participants Achieving Clinical Efficacy: With ACTH Deficiency vs. Without ACTH Deficiency.
Number of participants achieving clinical efficacy / Number of evaluable participants. Clinical efficacy was assessed comprehensively by physicians in three categories, 'effective', 'not effective', and 'not evaluable', based on the time profile of variables.
Time frame: 6 month
Population: The efficacy analysis population basically consists of the evaluable participants in whom clinical responses were assessed comprehensively based on the time profile of variables including IGF-I, blood pressures, pulse rate, lipid metabolism, body composition, QOL, and degree of participant's satisfaction.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin | Proportion of Participants Achieving Clinical Efficacy: With ACTH Deficiency vs. Without ACTH Deficiency. | 116 participants |
| >=65 Years | Proportion of Participants Achieving Clinical Efficacy: With ACTH Deficiency vs. Without ACTH Deficiency. | 66 participants |
Comparison: The risk factor tested was ACTH deficiency. The null hypothesis is that there is no difference between With ACTH deficiency and Without ACTH deficiency in the efficacy of somatropin.p-value: =0.037Fisher Exact