Trial Outcomes & Findings for Drug Use Investigation of Somatropin for GHD-ADULTS. (NCT NCT00601419)

NCT ID: NCT00601419

Last Updated: 2014-02-06

Results Overview

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.

• Recruitment status: COMPLETED
• Target enrollment: 230 participants
• Primary outcome timeframe: 6 month
• Results posted on: 2014-02-06

Participant Flow

Participant milestones

Measure	Somatropin Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.
Overall Study STARTED	230
Overall Study COMPLETED	226
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	Somatropin Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.
Overall Study Lost to Follow-up	3
Overall Study Missed Visits	1

Baseline Characteristics

Drug Use Investigation of Somatropin for GHD-ADULTS.

Baseline characteristics by cohort

Measure	Somatropin n=226 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.
Age, Customized <65 years	196 participants n=93 Participants
Age, Customized >=65 years	30 participants n=93 Participants
Sex: Female, Male Female	120 Participants n=93 Participants
Sex: Female, Male Male	106 Participants n=93 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Somatropin n=226 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Number of Participants With Treatment Related Adverse Events.	29 participants	—	—	—

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Somatropin n=226 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Number of Unlisted Treatment Related Adverse Events According to Japanese Package Insert.	8 events	—	—	—

PRIMARY outcome

Timeframe: 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.

To determine whether age is a significant risk factor in the frequency of treatment related adverse events.

Outcome measures

Measure	Somatropin n=196 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=30 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Number of Participants With Treatment Related Adverse Events of Somatropin: <65 Years of Age vs. >=65 Years of Age.	24 participants	5 participants	—	—

PRIMARY outcome

Timeframe: 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.

To determine whether gender is a significant risk factor in the frequency of treatment related adverse events.

Outcome measures

Measure	Somatropin n=106 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=120 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Number of Participants With Treatment Related Adverse Events of Somatropin by Gender.	13 participants	16 participants	—	—

PRIMARY outcome

Timeframe: 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.

To determine whether TSH deficiency is a significant risk factor in the frequency of treatment related adverse events.

Outcome measures

Measure	Somatropin n=169 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=56 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Number of Participants With Treatment Related Adverse Events of Somatropin: With Thyroid Stimulating Hormone (TSH) Deficiency vs. Without TSH Deficiency.	17 participants	12 participants	—	—

PRIMARY outcome

Timeframe: 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.

To determine whether past history of any disease is a significant risk factor in the frequency of treatment related adverse events.

Outcome measures

Measure	Somatropin n=143 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=83 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
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	12 participants	—	—

PRIMARY outcome

Timeframe: 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.

To determine whether initial dose of somatropin is a significant risk factor in the frequency of treatment related adverse events.

Outcome measures

Measure	Somatropin n=133 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=80 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week n=5 Participants Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week n=5 Participants Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Number of Participants With Treatment Related Adverse Events of Somatropin by Initial Dose of Somatropin.	23 participants	4 participants	0 participants	1 participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Somatropin n=199 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Proportion of Participants Achieving Clinical Efficacy. Effective	185 participants	—	—	—
Proportion of Participants Achieving Clinical Efficacy. Not effective	14 participants	—	—	—
Proportion of Participants Achieving Clinical Efficacy. Not evaluable	0 participants	—	—	—

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Somatropin n=174 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=25 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Proportion of Participants Achieving Clinical Efficacy: <65 Years of Age vs. >=65 Years of Age.	165 participants	20 participants	—	—

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Somatropin n=92 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=107 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Proportion of Participants Achieving Clinical Efficacy by Gender.	86 participants	99 participants	—	—

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Somatropin n=128 Participants Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=65 Years n=67 Participants Participants older than or equal to 65 years of age when taking somatropin for adult growth hormone deficiency according to Japanese package insert.	>=0.042 mg/kg/Week and <=0.084 mg/kg/Week Participants taking an initial dose of 0.042 mg/kg/week or more and 0.084 mg/kg/week or less of somatropin for adult growth hormone deficiency according to Japanese package insert.	>0.084 mg/kg/Week Participants taking an initial dose of more than 0.084 mg/kg/week of somatropin for adult growth hormone deficiency according to Japanese package insert.
Proportion of Participants Achieving Clinical Efficacy: With ACTH Deficiency vs. Without ACTH Deficiency.	116 participants	66 participants	—	—

Adverse Events

Somatropin

Serious events: 10 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	Somatropin n=226 participants at risk Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Pituitary tumour benign	0.88% 2/226 • Number of events 2 The frequency of treatment related adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Craniopharyngioma	0.88% 2/226 • Number of events 2 The frequency of treatment related adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Intracranial tumour haemorrhage	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Germ cell cancer	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Metabolism and nutrition disorders Diabetes mellitus	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Nervous system disorders Cerebral haemorrhage	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Hepatobiliary disorders Cholecystitis	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Investigations Intraocular pressure increased	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.

Other adverse events

Measure	Somatropin n=226 participants at risk Participants taking somatropin for adult growth hormone deficiency according to Japanese package insert.
Infections and infestations Pneumonia	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Infections and infestations Fungal skin infection	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Endocrine disorders Hypothyroidism	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Metabolism and nutrition disorders Type 2 diabetes mellitus	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Metabolism and nutrition disorders Hypercholesterolaemia	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Metabolism and nutrition disorders Diabetes mellitus	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Nervous system disorders Somnolence	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Nervous system disorders Headache	0.88% 2/226 • Number of events 2 The frequency of treatment related adverse events.
Respiratory, thoracic and mediastinal disorders Oropharyngeal discomfort	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Hepatobiliary disorders Hepatic function abnormal	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Skin and subcutaneous tissue disorders Acne	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Skin and subcutaneous tissue disorders Alopecia	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Musculoskeletal and connective tissue disorders Arthralgia	1.8% 4/226 • Number of events 4 The frequency of treatment related adverse events.
Musculoskeletal and connective tissue disorders Myalgia	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Musculoskeletal and connective tissue disorders Pain in extremity	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Musculoskeletal and connective tissue disorders Back pain	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
General disorders Feeling abnormal	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
General disorders Injection site haemorrhage	0.88% 2/226 • Number of events 2 The frequency of treatment related adverse events.
General disorders Oedema	0.88% 2/226 • Number of events 2 The frequency of treatment related adverse events.
General disorders Oedema peripheral	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Investigations Insulin-like growth factor increased	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Investigations Blood cholesterol increased	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.
Investigations Blood triglycerides increased	0.44% 1/226 • Number of events 1 The frequency of treatment related adverse events.

Additional Information

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Results disclosure agreements

• Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
• Publication restrictions are in place

Restriction type: OTHER