Genotropin Treatment in Short Prepubertal Children With I... | NCT01073605

NCT01073605

ID	Type	Description	Link
A6281186, CTN 93-8122-001

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Genotonorm A	Active Comparator	Continuous 0.7 IU/kg/week or 0.03 mg/kg/day	Drug: Genotonorm
Genotonorm B	Active Comparator	Continuous, 1.4 IU/kg/week or 0.06 mg/kg/day	Drug: Genotonorm
Genotonorm C	Active Comparator	Intermittent, 1.4 IU/kg/week or 0.06 mg/kg/day	Drug: Genotonorm

Name	Type	Description	Arm Group Labels	Other Names
Genotonorm	Drug	0.7 IU/kg/week or 0.03 mg/kg/day, daily subcutaneous injection	Genotonorm A

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change From Baseline in Annual Growth Rate Measured at 2 Years Following Treatment With Genotonorm	Annual growth rate was expressed as height velocity (centimeter [cm]/year). This was derived by substracting annual growth rate at Baseline from 2-year value. (Annual growth rate was calculated each year and rescaled to 1 year if the interval between x and x-1 was not 365 days, as long as a subject remains in the study): ANGRYx = (Height Yx - Height Y{x-1}) / {(Date of Yx - Date of Y{x-1}) /365.25}	Baseline, 2 years

Secondary Outcomes

Measure	Description	Time Frame
Annual Growth Rate Standard Deviation Score (SDS)	Calculated using Sempe reference means and standard deviations for growth rate according to age and sex. Standardization was performed for chronological age.	Baseline, 1 to 6 years
Change From Baseline in Annual Growth Rate SDS

Eligibility Module

Contacts/Locations Module

Name	Affiliation	Role
Pfizer CT.gov Call Center	Pfizer	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Pfizer Investigational Site		Angers	France	49933	France
Pfizer Investigational Site

References Module

Label	URL
To obtain contact information for a study center near you, click here.	View source

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 international unit (IU)/kilogram (kg)/week (0.03 milligram [mg]/kg/day) of Genotonorm growth hormone (GH) as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
FG001

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

Masking Info

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
BG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
BG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.
BG003	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Customized

Number

Participants

Title

Denominators

Categories

Title	Measurements
	OG0003.07± 2.21 OG0013.97± 1.72 OG0022.84± 1.71

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		Wilcoxon (Mann-Whitney)		0.02922	P-values for the 3 treatment comparisons should be interpreted in line with the Bonferroni stepwise (Holm) testing procedure, to maintain the Type 1 error at p=0.05.					2-Sided							No	Superiority or Other
OG000 OG002		Wilcoxon (Mann-Whitney)

Secondary

Annual Growth Rate Standard Deviation Score (SDS)

Calculated using Sempe reference means and standard deviations for growth rate according to age and sex. Standardization was performed for chronological age.

FAS. Number of Participants Analyzed = number of subjects with evaluable data at Baseline. n = number of subjects with evaluable data at each time point. Data beyond 6 years are not reported due to the low proportion of subjects followed up beyond 6 years.

Posted

Mean

Standard Deviation

SDS

Baseline, 1 to 6 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Secondary

Change From Baseline in Annual Growth Rate SDS

Calculated corresponding to the gender and chronological age by substracting annual growth rate SDS at Baseline from annual growth rate SDS at each year.

FAS. Number of Participants Analyzed = number of subjects with evaluable data at 1 year. n = number of subjects with evaluable data at each time point.

Posted

Mean

Standard Deviation

SDS

Baseline, 1 to 3 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Secondary

Height (cm)

Performed by use of a wallmounted device (eg, Harpenden Stadiometer). Each subject was measured 3 times and the mean of these measurements was recorded as the present height. Final Height: Children are defined as reaching their final height when annual Growth Rate is less than 2 cm in the previous year and bone age is equal to or greater than 17 years in boys and equal to or greater than 15 years in girls.

Posted

Mean

Standard Deviation

Baseline, 1 to 6 years, final height

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)

Secondary

Change From Baseline in Height (cm)

Calculated by substracting height at Baseline from height at each year. Final Height: Children are defined as reaching their final height when annual Growth Rate is less than 2 cm in the previous year and bone age is equal to or greater than 17 years in boys and equal to or greater than 15 years in girls.

FAS. Number of Participants Analyzed = number of subjects with evaluable data at Baseline. n = number of subjects with evaluable data at Baseline and each time point. Data beyond 6 years are not reported due to the low proportion of subjects followed up beyond 6 years.

Posted

Mean

Standard Deviation

Baseline, 1 to 6 years, final height

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)

Secondary

Height (SDS)

Calculated using Sempe reference means and standard deviations for height. Final Height: Children are defined as reaching their final height when annual Growth Rate is less than 2 cm in the previous year and bone age is equal to or greater than 17 years in boys and equal to or greater than 15 years in girls.

Posted

Mean

Standard Deviation

SDS

Baseline, 1 to 6 years, final height

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)

Secondary

Change From Baseline in Height (SDS)

Calculated by substracting height SDS at Baseline from height SDS at each year. Final Height: Children are defined as reaching their final height when annual Growth Rate is less than 2 cm in the previous year and bone age is equal to or greater than 17 years in boys and equal to or greater than 15 years in girls.

Posted

Mean

Standard Deviation

SDS

Baseline, 1 to 6 years, final height

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)

Secondary

Body Mass Index (BMI)

BMI was calculated by weight divided by height squared.

Posted

Mean

Standard Deviation

kg/m^2

Baseline, 1 to 6 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Secondary

Weight

Posted

Mean

Standard Deviation

Baseline, 1 to 6 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Secondary

Change From Baseline in Bone Age

Bone age was determined by the Greulich-Pyle method. Calculated by substracting bone age at Baseline from bone age at each year

Safety population = all subjects who received at least 1 study dose of GH. Number of Participants Analyzed = number of subjects with evaluable data at Baseline. n = number of subjects with evaluable data at Baseline and each time point.

Posted

Mean

Standard Deviation

Years

Baseline, 1 to 3 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Secondary

Change From Baseline in Bone Age/Change From Baseline in Chronological Age Ratio

Bone age was determined by the Greulich-Pyle method. Chronological Age (years) was calculated as: (Date minus Date of Birth) divided by 365.25. Chronological Age used was the age at the date that the corresponding Bone Age X-ray was performed. Ratio was calculated by change from Baseline in bone age divided by change from Baseline in chronological age.

Safety population = all subjects who received at least 1 study dose of GH. Number of Participants Analyzed = number of subjects with evaluable data at 1 year. n = number of subjects with evaluable data at each time point.

Posted

Mean

Standard Deviation

ratio

1 to 3 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)

Secondary

Chronological Age at Onset of Puberty

Chronological age (years) at first study visit with onset of puberty = (Date of study visit minus Date of Birth) divided by 365.25.

Safety population. Number of participants analyzed = number of subjects who started puberty by the end of the study.

Posted

Mean

Standard Deviation

Years

Onset of puberty

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Secondary

Number of Subjects Reaching Puberty

The defined criteria for reaching puberty were: boy=if right or left testes volume ≥4 ml; girl=if breast development ≥2. Tanner Adolescent Pubertal Staging Questionnaire documents the stage of development of secondary sexual characteristics rated in 5 stages: stage 1 (no development) to 5 (adult-like development in quantity and size). Onset of puberty was defined as the visit where the data recorded first met the above criteria for starting puberty.

Safety population. Number of Participants Analyzed = number of subjects with evaluable data at Baseline. Data beyond 6 years are not reported due to the low proportion of subjects followed up beyond 6 years. Started = started puberty; Not Started = not started puberty yet as per Tanner scale.

Posted

Number

participants

Baseline, 1 to 6 years

ID	Title	Description
OG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 IU/kg/week (0.03 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.
OG002

Arm/Groups

ID	Title	Description	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Genotonorm 0.7 (Continuous Treatment)	Subjects received 0.7 international unit (IU)/kilogram (kg)/week (0.03 milligram [mg]/kg/day) of Genotonorm growth hormone (GH) as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.	0	68	40	68
EG001	Genotonorm 1.4 (Continuous Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as a continuous treatment. Treatment was allowed to be taken until final height of the subject had been reached.	4	70	42	70
EG002	Genotonorm 1.4 (Intermittent Treatment)	Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.	3	68	35	68

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Tonsillectomy	Surgical and medical procedures	MedDRA 12.0	Systematic Assessment		EG0000 affected68 at risk EG0014 affected70 at risk EG0023 affected68 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Abdominal pain	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0013 affected70 at risk EG0024 affected68 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0005 affected68 at risk EG0010 affected70 at risk EG0024 affected68 at risk
Pyrexia	General disorders	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0013 affected70 at risk EG0023 affected68 at risk
Bronchitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG00013 affected68 at risk EG0019 affected70 at risk EG00217 affected68 at risk
Ear infection	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG00015 affected68 at risk EG00115 affected70 at risk EG0029 affected68 at risk
Fungal infection	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0011 affected70 at risk EG0020 affected68 at risk
Gastroenteritis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0018 affected70 at risk EG00211 affected68 at risk
Influenza	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0006 affected68 at risk EG0019 affected70 at risk EG0027 affected68 at risk
Nasopharyngitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG00016 affected68 at risk EG00116 affected70 at risk EG00211 affected68 at risk
Pharyngitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0002 affected68 at risk EG0016 affected70 at risk EG0027 affected68 at risk
Rhinitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0012 affected70 at risk EG0026 affected68 at risk
Tracheitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0003 affected68 at risk EG0015 affected70 at risk EG0022 affected68 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG00019 affected68 at risk EG00119 affected70 at risk EG00212 affected68 at risk
Varicella	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0008 affected68 at risk EG0016 affected70 at risk EG0021 affected68 at risk
Scoliosis	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected68 at risk EG0014 affected70 at risk EG0020 affected68 at risk
Convulsion	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected68 at risk EG0014 affected70 at risk EG0020 affected68 at risk
Headache	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected68 at risk EG0015 affected70 at risk EG0023 affected68 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0005 affected68 at risk EG0013 affected70 at risk EG0023 affected68 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected68 at risk EG0013 affected70 at risk EG0025 affected68 at risk
Eczema	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0011 affected70 at risk EG0021 affected68 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0004 affected68 at risk EG0010 affected70 at risk EG0021 affected68 at risk

ID	Term
D006130	Growth Disorders
D005317	Fetal Growth Retardation

Ancestor Terms

ID	Term
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms
D005315	Fetal Diseases
D011248	Pregnancy Complications
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ID	Term
D019382	Human Growth Hormone

Ancestor Terms

ID	Term
D013006	Growth Hormone
D010908	Pituitary Hormones, Anterior
D010907	Pituitary Hormones
D036361	Peptide Hormones
D006728	Hormones
D006730	Hormones, Hormone Substitutes, and Hormone Antagonists
D010455	Peptides
D000602	Amino Acids, Peptides, and Proteins

Subjects received 1.4 IU/kg/week (0.06 mg/kg/day) of the growth hormone Genotonorm as an intermittent treatment. Treatment was allowed to be taken until final height of the subject had been reached.

Units	Counts
Participants	OG00030 OG00135 OG00245