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Genotropin Treatment in Short Prepubertal Children With Intra-Uterine Growth Retardation

Phase 3
Completed
Conditions
Growth Disorders
Intrauterine Growth Retardation
Interventions
Drug: Genotonorm
Registration Number
NCT01073605
Lead Sponsor
Pfizer
Brief Summary

To evaluate the effect of continuous and intermittent administration of Genotonorm on stature in short prepubertal children with intra-uterine growth retardation

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
208
Inclusion Criteria
  • Chronological age (CA) between 3 and 10 for girls
  • Chronological age between 3 and 12 for boys
  • Height for CA below - 2 SD
  • Birth length for CA below -2SD
Exclusion Criteria
  • Endocrine disease except well-substituted hypothyroidism
  • Sever chronic disease
  • Skeletal dysplasia
  • Known chromosomal aberration
  • Ongoing treatment with steroids
  • Known intrauterine infection

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Genotonorm AGenotonormContinuous 0.7 IU/kg/week or 0.03 mg/kg/day
Genotonorm BGenotonormContinuous, 1.4 IU/kg/week or 0.06 mg/kg/day
Genotonorm CGenotonormIntermittent, 1.4 IU/kg/week or 0.06 mg/kg/day
Primary Outcome Measures
NameTimeMethod
Change From Baseline in Annual Growth Rate Measured at 2 Years Following Treatment With GenotonormBaseline, 2 years

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}

Secondary Outcome Measures
NameTimeMethod
Annual Growth Rate Standard Deviation Score (SDS)Baseline, 1 to 6 years

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

Change From Baseline in Annual Growth Rate SDSBaseline, 1 to 3 years

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

Height (cm)Baseline, 1 to 6 years, final height

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.

Change From Baseline in Height (cm)Baseline, 1 to 6 years, final height

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.

Height (SDS)Baseline, 1 to 6 years, final height

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.

Change From Baseline in Height (SDS)Baseline, 1 to 6 years, final height

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.

Body Mass Index (BMI)Baseline, 1 to 6 years

BMI was calculated by weight divided by height squared.

WeightBaseline, 1 to 6 years
Change From Baseline in Bone AgeBaseline, 1 to 3 years

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

Change From Baseline in Bone Age/Change From Baseline in Chronological Age Ratio1 to 3 years

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.

Chronological Age at Onset of PubertyOnset 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.

Number of Subjects Reaching PubertyBaseline, 1 to 6 years

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.

Trial Locations

Locations (1)

Pfizer Investigational Site

🇫🇷

Vandoeuvre Les Nancy Cedex, France

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