MedPath

Growth Retardation In Children With Special Pathological Conditions Or Disease

Phase 3
Completed
Conditions
Endocrine System Diseases
Interventions
Registration Number
NCT00163215
Lead Sponsor
Pfizer
Brief Summary

To show an increase in annual growth rate 3 years after Visit 2. Annual growth rate in standard deviation (SD) after 3 years will be compared to growth rate before the start of GH treatment.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
46
Inclusion Criteria
  • Bone age < 13 years for a boy and < 11 years for a girl
  • Naive child: Measured Height < -2.5 SD for CA
  • Child currently treated by GH
Exclusion Criteria
  • Idiopathic short stature
  • Syndrome known to be associated with an increased risk of cancer e.g. family history of adenomatous polyposis

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
SomatropinSomatropin-
Primary Outcome Measures
NameTimeMethod
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) PopulationBaseline, Month 36

Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.

Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) PopulationBaseline, Month 36

Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT PopulationBaseline, Month 12, Month 24, Month 36

Change in AGR at Yx was derived by subtracting AGR at baseline from Yx value. Annual growth rate was calculated each year and rescaled to 1 year if the interval between Yx and Y\[x-1\] was not 365 days, as long as a participant remained in the study. AGR at Yx was calculated using the previous height measurements (Y\[x-1\]) and height recorded at Yx (AGR Yx = \[height Yx-height Y{x-1}\] / (\[date of Yx - date of Y{x-1}\] /365.25).

Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP PopulationBaseline, Month 12, Month 24, Month 36

Change in AGR at Yx was derived by subtracting AGR at baseline from Yx value. Annual growth rate was calculated each year and rescaled to 1 year if the interval between Yx and Y\[x-1\] was not 365 days, as long as a participant remained in the study. AGR at Yx was calculated using the previous height measurements (Y\[x-1\]) and height recorded at Yx (AGR Yx = \[height Yx-height Y{x-1}\] / (\[date of Yx - date of Y{x-1}\] /365.25).

HeightBaseline, Month 12, Month 24, Month 36

Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded.

Change From Baseline in Height at Month 12, Month 24 and Month 36Baseline, Month 12, Month 24, Month 36

Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded.

Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)Baseline, Month 12, Month 24, Month 36

Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar the participant was to the reference population.

Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36Baseline, Month 12, Month 24, Month 36

Change in height SDS CA was derived by subtracting height SDS CA at baseline from Yx value. Height SDS CA (for both baseline and Yx) = (height - reference mean for CA)/reference SD for CA. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement - Date of birth)/365.25\*12. SDS indicates how similar the participant was to the reference population.

Mean Height Standard Deviation Score (SDS) for Bone Age (BA)Baseline, Month 12, Month 24, Month 36

Height SDS BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx. Height in SDS was calculated using Sempe reference means and SD for height. BA was estimated locally using an X-ray from the left wrist and hand. SDS indicates how similar the participant was to the reference population.

Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36Baseline, Month 12, Month 24, Month 36

Change in height SDS BA was derived by subtracting height SDS BA at baseline from Yx value. Height SDS BA (for both baseline and Yx) = (height-reference mean for BA)/reference SD for BA. Height in SDS was calculated using Sempe reference means and SD for height. BA was estimated locally using an X-ray from the left wrist and hand. SDS indicates how similar the participant was to the reference population.

Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT PopulationBaseline, Month 12, Month 24

Change in AGR SDS for CA derived by subtracting AGR SDS CA at baseline from Yx value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.

Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP PopulationBaseline, Month 12, Month 24

Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.

Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)Month 12, Month 24, Month 36

AGR at Yx was derived by subtracting AGR at baseline from Yx value. AGR was calculated each year and rescaled to 1 year if the interval between Yx and Y\[x-1\] was not 365 days, as long as a participant remained in the study. AGR at Yx = \[height Yx-height Y{x-1}\] / (\[date of Yx - date of Y{x-1}\] /365.25). GR in SDS was calculated using Sempe reference means and SD for growth. BA was estimated locally using an X-ray from the left wrist and hand. SDS indicates how similar the participant was to the reference population.

Body Mass Index (BMI)Baseline, Month 12, Month 24, Month 36

BMI was used to measure body fat based on height and weight. It was calculated as body weight (kilogram) divided by the height (meter) squared.

Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36Baseline, Month 12, Month 24, Month 36

BMI was used to measure body fat based on height and weight. It was calculated as body weight (kilogram) divided by the height (meter) squared.

Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36Baseline, Month 12, Month 24, Month 36

BA was estimated locally using an X-ray from the left wrist and hand.

Ratio of Bone Age (BA) to Chronological Age (CA)Baseline, Month 12, Month 24, Month 36

BA was estimated locally using an X-ray from the left wrist and hand. CA at the date of corresponding X-ray (Date of X-ray - Date of birth)/365.25. Ratio of BA/CA at each annual study visit was calculated.

Trial Locations

Locations (1)

Pfizer Investigational Site

🇫🇷

Vandoeuvre Les Nancy Cedex, France

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