Growth Retardation In Children With Special Pathological Conditions Or Disease

Endocrine System Diseases Clinical Trial

Official title:

Evolution Of Growth Rate In Children Suffering From A Disease Associated With Growth Retardation and Treated By Genotonorm. A Pilot Study.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00163215
• Other study ID #: A6281269
• Status: Completed
• Phase: Phase 3
• First received: September 9, 2005
• Last updated: November 19, 2012
• Start date: January 2005
• Est. completion date: October 2011

Study information

• Verified date: November 2012
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 46
• Est. completion date: October 2011
• Est. primary completion date: October 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 11 Years to 13 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Endocrine System Diseases

Intervention

Drug:
• Somatropin
Adapted dosage based on IGF 1 level and weight Form: liquid; Dosage and Frequency: from 0.0033mg/kg/day to 0.0067 mg/kg/day; Duration: 3 years

Locations

Country	Name	City	State
France	Pfizer Investigational Site	Annemasse Cedex
France	Pfizer Investigational Site	Besancon
France	Pfizer Investigational Site	Bordeaux
France	Pfizer Investigational Site	Bordeaux
France	Pfizer Investigational Site	Bron Cedex
France	Pfizer Investigational Site	Dijon
France	Pfizer Investigational Site	Grenoble Cedex
France	Pfizer Investigational Site	Lorient
France	Pfizer Investigational Site	Lyon Cedex 05
France	Pfizer Investigational Site	Marseille
France	Pfizer Investigational Site	Montpellier
France	Pfizer Investigational Site	Paris
France	Pfizer Investigational Site	Paris
France	Pfizer Investigational Site	Paris
France	Pfizer Investigational Site	Paris
France	Pfizer Investigational Site	PRINGY Cedex
France	Pfizer Investigational Site	Toulouse Cedex
France	Pfizer Investigational Site	Vandoeuvre Les Nancy Cedex

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population	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.	Baseline, Month 36	No
Primary	Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) Population	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.	Baseline, Month 36	No
Secondary	Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population	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).	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population	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).	Baseline, Month 12, Month 24, Month 36	No
Secondary	Height	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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Height at Month 12, Month 24 and 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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)	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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and 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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Mean Height Standard Deviation Score (SDS) for Bone Age (BA)	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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and 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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT Population	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.	Baseline, Month 12, Month 24	No
Secondary	Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP Population	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.	Baseline, Month 12, Month 24	No
Secondary	Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)	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.	Month 12, Month 24, Month 36	No
Secondary	Body Mass Index (BMI)	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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and 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.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36	BA was estimated locally using an X-ray from the left wrist and hand.	Baseline, Month 12, Month 24, Month 36	No
Secondary	Ratio of Bone Age (BA) to Chronological Age (CA)	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.	Baseline, Month 12, Month 24, Month 36	No

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