Growth Retardation In Children With Special Pathological Conditions Or Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00163215
First received: September 9, 2005
Last updated: November 19, 2012
Last verified: November 2012
  Purpose

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.


Condition Intervention Phase
Endocrine System Diseases
Drug: Somatropin
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evolution Of Growth Rate In Children Suffering From A Disease Associated With Growth Retardation and Treated By Genotonorm. A Pilot Study.

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population [ Time Frame: Baseline, Month 36 ] [ Designated as safety issue: No ]
    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) Population [ Time Frame: Baseline, Month 36 ] [ Designated as safety issue: No ]
    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:
  • Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 Population [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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).

  • Height [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 36 [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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) [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 36 [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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) [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 36 [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 Population [ Time Frame: Baseline, Month 12, Month 24 ] [ Designated as safety issue: No ]
    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 Population [ Time Frame: Baseline, Month 12, Month 24 ] [ Designated as safety issue: No ]
    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) [ Time Frame: Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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) [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 36 [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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 36 [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    BA was estimated locally using an X-ray from the left wrist and hand.

  • Ratio of Bone Age (BA) to Chronological Age (CA) [ Time Frame: Baseline, Month 12, Month 24, Month 36 ] [ Designated as safety issue: No ]
    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.


Enrollment: 46
Study Start Date: January 2005
Study Completion Date: October 2011
Primary Completion Date: October 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Somatropin 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

  Eligibility

Ages Eligible for Study:   11 Years to 13 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00163215

Locations
France
Pfizer Investigational Site
Annemasse Cedex, France, 74107
Pfizer Investigational Site
Besancon, France, 25030
Pfizer Investigational Site
Bordeaux, France, 33000
Pfizer Investigational Site
Bordeaux, France, 33076
Pfizer Investigational Site
Bron Cedex, France, 69677
Pfizer Investigational Site
Dijon, France, 21000
Pfizer Investigational Site
Grenoble Cedex, France, 38043
Pfizer Investigational Site
Lorient, France, 56100
Pfizer Investigational Site
Lyon Cedex 05, France, 69322
Pfizer Investigational Site
Marseille, France, 13385
Pfizer Investigational Site
Montpellier, France, 34059
Pfizer Investigational Site
Paris, France, 75019
Pfizer Investigational Site
Paris, France, 75015
Pfizer Investigational Site
Paris, France, 75571
Pfizer Investigational Site
Paris, France, 75674 Cedex
Pfizer Investigational Site
PRINGY Cedex, France, 74374
Pfizer Investigational Site
Toulouse Cedex, France, 31026
Pfizer Investigational Site
Vandoeuvre Les Nancy Cedex, France, 54511
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00163215     History of Changes
Other Study ID Numbers: A6281269
Study First Received: September 9, 2005
Results First Received: October 2, 2012
Last Updated: November 19, 2012
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Additional relevant MeSH terms:
Endocrine System Diseases

ClinicalTrials.gov processed this record on April 17, 2014