Evolution Of Growth Rate In Children Suffering From A Disease Associated With Growth Retardation and Treated By Genotonorm. A Pilot Study.
Overview
- Phase
- Phase 3
- Intervention
- Somatropin
- Conditions
- Endocrine System Diseases
- Sponsor
- Pfizer
- Enrollment
- 46
- Locations
- 1
- Primary Endpoint
- Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population
- Status
- Completed
- Last Updated
- 13 years ago
Overview
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.
Investigators
Eligibility 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
Arms & Interventions
Somatropin
Intervention: Somatropin
Outcomes
Primary Outcomes
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
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
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 Outcomes
- Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population(Baseline, Month 12, Month 24, Month 36)
- Height(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Height at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Mean Height Standard Deviation Score (SDS) for Bone Age (BA)(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT Population(Baseline, Month 12, Month 24)
- Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP Population(Baseline, Month 12, Month 24)
- Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)(Month 12, Month 24, Month 36)
- Body Mass Index (BMI)(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Ratio of Bone Age (BA) to Chronological Age (CA)(Baseline, Month 12, Month 24, Month 36)