Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy

Endocrine System Diseases Clinical Trial

Official title:

Treatment With Recombinant Human Growth Hormone Genotonorm (Registered) in Children With Short Stature Secondary to a Long Term Corticoid Therapy. A Study of Efficacy and Safety.

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00174187
• Other study ID #: 307-MET-9002-0009
• Secondary ID: A6281016
• Status: Terminated
• Phase: Phase 3
• First received: September 9, 2005
• Last updated: November 5, 2012
• Start date: September 2000
• Est. completion date: September 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 assess the effect of a long-term treatment by Genotonorm on linear growth in children with short stature receiving steroid therapy

• To assess the effect of a long term treatment with Genotonorm on bone mineralisation

• To assess the effect of a long term treatment with Genotonorm on body composition

Description:

This trial terminated on 10-Jun-2011 due to prolonged issues with drug accountability and data collection discrepancies. The decision to terminate was not based on any safety concerns.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 30
• Est. completion date: September 2011
• Est. primary completion date: September 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 11 Years to 17 Years

Eligibility

Inclusion Criteria:

• Children with juvenile arthritis or nephrotic syndrome

• Before or during puberty

Exclusion Criteria:

• Diabetes Type 1 and 2

• Endocrine disease, except well substituted hypothyroidism

Study Design

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

Related Conditions & MeSH terms

• Dwarfism
• Endocrine System Diseases

Intervention

Drug:
• Somatropin
liquid, daily, until final height Dosage: 0,46 mg/kg/week . The maximum dose should not exceed 50 µg/Kg/day

Locations

Country	Name	City	State
France	Pfizer Investigational Site	Paris
France	Pfizer Investigational Site	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Growth Velocity (GV)	Growth velocity measures the annual rate of increase in height.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Other	Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA])	GV measures the annual rate of increase in height. GV (SDS/CA) was obtained by measuring GV, subtracting the chronological age- and gender-appropriate mean GV and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Other	Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA])	GV measures the annual rate of increase in height. GV (SDS/BA) was obtained by measuring GV, subtracting the bone age- and gender-appropriate mean GV and dividing the result by standard deviation of that mean (as obtained from bone age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Other	Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3		Baseline, Year 1, 2, 3	No
Other	Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3		Year 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9, 9.5, 10; 0.5 and 1 year after somatropin discontinuation, Final Height (assessed up to Year 11)	No
Primary	Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3	Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 3	No
Primary	Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height	Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, when final height was reached (assessed up to Year 11)	No
Primary	Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height	Body weight was measured using a balance scale. Weight SDS was obtained by measuring the weight, subtracting age- and gender-appropriate mean weight and dividing the result by standard deviation of that mean (as obtained from age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, when final height was reached (assessed up to Year 11)	No
Primary	Puberty Stage at Final Height	Pubertal stage (graded from I to V for breast development and pubic hair development) according to the Tanner's method was collected. A low stage (Stage I) corresponds to a pre-pubertal stage and a high stage (Stage V) to an adult stage.	When final height was reached (assessed up to Year 11)	No
Secondary	Bone Age	Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Lean Body Mass	Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Annual Percent Change in Lean Body Mass at Year 1, 2 and 3	Lean body mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Lean body mass at current year minus lean body mass at previous year) divided by lean body mass at previous year, multiplied by 100.	Baseline, Year 1, 2, 3	No
Secondary	Percent Change From Baseline in Lean Body Mass at Year 3	Lean body mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Lean body mass at Year 3 minus lean body mass at baseline) divided by lean body mass at baseline, multiplied by 100.	Baseline, Year 3	No
Secondary	Lean Body Mass as Percentage of Total Weight	Lean body mass, a measurement of body composition, was assessed by DEXA scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)	Lean body mass was assessed by DEXA scan. Lean body mass SDS/CA was obtained by measuring lean body mass, subtracting the chronological age- and gender-appropriate mean lean body mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Fat Mass	Fat mass, a measurement of body composition, was assessed by DEXA scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Annual Percent Change in Fat Mass at Year 1, 2 and 3	Fat mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Fat mass at current year minus fat mass at previous year) divided by fat mass at previous year, multiplied by 100.	Baseline, Year 1, 2, 3	No
Secondary	Percent Change From Baseline in Fat Mass at Year 3	Fat mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Fat mass at Year 3 minus fat mass at baseline) divided by fat mass at baseline, multiplied by 100.	Baseline, Year 3	No
Secondary	Fat Mass as Percentage of Total Weight	Fat mass, a measurement of body composition, was assessed by DEXA scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)	Fat mass was assessed by DEXA scan. Fat mass SDS/CA was obtained by measuring fat mass, subtracting chronological age- and gender-appropriate mean fat mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])	BMAD (LS) was assessed by DEXA scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])	BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/CA) was obtained by measuring the BMAD (LS), subtracting chronological age- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])	BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/Tanner Puberty Stage) was obtained by measuring BMAD (LS), subtracting Tanner puberty stage- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Bone Mineral Density of Total Body (BMD [TB])	BMD (TB) was assessed by DEXA scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Bone Mineral Density of Lumbar Spine (BMD [LS])	BMD (LS) was assessed by DEXA scan.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Bone Mineral Content of Total Body (BMC [TB])	DEXA scan of BMC was used to evaluate potential bone effects of treatment. BMC is an estimate of the amount of mineral (such as calcium) in the bone.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3	BMC is an estimate of the amount of mineral (such as calcium) in the bone. Annual percent change: (BMC [TB] at current year minus BMC [TB] at previous year) divided by BMC [TB] at previous year, multiplied by 100.	Baseline, Year 1, 2, 3	No
Secondary	Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3	BMC is an estimate of the amount of mineral (such as calcium) in the bone. Percent change: (BMC [TB] at Year 3 minus BMC [TB] at baseline) divided by BMC [TB] at baseline, multiplied by 100.	Baseline, Year 3	No
Secondary	Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])	BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/CA) was obtained by measuring BMC (TB), subtracting the chronological age- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No
Secondary	Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])	BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/Tanner Puberty Stage) was obtained by measuring BMC (TB), subtracting the Tanner puberty stage- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11	No

External Links

•   To obtain contact information for a study center near you, click here.