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

This study has been terminated.
(See termination reason in detailed description.)
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00174187
First received: September 9, 2005
Last updated: November 5, 2012
Last verified: November 2012
  Purpose
  • 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

Condition Intervention Phase
Endocrine System Diseases
Drug: Somatropin
Phase 3

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

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3 [ Time Frame: Baseline, Year 3 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height [ Time Frame: Baseline, when final height was reached (assessed up to Year 11) ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height [ Time Frame: Baseline, when final height was reached (assessed up to Year 11) ] [ Designated as safety issue: No ]
    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.

  • Puberty Stage at Final Height [ Time Frame: When final height was reached (assessed up to Year 11) ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Bone Age [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray.

  • Lean Body Mass [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan.

  • Annual Percent Change in Lean Body Mass at Year 1, 2 and 3 [ Time Frame: Baseline, Year 1, 2, 3 ] [ Designated as safety issue: No ]
    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.

  • Percent Change From Baseline in Lean Body Mass at Year 3 [ Time Frame: Baseline, Year 3 ] [ Designated as safety issue: No ]
    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.

  • Lean Body Mass as Percentage of Total Weight [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    Lean body mass, a measurement of body composition, was assessed by DEXA scan.

  • Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Fat Mass [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    Fat mass, a measurement of body composition, was assessed by DEXA scan.

  • Annual Percent Change in Fat Mass at Year 1, 2 and 3 [ Time Frame: Baseline, Year 1, 2, 3 ] [ Designated as safety issue: No ]
    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.

  • Percent Change From Baseline in Fat Mass at Year 3 [ Time Frame: Baseline, Year 3 ] [ Designated as safety issue: No ]
    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.

  • Fat Mass as Percentage of Total Weight [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    Fat mass, a measurement of body composition, was assessed by DEXA scan.

  • Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    BMAD (LS) was assessed by DEXA scan.

  • Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Bone Mineral Density of Total Body (BMD [TB]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    BMD (TB) was assessed by DEXA scan.

  • Bone Mineral Density of Lumbar Spine (BMD [LS]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    BMD (LS) was assessed by DEXA scan.

  • Bone Mineral Content of Total Body (BMC [TB]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3 [ Time Frame: Baseline, Year 1, 2, 3 ] [ Designated as safety issue: No ]
    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.

  • Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3 [ Time Frame: Baseline, Year 3 ] [ Designated as safety issue: No ]
    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.

  • Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.


Other Outcome Measures:
  • Growth Velocity (GV) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    Growth velocity measures the annual rate of increase in height.

  • Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA]) [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 ] [ Designated as safety issue: No ]
    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.

  • Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3 [ Time Frame: Baseline, Year 1, 2, 3 ] [ Designated as safety issue: No ]
  • Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3 [ Time Frame: 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) ] [ Designated as safety issue: No ]

Enrollment: 30
Study Start Date: September 2000
Study Completion Date: September 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Somatropin Drug: Somatropin
liquid, daily, until final height Dosage: 0,46 mg/kg/week . The maximum dose should not exceed 50 µg/Kg/day

Detailed 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.

  Eligibility

Ages Eligible for Study:   11 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00174187

Locations
France
Pfizer Investigational Site
Paris, France, 75743
Pfizer Investigational Site
Paris, France, 75019
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: NCT00174187     History of Changes
Other Study ID Numbers: 307-MET-9002-0009, A6281016
Study First Received: September 9, 2005
Results First Received: September 11, 2012
Last Updated: November 5, 2012
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Additional relevant MeSH terms:
Dwarfism
Endocrine System Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on April 17, 2014