Clinical trial NCT00174187: Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy
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.
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Endocrine System Diseases
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2005-09-15
Enrollment: 30
Sex: ALL
Minimum age (years): 11
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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

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 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

Primary outcomes:
measure: Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3
timeFrame: Baseline, Year 3
measure: Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height
timeFrame: Baseline, when final height was reached (assessed up to Year 11)
measure: Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height
timeFrame: Baseline, when final height was reached (assessed up to Year 11)
measure: Puberty Stage at Final Height
timeFrame: When final height was reached (assessed up to Year 11)
Secondary outcomes:
measure: Bone Age
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Lean Body Mass
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Annual Percent Change in Lean Body Mass at Year 1, 2 and 3
timeFrame: Baseline, Year 1, 2, 3
measure: Percent Change From Baseline in Lean Body Mass at Year 3
timeFrame: Baseline, Year 3
measure: Lean Body Mass as Percentage of Total Weight
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Fat Mass
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Annual Percent Change in Fat Mass at Year 1, 2 and 3
timeFrame: Baseline, Year 1, 2, 3
measure: Percent Change From Baseline in Fat Mass at Year 3
timeFrame: Baseline, Year 3
measure: Fat Mass as Percentage of Total Weight
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Bone Mineral Density of Total Body (BMD [TB])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Bone Mineral Density of Lumbar Spine (BMD [LS])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Bone Mineral Content of Total Body (BMC [TB])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3
timeFrame: Baseline, Year 1, 2, 3
measure: Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3
timeFrame: Baseline, Year 3
measure: Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Other outcomes:
measure: Growth Velocity (GV)
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA])
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
measure: Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3
timeFrame: Baseline, Year 1, 2, 3
measure: Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3
timeFrame: 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)
Locations:
city: Paris
country: France
facility: Pfizer Investigational Site
city: Paris
country: France
facility: Pfizer Investigational Site
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: David H, Aupiais C, Louveau B, Quartier P, Jacqz-Aigrain E, Carel JC, Simon D. Growth Outcomes After GH Therapy of Patients Given Long-Term Corticosteroids for Juvenile Idiopathic Arthritis. J Clin Endocrinol Metab. 2017 Dec 1;102(12):4578-4587. doi: 10.1210/jc.2017-01455.