Evolution Of Growth Rate In Children With Growth Retardation Due to Glucocorticosteroid Therapy And Treated By Genotonorm

Evolution Of Growth Rate In Children With Growth Retardation Related To Long-term Corticotherapy And Treated By Genotonorm

To estimate the evolution of height and growth rate over 5 years of growth hormone (GH) treatment To estimate the prognostic factors of growth rate with GH treatment To confirm the good clinical and biological safety of GH treatment in such children

Study Overview

• Status: Completed
• Conditions: Growth Hormone Deficiency
• Intervention / Treatment: Drug: Somatropin

• Study Type: Interventional
• Enrollment (Actual): 98
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Amiens, France, 80030
    • Hopital Nord
  • Annemasse Cedex, France, 74107
    • Centre Hospitalier Intercommunal d'Annemasse-Bonneville, Service de Pédiatrie et Néonatologie
  • Besancon Cedex, France, 25030
    • Hôpital Saint Jacques
  • Bron, France, 69677
    • Groupe hospitalier Est- Hôpital Femme, Mère, Enfant
  • Bron, France, 69677
    • Groupe hospitalier Est-Hôpital Femme, Mère, Enfant
  • Clermont-Ferrand Cedex 1, France, 63003
    • Chu D'Estaing
  • Grenoble Cedex 9, France, 38043
    • CHU de Grenoble, Hôpital Couple enfant.
  • Marseille Cedex 5, France, 13385
    • CHU Timone Enfants
  • Montpellier, France, 34059
    • Hôpital Arnaud de Villeneuve
  • Nantes cedex 1, France, 44093
    • CHU de Nantes, Hôpital Mère Enfant
  • Nice, France, 06200
    • Hôpital Lenval
  • Paris, France, 75019
    • Hôpital Robert Debré
  • Paris, France, 75571
    • Hôpital Armand Trousseau
  • Paris cedex 15, France, 75743
    • Groupe Hospitalier Necker - Enfants Malades
  • Roscoff Cedex, France, 29684
    • Centre de Perharidy
  • Rouen Cedex, France, 76031
    • CHU Charles Nicolle
  • Saint Germain-en-Laye Cedex, France, 78105
    • Service de Pédiatrie- Centre Hospitalier Intercommunal
  • Toulouse Cedex 9, France, 31059
    • Hôpital des Enfants
  • Tours Cedex 1, France, 37044
    • CHU Tours - Centre Pediatrique Gatien de Cocheville

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Glucocorticosteroid treatment for 12 months at least
• Bone age < 15 years for a boy and < 13 years for a girl
• Child measured height < - 2 SD, Child currently treated by GH

Exclusion Criteria:

• Known diabetes (type 1 or type 2)
• A previous history of intolerance or hypersensitivity to the study drug or to drugs with similar chemical structures

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Somatropin	Drug: Somatropin; Liquid, daily, during 3 years and extended to 5 years Dosage: 0,46 mg/kg/week - the maximum dose should not exceed 50 µg/kg/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population	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; Yx refers to the value at particular timepoint x. 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.	Baseline, Month 36
Change From Baseline in Height Standard Deviation Score (SD) for Chronological Age (CA) at Month 36: Per Protocol (PP) Population	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; Yx refers to the value at particular timepoint x. 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.	Baseline, Month 36

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Height	The standing height measurements were performed 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.	Baseline, Month 12, 24, 36, 48, 60
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)	The standing height measurements were performed 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 BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. 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.	Baseline, Month 12, 24, 36, 48, 60
Annual Growth Rate (AGR)	AGR at Yx was derived by subtracting AGR at baseline from Yx value. AGR was calculated each year and re scaled 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). Yx refers to the value at particular timepoint x.	Baseline, Month 12, 24, 36, 48, 60
Growth Rate (GR) Standard Deviation Score (SDS) for Bone Age (BA)	GR SDS BA Yx = (GR Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. GR in SDS was calculated using Sempe reference means and SD for GR. BA was estimated locally using an X-ray from the left wrist and hand.	Month 12, 24, 36, 48, 60
Growth Rate (GR) Standard Deviation Score (SDS) for Chronological Age (CA)	GR SDS CA Yx = (GR Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. GR in SDS was calculated using Sempe reference means and SD for GR. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.	Baseline, Month 12, 24, 36, 48, 60
Body Mass Index (BMI)	BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m^2).	Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Height at Month 12, 24, 36, 48 and 60	The standing height measurements were performed 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.	Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, 24, 48 and 60	The standing height measurements were performed 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; Yx refers to the value at particular timepoint x. 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.	Baseline, Month 12, 24, 48, 60
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, 24, 36, 48 and 60	The standing height measurements were performed 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 BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. 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.	Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Bone Age (BA) at Month 12, 24, 36, 48 and 60	BA was estimated locally using an X-ray from the left wrist and hand.	Baseline, Month 12, 24, 36, 48, 60
Ratio of Bone Age (BA) to Chronological Age (CA)	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.	Baseline, Month 12, 24, 36, 48, 60

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)	An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state. AEs include both SAEs and non-SAEs.	Baseline up to 28 days after last study treatment
Number of Participants With Significant Changes in Physical Examinations	Number of participants with clinically significant physical examinations changes since previous visit were reported. Physical examination including estimation of pubertal stage and blood pressure measurement;	Baseline, Month 12, 24, 36, 48, 60, End of Treatment (EOT)
Number of Participants With at Least 1 Medical or Surgical History		Screening
Number of Participants Who Received Concomitant Medications		Baseline up to Month 60
Fasting Serum Insulin Like Growth Factor-1 (IGF-1) Levels		Screening, Month 6, 12, 18, 24, 30, 36, 42, 48, 54, 60
Fasting and Postprandial Plasma Glucose Levels at Month 12, 24, 36, 48 and 60	Fasting and 2 hours plasma glucose levels were assessed using standard oral glucose tolerance test (OGTT).	Screening, Month 12, 24, 36, 48, 60

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

• Basmaison O, Ranchin B, Zouater H, Robertson A, Gomez R, Koppiker N. Efficacy and safety of recombinant growth hormone treatment in children with growth retardation related to long-term glucocorticosteroid therapy. Ann Endocrinol (Paris). 2019 Sep;80(4):202-210. doi: 10.1016/j.ando.2019.02.001. Epub 2019 Mar 4.

Helpful Links

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

Study record dates

Study Major Dates

• Study Start: January 1, 2005
• Primary Completion (Actual): December 1, 2014
• Study Completion (Actual): December 1, 2014

Study Registration Dates

• First Submitted: September 9, 2005
• First Submitted That Met QC Criteria: September 9, 2005
• First Posted (Estimated): September 13, 2005

Study Record Updates

• Last Update Posted (Estimated): February 21, 2024
• Last Update Submitted That Met QC Criteria: February 19, 2024
• Last Verified: June 1, 2015

More Information

Terms related to this study

• Other Study ID Numbers: A6281271; 2004-002992-17 (EudraCT Number)