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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00163189
First received: September 9, 2005
Last updated: June 22, 2015
Last verified: June 2015
  Purpose

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


Condition Intervention Phase
Growth Hormone Deficiency
Drug: Somatropin
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evolution Of Growth Rate In Children With Growth Retardation Related To Long-term Corticotherapy And Treated By Genotonorm

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population [ Time Frame: Baseline, Month 36 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Height Standard Deviation Score (SD) for Chronological Age (CA) at Month 36: Per Protocol (PP) Population [ Time Frame: Baseline, Month 36 ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Mean Height [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Mean Height Standard Deviation Score (SDS) for Bone Age (BA) [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Annual Growth Rate (AGR) [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Growth Rate (GR) Standard Deviation Score (SDS) for Bone Age (BA) [ Time Frame: Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Growth Rate (GR) Standard Deviation Score (SDS) for Chronological Age (CA) [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Body Mass Index (BMI) [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m^2).

  • Change From Baseline in Height at Month 12, 24, 36, 48 and 60 [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, 24, 48 and 60 [ Time Frame: Baseline, Month 12, 24, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, 24, 36, 48 and 60 [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Bone Age (BA) at Month 12, 24, 36, 48 and 60 [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    BA was estimated locally using an X-ray from the left wrist and hand.

  • Ratio of Bone Age (BA) to Chronological Age (CA) [ Time Frame: Baseline, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: No ]
    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.


Other Outcome Measures:
  • Number of Participants With Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 28 days after last study treatment ] [ Designated as safety issue: Yes ]
    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.

  • Number of Participants With Significant Changes in Physical Examinations [ Time Frame: Baseline, Month 12, 24, 36, 48, 60, End of Treatment (EOT) ] [ Designated as safety issue: Yes ]
    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;

  • Number of Participants With at Least 1 Medical or Surgical History [ Time Frame: Screening ] [ Designated as safety issue: Yes ]
  • Number of Participants Who Received Concomitant Medications [ Time Frame: Baseline up to Month 60 ] [ Designated as safety issue: Yes ]
  • Fasting Serum Insulin Like Growth Factor-1 (IGF-1) Levels [ Time Frame: Screening, Month 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 ] [ Designated as safety issue: Yes ]
  • Fasting and Postprandial Plasma Glucose Levels at Month 12, 24, 36, 48 and 60 [ Time Frame: Screening, Month 12, 24, 36, 48, 60 ] [ Designated as safety issue: Yes ]
    Fasting and 2 hours plasma glucose levels were assessed using standard oral glucose tolerance test (OGTT).


Enrollment: 98
Study Start Date: January 2005
Study Completion Date: December 2014
Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00163189

Locations
France
Hôpital Nord
Amiens, France, 80030
Centre Hospitalier Intercommunal d'Annemasse-Bonneville, Service de Pédiatrie et Néonatologie
Annemasse Cedex, France, 74107
Hôpital Saint Jacques
Besancon Cedex, France, 25030
Groupe hospitalier Est- Hôpital Femme, Mère, Enfant
Bron, France, 69677
Groupe hospitalier Est-Hôpital Femme, Mère, Enfant
Bron, France, 69677
CHU d'Estaing
Clermont-Ferrand Cedex 1, France, 63003
CHU de Grenoble, Hôpital Couple enfant.
Grenoble Cedex 9, France, 38043
CHU Timone Enfants
Marseille Cedex 5, France, 13385
Hôpital Arnaud De Villeneuve
Montpellier, France, 34059
CHU de Nantes, Hôpital Mère Enfant
Nantes cedex 1, France, 44093
Hôpital Lenval
Nice, France, 06200
Hôpital Armand Trousseau
Paris, France, 75571
Hôpital Robert Debré
Paris, France, 75019
Groupe Hospitalier Necker - Enfants Malades
Paris cedex 15, France, 75743
Centre de Perharidy
Roscoff Cedex, France, 29684
CHU Charles Nicolle
Rouen Cedex, France, 76031
Service de Pédiatrie- Centre Hospitalier Intercommunal
Saint Germain-en-Laye Cedex, France, 78105
Hôpital des Enfants
Toulouse Cedex 9, France, 31059
CHU Tours - Centre Pediatrique Gatien de Cocheville
Tours Cedex 1, France, 37044
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: NCT00163189     History of Changes
Other Study ID Numbers: A6281271, 2004-002992-17
Study First Received: September 9, 2005
Results First Received: June 1, 2015
Last Updated: June 22, 2015
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Additional relevant MeSH terms:
Dwarfism, Pituitary
Bone Diseases
Bone Diseases, Developmental
Bone Diseases, Endocrine
Brain Diseases
Central Nervous System Diseases
Dwarfism
Endocrine System Diseases
Hypopituitarism
Hypothalamic Diseases
Musculoskeletal Diseases
Nervous System Diseases
Pituitary Diseases

ClinicalTrials.gov processed this record on July 28, 2015