Clinical trial NCT00174460: Somatropin Therapy In Children Born Preterm But Appropriate For Gestational Age
Official title: Somatropin Therapy For Short Children Born Of Premature Gestation, A Controlled, Prospective Randomized, Multicenter Study With An Untreated Control Group.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Growth Hormone Therapy
Infant, Very Low Birth Weight
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2005-09-15
Enrollment: 33
Sex: ALL
Minimum age (years): 4
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Safety and efficacy of Somatropin will be evaluated in short children born with a list weight below 1500 g and that did not catch up to normal height at the age of 4.


Eligibility criteria: Inclusion Criteria:

Subjects must meet all of the following inclusion criteria to be eligible for enrollment into the trial:

* Prepubertal caucasian boys between 4 and 10 years of age and girls between 4 and 9 years of age.
* Girls: Tanner stage 1 breast development
* Boys: Testis volume \<= 3ml
* Tanner stage 1 pubic hair development (to exclude confounding effect of adrenarche on growth velocity, insulin sensitivity and body composition).
* (In case of any signs or symptoms of gonadal puberty a GnRH-Test must decide if the subject is still pubertal.)
* Height \<=-2 SD for chronological age (Brandt/Reinken).
* Growth velocity SDS below 0 SD for chronological age (Brandt/Reinken based on 12+/- 3 months observation period before screening).
* Premature born defined as \<=1500 g birth weight.
* GH sufficiency (GH level \> 7 ug/l following any routine GH stimulation test).
* Written informed consent of both parents (legal guardians) and oral/written consent of subject due to age specific information.

Exclusion Criteria:

Subjects presenting with any of the following will not be included in the trial:

* Other endocrine diseases except for well substituted hypothyroidism.
* Severe chronic diseases or medication that might influence linear growth or insulin sensitivity (e.g. Glucocorticoids).
* Positive GAD and IA-2 antibodies (for type 1 diabetes).
* History of malignancy
* Children who meet all of the following 4 criteria:
* actual body height \< -2,5 SDS (Brandt/Reinken) and parent adjusted target height \< -1 SDS (Hermanussen and Cole, 2003)
* length and/or body weight retardations adjusted to gestational age at birth \< -2,0 SDS (Lawrence et al., 1989, Voigt et al., 1996)
* children with chronological age \> = 4 years and
* growth velocity \< 0 SDS during the last year before inclusion.
* Chromosomal aberrations or syndromes.
* Suspected non-compliance or impossibility to follow the two or three year treatment schedule, respectively (e.g. social implications).
* Severe hemiparesis and severe CNS defects
* Retinopathia \> third degree or laser treatment as newborns.
* Participation in any other clinical trial during active treatment phase.
* Other severe acute or chronic medical or psychiatric condition or clinically relevant laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgement of the investigator, would make the subject inappropriate for entry into this trial.

Primary outcomes:
measure: Change in Height Standard Deviation Score (SDS) After 1 Year
timeFrame: Baseline to 1 year (Month 12)
measure: Change in Growth Velocity Standard Deviation Score (SDS) After 1 Year
timeFrame: Baseline to 1 year (Month 12)
Secondary outcomes:
measure: Change From Baseline in Growth Velocity After 1 Year and After 2 Years
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Growth Velocity SDS After 2 Years
timeFrame: Baseline, Month 24
measure: Change From Baseline in Height After 1 Year and After 2 Years
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Height SDS After 2 Years
timeFrame: Baseline, Month 24
measure: Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Triceps
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Subscapular
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Suprailiac
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Volumetric Cortical Bone Mineral Density (BMD) Using Peripheral Quantitative Computed Tomography (pQCT) After 1 Year and After 2 Years
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Cortical Cross-sectional Area (CSA)
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Bone Structure Using Peripheral Quantitative Computed Tomography (pQCT) After 1 Year and 2 Years: Total Cross-sectional Area (CSA)
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Muscle Cross-sectional Area (CSA)
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Cortical Thickness (CT)
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Marrow Area (MA)
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Bone Stability Using pQCT After 1 Year and After 2 Years: Strength-strain Index (SSI)
timeFrame: Baseline, Month 12, Month 24
measure: Change From Baseline in Muscle Strength: Hand Grip SDS After 1 Year and After 2 Years
timeFrame: Baseline, Month 12, Month 24
measure: Number of Participants With Change in Insulin Sensitivity: Somatropin
timeFrame: Baseline, Month 24
measure: Number of Participants With Change in Insulin Sensitivity: Control Arm
timeFrame: Baseline, Month 36
measure: Growth Curve Comparison Based on Height SDS
timeFrame: Month 12, Month 24
measure: Growth Curve Comparison Based on Height SDS: Control Arm
timeFrame: Month 36
measure: Growth Curve Comparison Based on Height
timeFrame: Month 12, Month 24
measure: Growth Curve Comparison Based on Height: Control Arm
timeFrame: Month 36
Other outcomes:
Locations:
city: Chemnitz
country: Germany
facility: Pfizer Investigational Site
city: Erlangen
country: Germany
facility: Pfizer Investigational Site
city: Freiburg
country: Germany
facility: Pfizer Investigational Site
city: Heidelberg
country: Germany
facility: Pfizer Investigational Site
city: Homburg
country: Germany
facility: Pfizer Investigational Site
city: Koeln
country: Germany
facility: Pfizer Investigational Site
city: Leipzig
country: Germany
facility: Pfizer Investigational Site
city: Tuebingen
country: Germany
facility: Pfizer Investigational Site
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: