The purpose of this study is to compare a new weekly administered growth hormone preparation
with standard daily treatment in children with insufficient secretion of growth hormone
The primary objective of this study is to demonstrate the clinical comparability in terms of
safety and efficacy of a new sustained release recombinant human growth hormone formulation
to that of daily growth hormone.
Inclusion Criteria:
- Pre-pubertal children (boys age: > 3 and <12 years or girls: age >3 and <11 years)
with isolated GH insufficiency, GH insufficiency as part of multiple pituitary
hormone deficiencies, or organic GH insufficiency. If GH insufficiency occurred after
treatment for any brain tumour, the patient has to be at least one year in clinical
remission which has to be confirmed by computer tomography (CT) or magnetic resonance
imaging (MRI) scan (with contrast) within 3 months prior to study entry
- Children with negative signs for intracranial tumour or tumour growth as confirmed
with a CT or MRI scan (with contrast) within 12 months prior to inclusion or at
inclusion visit
- Confirmed diagnosis of GH insufficiency as determined by two different GH provocation
tests, defined as a peak plasma GH level of ≤7 ng/ml
- No prior exposure to rhGH therapy (GH-treatment naive)
- Height (HT), except in children suffering from organic GH insufficiency, of at least
2. 0 standard deviations (SD) (HT SDS £-2. 0) below the mean height for chronological
age (CA) and sex according to the 2000 standards from the Centers for Disease Control
and Prevention (CDC).
- Height velocity (HV) of at least 1 SD (HV SDS £-1) below the mean HV for CA and sex
according to the standards of Prader. The minimum time between two standard height
measurements should be at least 6 month before inclusion.
- Baseline IGF-I level of at least 0. 5 SD (IGF-1 SDS£-0. 5) below the mean IGF-1 level
standardised for age and sex according to the central laboratory reference values.
- Written informed consent of parent or legal guardian of subject.
Exclusion Criteria:
- Any clinically significant abnormality likely to affect growth or the ability to
evaluate growth, such as, but not limited to, chronic diseases like renal
insufficiency, spinal cord irradiation, and malnutrition (BMI must be above - 2SD and
below +2SD of mean BMI for the chronological age and sex according to the CDC
standards, and albumin must be above lower limit of normal (LLN) of the central
laboratory for a patient to be included).
- Patients with overt diabetes mellitus (Fasting blood sugar >126 mg/dl) and impaired
fasting sugar (Fasting blood sugar >100 mg/dl after repeated blood analysis)
- Chromosomal abnormalities and medical “syndromes” (Turner’s syndrome, Laron syndrome,
Noonan syndrome or absence of growth hormone receptors), with the exception of
septo-optic dysplasia
- Congenital abnormalities (causing skeletal abnormalities), Russell-Silver Syndrome,
skeletal dysplasias
- Closed epiphyses
- Other growth promoting medication such as anabolic steroids, with the exception of
pituitary hormone replacement therapy, thyroxine, hydrocortisone and desmopressin
(DDAVP) replacement therapies
- Children requiring glucocorticoid therapy (e. g. asthma) that are on the dose of more
than 400 µg/d of inhaled budesonide or equivalents inhaled for longer than 1 month
during a calendar year
- Bone age (BA) higher than chronological age
- Poorly controlled or uncontrolled pituitary insufficiencies of other axes (e. g.,
thyroid-stimulating hormone, adrenocorticotropic hormone/cortisol, vasopressin
deficiency): Children who are on stable replacement therapy for less than 6 months
for thyroid replacement therapy, and less than 3 months for other hormonal
deficiencies prior to enrolment
- Major medical conditions and/or presence of contraindication to rhGH treatment
- Known or suspected HIV-positive patient or patient with advanced diseases such as
AIDS or tuberculosis
- Drug, substance, or alcohol abuse
- Known hypersensitivity to the components of study medication
- Evidence of tumour growth or malignant disease
- Presence of anti-hGH antibodies at screening
- The patient and/or the parent/legal guardian are likely to be non-compliant in
respect to study conduct.
