Treatment of Children With Insufficient Secretion of Growth Hormone (BPLG-004)

LG Life Sciences

Status and phase

Unknown

Phase 3

Conditions

Growth Hormone Deficiency (GHD)

Treatments

Drug: growth hormone (somatropin)

Study type

Interventional

Funder types

Industry

Identifiers

NCT00271518

BPLG-004

Details and patient eligibility

About

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

Full description

Treatment with recombinant human growth hormone (somatropin) has been proven to be effective in stimulating height velocity and improving height in children with short stature due to insufficient endogenous Growth Hormone secretion. Currently somatropin is available in daily injection formulations. The requirement of daily administration causes significant burden and interruption of normal daily life. A product with less frequent dosing regimen will provide considerable improvement over currently available conventional replacement therapy regimens.

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.

Enrollment

144 estimated patients

Sex

All

Ages

3 to 11 years old

Volunteers

No Healthy Volunteers

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.