Growth Hormone Deficiency (GHD)Phase 3BPLG-004NCT00271518
Treatment of Children With Insufficient Secretion of Growth Hormone
A Phase 3 study testing growth hormone (somatropin) in people with Growth Hormone Deficiency (GHD), run by LG Life Sciences at 1 site in United States.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Growth Hormone Deficiency (GHD)
- What is being tested?
- growth hormone (somatropin)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 144 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2005to Dec 2013
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Show all 16Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Division of Endocrinology, Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
About this study
Official title: A Phase III, Multi-centre, Randomised, Parallel Group Study of Safety and Efficacy of the LB03002 a New Sustained Release Formulation of Human Recombinant Growth Hormone as Compared to Standard Daily Therapy in Treatment Naive Children With Growth Failure Due to Insufficient Secretion of Endogenous Growth Hormone
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
Read the detailed 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.
- Conditions
- Growth Hormone Deficiency (GHD)
- Being tested
- growth hormone (somatropin)
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
growth hormone (somatropin)Drug
dosing regimen is weight based.
| Study group | Role | What they receive |
|---|---|---|
| LB03002, sustained release human hGH | Experimental | LB03002 Receives: Drug: growth hormone (somatropin) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Height velocity at the end of 12 months treatment
Measured: 12 months
Secondary outcomes (1)
1.Height velocity SD score (HV SDS) after 12 months treatment, 2.Serum IGF-I levels3. Serum IGFBP-3 levels
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jan 2006 (estimated)
- Study startSep 2005
- Primary completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Growth Hormone Deficiency (GHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 144 participants (estimated)
Sponsor and investigators
- Lead sponsor
- LG Life Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- BioPartners GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00271518
- Sponsor's protocol ID
- BPLG-004
- Acronym
- BPLG-004
- Status last verified
- Mar 2010
- First submitted
- 29 Dec 2005
- Last updated
- 17 Mar 2010
- Results posted
- No
- Data monitoring committee
- Yes
- Expanded access
- Available
Publications
Khadilkar V, Radjuk KA, Bolshova E, Khadgawat R, El Kholy M, Desai M, Peterkova V, Mericq V, Kratzsch J, Siepl EC, Martin D, Lopez P, Ji HJ, Bae YJ, Lee JH, Saenger PH. 24-month use of once-weekly GH, LB03002, in prepubertal children with GH deficiency. J Clin Endocrinol Metab. 2014 Jan;99(1):126-32. doi: 10.1210/jc.2013-2502. Epub 2013 Dec 20.
PubMed 24170106
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Multi-centre, Randomised, Parallel Group Study of Safety and Efficacy of the LB03002 a New Sustained Release Formulation of Human Recombinant Growth Hormone as Compared to Standard Daily Therapy in Treatment Naive Children With Growth Failure Due to Insufficient Secretion of Endogenous Growth Hormone
- ClinicalTrials.gov ID
- NCT00271518
- Acronym / sponsor ID
- BPLG-004 / BPLG-004
- Conditions
- Growth Hormone Deficiency (GHD)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 144 participants (estimated)
- Lead sponsor
- LG Life SciencesIndustry (pharma, biotech, devices)
- Collaborators
- BioPartners GmbH
- Study start
- Sep 2005
- Primary completion
- Dec 2013 (estimated)
- First posted
- 2 Jan 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LB03002, sustained release human hGH | Experimental | Drug: growth hormone (somatropin)LB03002 |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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).
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| growth hormone (somatropin)dosing regimen is weight based. | Drug | LB03002, sustained release human hGH |
Primary outcome
Height velocity at the end of 12 months treatment
Time frame: 12 months
1 secondary outcome
1.Height velocity SD score (HV SDS) after 12 months treatment, 2.Serum IGF-I levels3. Serum IGFBP-3 levels
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2010.
- Derived from this study: PubMed 24170106Khadilkar V, Radjuk KA, Bolshova E, Khadgawat R, El Kholy M, Desai M, Peterkova V, Mericq V, Kratzsch J, Siepl EC, Martin D, Lopez P, Ji HJ, Bae YJ, Lee JH, Saenger PH. 24-month use of once-weekly GH, LB03002, in prepubertal children with GH deficiency. J Clin Endocrinol Metab. 2014 Jan;99(1):126-32. doi: 10.1210/jc.2013-2502. Epub 2013 Dec 20.
Related trials for Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Active, not recruitingObservational study
Ngenla Subcutaneous Injection Special Investigation
Growth Hormone Deficiency Without Epiphyseal Closure
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing growth hormone (somatropin) in people with Growth Hormone Deficiency (GHD), run by LG Life Sciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Mar 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Height velocity at the end of 12 months treatment (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00271518, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2010.