DwarfismPhase 4NCT01157793
A Multicentre, Randomised, Open-label, Controlled Study to Evaluate the Effects of Saizen® on Cardiac Function in Growth Hormone Deficient(GHD) Subjects During the Transition Phase From Childhood to Adulthood
A Phase 4 study testing r-hGH in people with Dwarfism and Growth Hormone Deficiency, run by Merck KGaA, Darmstadt, Germany.
Trial summary
This was a 48-week, open-label, prospective, multicentric, randomised, comparative with parallel control, Phase 4 study to evaluate the effects of Saizen on cardiac function in GHD subjects during the transition phase from childhood to adulthood. Read more about this study
- Who is it for?
- Ages 14 to 25 years
- Conditions the study is for
- Dwarfism, Growth Hormone Deficiency
- What is being tested?
- r-hGH
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- No sites listed yet
- Study dates
- Sep 2003to Feb 2005
- Phase
- Phase 4
- 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 14 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects with diagnosis of childhood onset GH deficiency and previously treated with GH
- Subjects who had attained final height
- Male or female subjects, aged between 14 and 25 years of age inclusively at baseline
- Subjects with GH deficiency of <5μg/L (acquired or idiopathic), established by any 1 type of GH secretion test within 3 years prior to Study Day 1
- If hypopituitary, subject must have been on adequate replacement therapy (if required) of glucocorticosteroids, thyroid \& sex hormones (hormones levels on replacement being in normal/mildly elevated range) for at least 6 months prior to study entry
- Subjects who were willing and able to comply with the protocol for the duration of the study.
- Subjects who had given written informed consent before any study-related procedure not part of the subject's normal medical care, with the understanding that the subject might withdraw consent at any time without prejudice to future medical care
- Female subjects must be neither pregnant nor breast-feeding, and use a hormonal contraceptive, intra-uterine device, diaphragm with spermicide or condom with spermicide for the duration of the study. Confirmation that a female subject was not pregnant was established by a negative urinary human chorionic gonadotropin (hCG) pregnancy test at baseline.
Who cannot join
- Subject who had a known allergy or hypersensitivity to growth hormone or diluent
- Subject who had been treated with r-hGH in previous six months
- Subject with chronic severe kidney disease
- Subject with chronic severe liver disease
- Subject with acute or severe illness during the previous 6 months
- Subject with significant concomitant illness which would interfere with his/her participation or assessment in this study
Show all 14Show fewer
- Subject with active malignancy (except non-melanomatous skin malignancies)
- Subjects with unstable hypertension (supine systolic blood pressure persistently above 160 mmHg or diastolic blood pressure persistently above 100 mmHg)
- Subjects with benign cranial hypertension
- Subjects with a history of carpal tunnel syndrome, unless surgically released
- Subjects with known positive human immunodeficiency virus (HIV), Hepatitis B surface antigen (HBsAg) and/or Hepatitis C virus (HCV) serology based on past medical history
- Subjects with known active drug addiction, including alcoholism, or use of drugs for nontherapeutic purposes
- Subject who had previously participated in this study
- Subject taking an investigational drug or enrolled in another clinical study
About this study
Official title: A Multicentre, Randomised, Open-label, Controlled Study to Evaluate the Effects of Saizen® on Cardiac Function in GHD Subjects During the Transition Phase From Childhood to Adulthood
This was a 48-week, open-label, prospective, multicentric, randomised, comparative with parallel control, Phase 4 study to evaluate the effects of Saizen on cardiac function in GHD subjects during the transition phase from childhood to adulthood.
The study was designed to evaluate whether recombinant-human growth hormone (r-hGH) treatment also benefits young subjects with GHD. Some trials have already been published on this subject, but they were mainly focused on the bone density.
Read the detailed description
Growth hormone is a 191 amino acid polypeptide hormone (MW 22,000) normally synthesised and secreted by the somatotrophic cells of the anterior lobe of the pituitary gland. In normal development, growth hormone and somatomedins are responsible for many of the manifestations of normal growth and GHD is manifested by a marked short stature. Growth hormone deficiency has been treated by human growth hormone for many years. Serono's r-hGH (Saizen) is produced from genetically engineered mammalian cells.
The findings from previous clinical studies on GH treatment in GH-deficient adults, collectively indicate that the majority of adults with long-standing GH deficiency, whether dating from childhood or acquired in adult life, are compromised both physically and psychologically and can derive clinical benefit from GH replacement. Based on observations in the clinical trials to date , a GH dose of 0.01 mg/kg/day (50% of the dose used in children), is likely to be satisfactory for many subjects. Moreover, it should be possible to minimise early side effects, particularly fluid retention, by initiating treatment with half of this dose and increasing to the final dose after 4 weeks if well tolerated.
In this study, it was proposed to enroll a group of childhood onset GHD subjects who were not treated with r-hGH. Half of the study population started treatment for six months whilst the other half remained on no r-hGH treatment. After six months the group already on r-hGH therapy continued treatment for a further six months and the second group presently on no r-hGH treatment started r hGH treatment for the remaining six months of the study.
OBJECTIVES
Primary objective:
- To compare the effects of Saizen on cardiac function (as assessed by percentage ejection fraction) in subjects where 50% of the study population started r-hGH treatment for 24 weeks and then remained on r-hGH treatment for a further 24 weeks and subjects who continued on no r-hGH for 24 weeks before starting r-hGH for 24 weeks during the transition phase from childhood to adulthood.
Secondary objectives:
- To assess the safety and tolerability of r-hGH in subjects who were transitioning from childhood to adulthood, and to assess the change in body composition and lean body mass. Subsidiary analyses of the other echocardiography parameters was also performed.
After entry into the trial, the subjects were randomised to one of two groups for a 48-week period:
- Group 1: Saizen (r-hGH), 0.15-1.00 mg/day for 48 weeks, subcutaneous (s.c.)
- Group 2: No treatment for the first 24 weeks followed by Saizen (r-hGH)0.15-1.00 mg/day for the next 24 weeks, s.c.
Subjects' visits to the study site was scheduled as follows:
- Group 1 - Baseline (study day 1), weeks 4, 12, 24, 36 \& 48.
- Group 2 - Baseline (study day 1), weeks 12, 24, 28, 36 \& 48. The study drug was administered subcutaneously once daily in the evenings during the active treatment period. The dose was to be adjusted stepwise, controlled by Insulin-Growth Factor-I (IGF-I) values. The recommended final r-hGH dose was not to exceed 1.00mg/day
- Conditions
- Dwarfism, Growth Hormone Deficiency
- Being tested
- r-hGH
- Keywords
- Dwarfism, Growth hormone deficiency, Growth hormone
- Medical subject headings
- Dwarfism, Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
r-hGHDrug
Also called Saizen®
r-hGH at a dose of 0.15-1.00 mg/day administered for 48 weeks by s.c. route
r-hGHDrug
Also called Saizen®
Initially no treatment for the first 24 weeks followed by administration of r-hGH at a dose of 0.15 1.00 mg/day for the next 24 weeks, by s.c. route
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Not described Receives: Drug: r-hGH |
| Group 2 | Experimental | Not described Receives: Drug: r-hGH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in percentage ejection fraction in subjects during the transition phase from childhood to adulthood
Measured: Baseline to study week 48
Secondary outcomes (2)
Subsidiary analysis of the other echocardiography parameters and lean body mass
Measured: Baseline to study week 48
Evaluation of laboratory parameters and monitoring of adverse events
Measured: Baseline to study week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2010 (estimated)
- Study startSep 2003
- Primary completionFeb 2005
- Study completionFeb 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DwarfismStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Theodor Wee, MD, Study director, Merck Serono International SA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01157793
- Sponsor's protocol ID
- IMP24632
- Status last verified
- Jul 2010
- First submitted
- 6 Jul 2010
- Last updated
- 11 Jul 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicentre, Randomised, Open-label, Controlled Study to Evaluate the Effects of Saizen® on Cardiac Function in GHD Subjects During the Transition Phase From Childhood to Adulthood
- ClinicalTrials.gov ID
- NCT01157793
- Acronym / sponsor ID
- IMP24632
- Conditions
- Dwarfism, Growth Hormone Deficiency
- Keywords
- Dwarfism, Growth hormone deficiency, Growth hormone
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Study start
- Sep 2003
- Primary completion
- Feb 2005 (actual)
- Study completion
- Feb 2005 (actual)
- First posted
- 7 Jul 2010 (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.
This was a 48-week, open-label, prospective, multicentric, randomised, comparative with parallel control, Phase 4 study to evaluate the effects of Saizen on cardiac function in GHD subjects during the transition phase from childhood to adulthood.
The study was designed to evaluate whether recombinant-human growth hormone (r-hGH) treatment also benefits young subjects with GHD. Some trials have already been published on this subject, but they were mainly focused on the bone density.
Detailed description
Growth hormone is a 191 amino acid polypeptide hormone (MW 22,000) normally synthesised and secreted by the somatotrophic cells of the anterior lobe of the pituitary gland. In normal development, growth hormone and somatomedins are responsible for many of the manifestations of normal growth and GHD is manifested by a marked short stature. Growth hormone deficiency has been treated by human growth hormone for many years. Serono's r-hGH (Saizen) is produced from genetically engineered mammalian cells.
The findings from previous clinical studies on GH treatment in GH-deficient adults, collectively indicate that the majority of adults with long-standing GH deficiency, whether dating from childhood or acquired in adult life, are compromised both physically and psychologically and can derive clinical benefit from GH replacement. Based on observations in the clinical trials to date , a GH dose of 0.01 mg/kg/day (50% of the dose used in children), is likely to be satisfactory for many subjects. Moreover, it should be possible to minimise early side effects, particularly fluid retention, by initiating treatment with half of this dose and increasing to the final dose after 4 weeks if well tolerated.
In this study, it was proposed to enroll a group of childhood onset GHD subjects who were not treated with r-hGH. Half of the study population started treatment for six months whilst the other half remained on no r-hGH treatment. After six months the group already on r-hGH therapy continued treatment for a further six months and the second group presently on no r-hGH treatment started r hGH treatment for the remaining six months of the study.
OBJECTIVES
Primary objective:
- To compare the effects of Saizen on cardiac function (as assessed by percentage ejection fraction) in subjects where 50% of the study population started r-hGH treatment for 24 weeks and then remained on r-hGH treatment for a further 24 weeks and subjects who continued on no r-hGH for 24 weeks before starting r-hGH for 24 weeks during the transition phase from childhood to adulthood.
Secondary objectives:
- To assess the safety and tolerability of r-hGH in subjects who were transitioning from childhood to adulthood, and to assess the change in body composition and lean body mass. Subsidiary analyses of the other echocardiography parameters was also performed.
After entry into the trial, the subjects were randomised to one of two groups for a 48-week period:
- Group 1: Saizen (r-hGH), 0.15-1.00 mg/day for 48 weeks, subcutaneous (s.c.)
- Group 2: No treatment for the first 24 weeks followed by Saizen (r-hGH)0.15-1.00 mg/day for the next 24 weeks, s.c.
Subjects' visits to the study site was scheduled as follows:
- Group 1 - Baseline (study day 1), weeks 4, 12, 24, 36 \& 48.
- Group 2 - Baseline (study day 1), weeks 12, 24, 28, 36 \& 48. The study drug was administered subcutaneously once daily in the evenings during the active treatment period. The dose was to be adjusted stepwise, controlled by Insulin-Growth Factor-I (IGF-I) values. The recommended final r-hGH dose was not to exceed 1.00mg/day
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Drug: r-hGH |
| Group 2 | Experimental | Drug: r-hGH |
Who may qualify
- Ages
- 14 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Subjects with diagnosis of childhood onset GH deficiency and previously treated with GH
- Subjects who had attained final height
- Male or female subjects, aged between 14 and 25 years of age inclusively at baseline
- Subjects with GH deficiency of <5μg/L (acquired or idiopathic), established by any 1 type of GH secretion test within 3 years prior to Study Day 1
- If hypopituitary, subject must have been on adequate replacement therapy (if required) of glucocorticosteroids, thyroid \& sex hormones (hormones levels on replacement being in normal/mildly elevated range) for at least 6 months prior to study entry
Key exclusion criteria (14 listed)
- Subject who had a known allergy or hypersensitivity to growth hormone or diluent
- Subject who had been treated with r-hGH in previous six months
- Subject with chronic severe kidney disease
- Subject with chronic severe liver disease
- Subject with acute or severe illness during the previous 6 months
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 |
|---|---|---|
| r-hGHAlso: Saizen®r-hGH at a dose of 0.15-1.00 mg/day administered for 48 weeks by s.c. route | Drug | Group 1 |
| r-hGHAlso: Saizen®Initially no treatment for the first 24 weeks followed by administration of r-hGH at a dose of 0.15 1.00 mg/day for the next 24 weeks, by s.c. route | Drug | Group 2 |
Primary outcome
Change in percentage ejection fraction in subjects during the transition phase from childhood to adulthood
Time frame: Baseline to study week 48
2 secondary outcomes
Subsidiary analysis of the other echocardiography parameters and lean body mass
Time frame: Baseline to study week 48
Evaluation of laboratory parameters and monitoring of adverse events
Time frame: Baseline to study week 48
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2014.
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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.
Theodor Wee, MD study director
Merck Serono International SA
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing r-hGH in people with Dwarfism and Growth Hormone Deficiency, run by Merck KGaA, Darmstadt, Germany.
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 14 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: finished, as of the sponsor's last update on 11 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change in percentage ejection fraction in subjects during the transition phase from childhood to adulthood (time frame: Baseline to study week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Jul 2014.