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Dwarfism, PituitaryPhase 4NCT01187550

Predictive Markers in Chinese Growth Hormone Deficiency (GHD) Children Treated With Saizen®

A Phase 4 study testing Recombinant human growth hormone (r-hGH) in people with Dwarfism, Pituitary, run by Merck KGaA, Darmstadt, Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is an open-label, prospective, multicentric, non-comparative, non-randomized Phase IV interventional study in which subjects pre-diagnosed with Growth Hormone Deficiency (GHD) were treated for 4 weeks with Saizen to compare the response between GHD children born appropriate for gestational age (AGA) and those… Read more about this study

Who is it for?
Any age
Conditions the study is for
Dwarfism, Pituitary
What is being tested?
Recombinant human growth hormone (r-hGH)
Type of treatment
Injection or infusion
How many people
214 participants
Where
No sites listed yet
Study dates
Mar 2007to Apr 2009
Phase
Phase 4
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male and female subjects with documented pre-established diagnosis of GHD with a GH peak response of <10 microgram/liter (mcg/L) with 2 GH stimulation tests, without priming with estradiol
  • Subjects with SGA defined as birth weight and/or length at least 2 standard deviations (SDs) below the mean for gestational age
  • Subjects with prepubertal status according to Tanner
  • Subjects with pre-established history of normal thyroid function or adequate substitution for at least 3 months
  • Subjects with weight for stature within the population specific normal range (>5th and <95th percentiles) for gender
  • Subjects with willingness and ability to comply with the protocol for the duration of the study
  • Subjects whose parents or guardians written informed consent given before any study-related procedure that was not part of the subjects normal medical care, with the understanding that the subject or parent/guardian might withdraw consent at any time without prejudice to future medical care. If the child was old enough to read and write, a separate assent form was given

Who cannot join

  • Subjects who acquired GHD due to central nervous system tumor, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
  • Subjects with previous treatment with GH, growth hormone releasing hormone (GHRH), anabolic steroids or any treatment affecting growth
  • Subjects who had previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution were also allowed if the condition and the treatment regimen had been stable for at least 3 months
  • Subjects with severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia
  • Subjects with chronic severe kidney disease
  • Subjects with chronic severe liver disease
Show all 15Show fewer
  • Subjects with chronic infectious disease
  • Subjects with acute or severe illness during the previous 6 months
  • Subjects with significant concomitant illness that would interfere with participation or assessment in this study
  • Subjects who had active malignancy (except non-melanomatous skin malignancies that had undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
  • Subjects with history or active idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri)
  • Subjects with diabetes mellitus type I \& II
  • Subjects with any autoimmune disease
  • Subjects who had previous screening failure in this study
  • Subjects who had used an investigational drug or participated in another clinical study within the last 3 months

About this study

Official title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient Pre-pubertal Children Treated With Saizen®

This is an open-label, prospective, multicentric, non-comparative, non-randomized Phase IV interventional study in which subjects pre-diagnosed with Growth Hormone Deficiency (GHD) were treated for 4 weeks with Saizen to compare the response between GHD children born appropriate for gestational age (AGA) and those born small for gestation age (SGA) after 4 weeks of Saizen therapy.

Read the detailed description

The response to growth hormone (GH) treatment, short-term as well as long-term, displays considerable inter individual variability. This is particularly evident for the endpoint of paediatric GH administration, that is (i.e.) the growth response, which is pronounced in children who are affected by GHD. This is an open-label, multicentric study in which subjects pre-diagnosed with GHD were treated for 4 weeks with Saizen. Two hundred fourteen GHD evaluable pre-pubertal subjects were planned to be recruited in approximately 9 sites in China. Demographic data, medical history, tanner stage, physical examination, body weight, height, bone age measurement, body mass index, review of baseline medications and procedures and blood sampling were performed at baseline visit, end of treatment visit (week 4) and at 4 week follow-up visit.

OBJECTIVES

Primary objective:

  • To compare the response between GHD children born AGA and those born SGA after 4 weeks of Saizen therapy

Secondary Objectives:

  • To explore the contribution of selected genes to the phenotype of GHD children
  • To explore the impact of gene polymorphisms on the levels of specific serum biomarkers in GHD children after 4 weeks of Saizen therapy
  • To explore the relationships between changes in gene expression and changes in serum biomarkers after 4 weeks of Saizen therapy and the spectrum of gene polymorphisms in GHD children
Being tested
Recombinant human growth hormone (r-hGH)
Keywords
Dwarfism, pituitary, Growth hormone
Medical subject headings
Dwarfism, Pituitary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Recombinant human growth hormone (r-hGH)Drug

    Also called Saizen

    Recombinant human growth hormone (r-hGH) administered at dose of 0.033 milligram/kilogram (mg/kg) body weight (0.1 International Unit [IU]/kg body weight) per day by subcutaneous injection.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4

    Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) was calculated as logarithm (log) 10 actual value of IGF-1 - log 10 (mean reference value of IGF-1) divided by log10 reference standard deviation of IGF-1.

    Measured: Baseline and Week 4

Secondary outcomes (5)

  1. Change From Baseline in Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels at Week 4

    Measured: Baseline and Week 4

  2. Change From Baseline in Fasting Glucose at Week 4

    Measured: Baseline and Week 4

  3. Change From Baseline in Fasting Insulin at Week 4

    Measured: Baseline and Week 4

  4. Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) Test at Week 4

    HOMA-IR is used to assess insulin resistance and calculated by an empirical mathematical formula based on fasting plasma glucose and fasting plasma insulin levels. HOMA-IR = fasting plasma insulin (picomole/liter [pmol/L]) * fasting plasma glucose (millimole/liter [mmol/L]) divided by 22.5.

    Measured: Baseline and Week 4

  5. Change From Baseline in Lipid Profile at Week 4

    Total cholesterol, high-density lipoprotein (HDL)-cholesterol, low-density lipoprotein (LDL)-cholesterol and triglycerides levels were evaluated.

    Measured: Baseline and Week 4

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Aug 2010 (estimated)
  2. Study startMar 2007
  3. Primary completionSep 2008
  4. Study completionApr 2009

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
214 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01187550
Sponsor's protocol ID
27709
Status last verified
Jan 2014
First submitted
22 Aug 2010
Last updated
13 Feb 2014

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Recombinant human growth hormone (r-hGH) in people with Dwarfism, Pituitary, 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 7 inclusion and 15 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 13 Feb 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?

Yes. Results are posted in the registry (first posted 7 Aug 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4 (time frame: Baseline and Week 4). 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 NCT01187550.

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 13 Feb 2014.

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