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

Primary Purpose

Dwarfism, Pituitary

Status

Completed

Phase

Phase 4

Locations

Study Type

Interventional

Intervention

Recombinant human growth hormone (r-hGH)

About this trial

This is an interventional treatment trial for Dwarfism, Pituitary focused on measuring Dwarfism, pituitary, Growth hormone

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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
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

Sites / Locations

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

Change From Baseline in Fasting Glucose at Week 4

Change From Baseline in Fasting Insulin at Week 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.

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.

Full Information

NCT ID

NCT01187550

First Posted

August 22, 2010

Last Updated

January 20, 2014

Sponsor

Merck KGaA, Darmstadt, Germany

Collaborators

Merck Serono Co., Ltd., China

1. Study Identification

Unique Protocol Identification Number

NCT01187550

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2014

Overall Recruitment Status

Completed

Study Start Date

March 2007 (undefined)

Primary Completion Date

September 2008 (Actual)

Study Completion Date

April 2009 (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Merck KGaA, Darmstadt, Germany

Collaborators

Merck Serono Co., Ltd., China

4. Oversight

5. Study Description

Brief 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 born small for gestation age (SGA) after 4 weeks of Saizen therapy.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dwarfism, Pituitary

Keywords

Dwarfism, pituitary, Growth hormone

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

214 (Actual)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Recombinant human growth hormone (r-hGH)

Other Intervention Name(s)

Saizen

Intervention Description

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.

Primary Outcome Measure Information:

Title

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

Description

Time Frame

Baseline and Week 4

Secondary Outcome Measure Information:

Title

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

Time Frame

Baseline and Week 4

Title

Change From Baseline in Fasting Glucose at Week 4

Time Frame

Baseline and Week 4

Title

Change From Baseline in Fasting Insulin at Week 4

Time Frame

Baseline and Week 4

Title

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

Description

Time Frame

Baseline and Week 4

Title

Change From Baseline in Lipid Profile at Week 4

Description

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

Time Frame

Baseline and Week 4

10. Eligibility

Sex

All

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Medical Responsible

Organizational Affiliation

Merck Serono Co., Ltd., China

Official's Role

Study Director

12. IPD Sharing Statement

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

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