Dwarfism, Pituitary Clinical Trial
Official title:
A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient Pre-pubertal Children Treated With Saizen®
Verified date | January 2014 |
Source | Merck KGaA |
Contact | n/a |
Is FDA regulated | No |
Health authority | China: Ethics Committee |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 214 |
Est. completion date | April 2009 |
Est. primary completion date | September 2008 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A and older |
Eligibility |
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 |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Merck KGaA | Merck Serono Co., Ltd., China |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | Baseline and Week 4 | No |
Secondary | Change From Baseline in Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels at Week 4 | Baseline and Week 4 | No | |
Secondary | Change From Baseline in Fasting Glucose at Week 4 | Baseline and Week 4 | No | |
Secondary | Change From Baseline in Fasting Insulin at Week 4 | Baseline and Week 4 | No | |
Secondary | 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. | Baseline and Week 4 | No |
Secondary | 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. | Baseline and Week 4 | No |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT02314676 -
Phase IV Clinical Study of Pegylated Somatropin (PEG Somatropin) to Treat Growth Hormone Deficiency Children
|
Phase 4 | |
Completed |
NCT00102258 -
Role of Nutrition and Hormones in Boys With Disordered Growth
|
Phase 2 | |
Completed |
NCT00097513 -
National Cooperative Growth Study (NCGS) of Optimal Nutropin AQ and Nutropin Dosing in Pubertal Growth Hormone-Deficient (GHD) Patients
|
Phase 4 | |
Completed |
NCT00097526 -
Bone Mineral Density (BMD) in Adolescents With Growth Hormone Deficiency (GHD)
|
Phase 4 |