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Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®

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ClinicalTrials.gov Identifier: NCT00256126
Recruitment Status : Completed
First Posted : November 21, 2005
Results First Posted : March 20, 2018
Last Update Posted : March 20, 2018
Sponsor:
Information provided by (Responsible Party):
Merck KGaA

Brief Summary:
The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency Turner Syndrome Drug: Saizen Phase 4

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 318 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children Treated With SAIZEN®
Actual Study Start Date : May 31, 2005
Actual Primary Completion Date : September 30, 2007
Actual Study Completion Date : September 30, 2007


Arm Intervention/treatment
Experimental: Turner Syndrome (TS) Drug: Saizen
Subjects with TS will receive SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month
Experimental: Growth Hormone Deficiency (GHD) Drug: Saizen
Subjects with GHD will receive SAIZEN® as subcutaneous injection at a dose of 0.035 mg/kg of body weight per day (within the recommended dosage 0.025-0.035 mg/kg body weight) for a period of 1 month.



Primary Outcome Measures :
  1. Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1 [ Time Frame: Baseline, Month 1 ]
    IGF-1 SDS was calculated using the Elmlinger reference method. Change in within subject IGF-1 levels (standard deviation scores) at Month 1 from Baseline was assessed. Descriptive statistics were determined for the Baseline and Month 1 assessments, and also for the level of change between these two assessments. If either the Baseline or Month 1 IGF-1 level was missing, then the within-subject change in IGF-1 was assumed to be missing.


Secondary Outcome Measures :
  1. Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1 [ Time Frame: Baseline, Month 1 ]
  2. Change From Baseline in Fasting Glucose Levels at Month 1 [ Time Frame: Baseline, Month 1 ]
  3. Change From Baseline in Fasting Insulin Levels at Month 1 [ Time Frame: Baseline, Month 1 ]
  4. Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1 [ Time Frame: Baseline, Month 1 ]
    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.

  5. Change From Baseline in Bone Alkaline Phosphatase Levels at Month 1 [ Time Frame: Baseline, Month 1 ]


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Ages Eligible for Study:   2 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • One of the following diagnoses and candidacy for SAIZEN® therapy:

A) GHD: documented pre-established diagnosis of GHD with a growth hormone (GH) peak response of <10 microgram per liter (mcg/L) with 2 GH stimulation tests, without priming with oestradiol.

B) Turner syndrome: documented pre-established diagnosis by karyotype.

  • Prepubertal status according to Tanner Pre-established history of normal thyroid function or adequate substitution for at least 3 months.
  • Weight for stature within the population specific normal range (>5th and <95th percentiles) for gender Willingness and ability to comply with the protocol for the duration of the study.
  • Parent's or guardian's written informed consent, given before any study related procedure that is not part of the subject's normal medical care, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough to read and write, a separate assent form will be given.

Exclusion Criteria:

  • Acquired GHD due to central nervous system tumour, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
  • Previous treatment with GH, growth hormone-releasing hormone (GHRH), anabolic steroids or any treatment affecting growth.
  • Previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution are also allowed if the condition and the treatment regimen have been stable for at least 3 months.
  • Severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia.
  • Chronic severe kidney disease.
  • Chronic severe liver disease.
  • Chronic infectious disease.
  • Acute or severe illness during the previous 6 months.
  • Significant concomitant illness that would interfere with participation or assessment in this study.
  • Active malignancy (except non-melanomatous skin malignancies that have undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
  • History or active Idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri).
  • Diabetes Mellitus type I & II.
  • Any autoimmune disease.
  • Previous screening failure in this study.
  • Use of an investigational drug or participation in another clinical study within the last three months.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00256126


Locations
Argentina
Local Medical Information Office
Buenos Aires, Argentina
Australia
Local Medical Information Office
Sydney, Australia
Austria
Local Medical Information Office
Vienna, Austria
Canada
Local Medical Information Office
Mississauga, Canada
France
Local Medical InformationOffice
Paris, France
Germany
Local Medical Information Office
Munich, Germany
Italy
Local Medical Information Office
Rome, Italy
Norway
Local Medical Information Office
Oslo, Norway
Russian Federation
Local Medical Information Office
Russia, Russian Federation
Singapore
Local Medical Information Office
Singapore, Singapore
Spain
Local Medical Information Office
Madrid, Spain
Sweden
Local Medical Information Office
Stockholm, Sweden
United Kingdom
Local Medical Information Office
Feltham, United Kingdom
Sponsors and Collaborators
Merck KGaA
Investigators
Study Director: Medical Responsible Merck KGaA

Additional Information:
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Merck KGaA
ClinicalTrials.gov Identifier: NCT00256126     History of Changes
Other Study ID Numbers: 24531
First Posted: November 21, 2005    Key Record Dates
Results First Posted: March 20, 2018
Last Update Posted: March 20, 2018
Last Verified: August 2017

Additional relevant MeSH terms:
Syndrome
Endocrine System Diseases
Dwarfism, Pituitary
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Disease
Pathologic Processes
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Disorders of Sex Development
Urogenital Abnormalities
Sex Chromosome Disorders of Sex Development
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Sex Chromosome Disorders
Chromosome Disorders
Genetic Diseases, Inborn