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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00256126




Registration number
NCT00256126
Ethics application status
Date submitted
18/11/2005
Date registered
21/11/2005
Date last updated
26/06/2018

Titles & IDs
Public title
Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®
Scientific title
A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children Treated With SAIZEN®
Secondary ID [1] 0 0
24531
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Growth Hormone Deficiency 0 0
Turner Syndrome 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Saizen
Treatment: Drugs - Saizen

Experimental: Turner Syndrome (TS) -

Experimental: Growth Hormone Deficiency (GHD) -


Treatment: Drugs: 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

Treatment: Drugs: 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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1
Timepoint [1] 0 0
Baseline, Month 1
Secondary outcome [1] 0 0
Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1
Timepoint [1] 0 0
Baseline, Month 1
Secondary outcome [2] 0 0
Change From Baseline in Fasting Glucose Levels at Month 1
Timepoint [2] 0 0
Baseline, Month 1
Secondary outcome [3] 0 0
Change From Baseline in Fasting Insulin Levels at Month 1
Timepoint [3] 0 0
Baseline, Month 1
Secondary outcome [4] 0 0
Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1
Timepoint [4] 0 0
Baseline, Month 1
Secondary outcome [5] 0 0
Change From Baseline in Bone Alkaline Phosphatase Levels at Month 1
Timepoint [5] 0 0
Baseline, Month 1

Eligibility
Key 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.
Minimum age
2 Years
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/05/2005
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/09/2007
Sample size
Target
Accrual to date
Final
318
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Local Medical Information Office - Sydney
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
Argentina
State/province [1] 0 0
Buenos Aires
Country [2] 0 0
Austria
State/province [2] 0 0
Vienna
Country [3] 0 0
Canada
State/province [3] 0 0
Mississauga
Country [4] 0 0
France
State/province [4] 0 0
Paris
Country [5] 0 0
Germany
State/province [5] 0 0
Munich
Country [6] 0 0
Italy
State/province [6] 0 0
Rome
Country [7] 0 0
Norway
State/province [7] 0 0
Oslo
Country [8] 0 0
Russian Federation
State/province [8] 0 0
Russia
Country [9] 0 0
Singapore
State/province [9] 0 0
Singapore
Country [10] 0 0
Spain
State/province [10] 0 0
Madrid
Country [11] 0 0
Sweden
State/province [11] 0 0
Stockholm
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Feltham

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Merck KGaA, Darmstadt, Germany
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00256126
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Responsible
Address 0 0
Merck KGaA, Darmstadt, Germany
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries