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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00256126
• Date of registration: 18/11/2005
• Prospective Registration: No
• Primary sponsor: Merck KGaA, Darmstadt, Germany
• 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®
• Date of first enrolment: May 31, 2005
• Target sample size: 318
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00256126
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Argentina, Australia, Austria, Canada, France, Germany, Italy, Norway, Russian Federation, Singapore, Spain, Sweden, United Kingdom

Contacts

• Name: Medical Responsible
• Affiliation: Merck KGaA, Darmstadt, Germany

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 16 Years
Gender: All

Health Condition(s) or Problem(s) studied

Turner Syndrome

Growth Hormone Deficiency

Intervention(s)

Drug: Saizen

Primary Outcome(s)

Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1 [Time Frame: Baseline, Month 1]

Secondary Outcome(s)

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

Change From Baseline in Fasting Insulin Levels at Month 1 [Time Frame: Baseline, Month 1]

Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1 [Time Frame: Baseline, Month 1]

Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1 [Time Frame: Baseline, Month 1]

Change From Baseline in Fasting Glucose Levels at Month 1 [Time Frame: Baseline, Month 1]

Secondary ID(s)

24531

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 20/03/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00256126