Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®
Condition: Growth Hormone Deficiency; Turner Syndrome
MeSH terms: Dwarfism, Pituitary; Turner Syndrome; Gonadal Dysgenesis; Syndrome
Intervention: Saizen; Saizen
Intervention description: 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; 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.
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 318.0
Sex: All
Minimum age: 2 Years
Maximum age: 16 Years
Healthy volunteers: No

Summary:

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

Eligibility 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.