This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
33
somatropin 0.245 mg/kg/week
somatropin 0.084 mg/kg/week
somatropin 0.084 mg/kg/week
Kanagawa Children's Medical Center
Yokohama, Kanagawa, Japan
Osaka Women's and Children's Hospital
Izumi, Osaka, Japan
Dokkyo Medical University Saitama Medical Center
Koshigaya, Saitama, Japan
Hamamatsu University Hospital
Hamamatsu, Shizuoka, Japan
Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass kilogram (kg) / (lean body mass \[kg\] + fat mass \[kg\]) \*100.
Time frame: Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass (kg) / (lean body mass \[kg\] + fat mass \[kg\])\*100.
Time frame: Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass (kg) / (lean body mass \[kg\] + fat mass \[kg\])\*100.
Time frame: Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass (kg) / (lean body mass \[kg\] + fat mass \[kg\])\*100.
Time frame: Baseline, Month 12
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort
Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / \[lean body mass (kg) + body fat (kg)\]\*100.
Time frame: Baseline, Month 12
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
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National Center for Child Health and Development
Setagaya-ku, Tokyo, Japan
Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / \[lean body mass (kg) + body fat (kg)\]\*100.
Time frame: Baseline, Month 12
Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)
Adipose tissue distribution was measured by abdominal CT. Areas of subcutaneous adipose tissue (SAT) (centimeter square \[cm\^2\]), visceral adipose tissue (VAT) (cm\^2) were measured at the level of the umbilicus by abdominal CT.
Time frame: Baseline, Month 12
Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass (kg) / (lean body mass \[kg\] + fat mass \[kg\])\*100.
Time frame: Baseline, Month 6
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
An adverse event was any untoward medical occurrence in administered medicinal product, event need not necessarily have a causal relationship with product treatment or usage. A serious adverse event was any untoward medical occurrence in a participant administered a medicinal or nutritional product (including pediatric formulas) at any dose that: resulted in death; was life-threatening; required inpatient hospitalization or prolongation of hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); resulted in congenital anomaly/birth defect. Treatment emergent AEs were events emerged during treatment period and were absent before treatment or that worsened relative to pretreatment state. TEAEs consist of SAEs and non-SAEs
Time frame: From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months)
Number of Participants With Laboratory Test Abnormalities
Criteria for abnormal laboratory values for chemistry parameters: alanine aminotransferase, alkaline phosphatase greater than (\>) 3.0\*upper limit of normal (ULN), albumin \> 1.2\*ULN, urea nitrogen millimoles per liter (mmol/L) \> 1.3\*ULN, HDL cholesterol mmol/L less than (\<) 0.8\* lower limit of normal (LLN), LDL cholesterol mmol/L \>1.2\*ULN, triglycerides mmol/L \> 1.3\*ULN, thyrotropin milliunits per liter (mU/L) \<0.8\*LLN and \>1.2\*ULN, glucose (mmol/L) \>1.5\*ULN, hemoglobin A1C liter of cells per liter of blood (L/L) \>1.3\*ULN.
Time frame: From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months)
Bone Maturation
Bone maturation is the process whereby the tissue undergoes changes from the embryonic rudiment of bone to the adult form. Bone maturation was calculated as bone age divided by chronological age. Participants with bone maturation value greater than 1 is presented in this outcome measure.
Time frame: 12 months