Trial Outcomes & Findings for Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS (NCT NCT04697381)

Last Updated: 2026-01-14

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

33 participants

Primary outcome timeframe

Baseline, Month 12

Results posted on

2026-01-14

Participant Flow

Participants diagnosed with Prader-Willi syndrome (PWS ) received somatropin, a recombinant human growth hormone (r-hGH) in this study.

This study had 3 cohorts (GH naive pediatric cohort, GH treated pediatric cohort and adult cohort).

Participant milestones

Participant milestones
Measure	GH Naive Pediatric Cohort Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 milligram per kilogram per week (mg/kg/week) subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and insulin-like growth factor 1 (IGF-1) levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Treatment Period STARTED	6	7	20
Treatment Period COMPLETED	6	7	20
Treatment Period NOT COMPLETED	0	0	0
Extension Period STARTED	6	7	20
Extension Period COMPLETED	6	7	14
Extension Period NOT COMPLETED	0	0	6

Reasons for withdrawal

Reasons for withdrawal
Measure	GH Naive Pediatric Cohort Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 milligram per kilogram per week (mg/kg/week) subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and insulin-like growth factor 1 (IGF-1) levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Extension Period Adverse Event	0	0	3
Extension Period Physician Decision	0	0	2
Extension Period Other	0	0	1

Baseline Characteristics

Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=7 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort n=20 Participants Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Total n=33 Participants Total of all reporting groups
Age, Customized Less than (<6) years	3 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	3 Participants n=78 Participants
Age, Customized 6-12 years	3 Participants n=14 Participants	1 Participants n=10 Participants	0 Participants n=24 Participants	4 Participants n=78 Participants
Age, Customized 13-17 years	0 Participants n=14 Participants	5 Participants n=10 Participants	0 Participants n=24 Participants	5 Participants n=78 Participants
Age, Customized 18-44 years	0 Participants n=14 Participants	1 Participants n=10 Participants	20 Participants n=24 Participants	21 Participants n=78 Participants
Age, Customized More than equal to (>=) 45 years	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Sex: Female, Male Female	2 Participants n=14 Participants	3 Participants n=10 Participants	13 Participants n=24 Participants	18 Participants n=78 Participants
Sex: Female, Male Male	4 Participants n=14 Participants	4 Participants n=10 Participants	7 Participants n=24 Participants	15 Participants n=78 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=14 Participants	7 Participants n=10 Participants	20 Participants n=24 Participants	33 Participants n=78 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Race (NIH/OMB) Asian	6 Participants n=14 Participants	7 Participants n=10 Participants	20 Participants n=24 Participants	33 Participants n=78 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Race (NIH/OMB) Black or African American	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Race (NIH/OMB) White	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Race (NIH/OMB) More than one race	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants	0 Participants n=78 Participants

PRIMARY outcome

Timeframe: Baseline, Month 12

Population: Efficacy evaluable set included all participants assigned to study intervention and who took at least one dose of study intervention and had at least one efficacy evaluation. Here "Number of Participants Analyzed" signifies participants evaluable for this outcome measure and this time point.

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=19 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort	3.09 Percentage of body mass Interval 1.85 to 4.33	—	—

PRIMARY outcome

Timeframe: Baseline, Month 12

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=6 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort	4.59 Percentage of body mass Standard Deviation 4.490	-1.34 Percentage of body mass Standard Deviation 3.238	—

SECONDARY outcome

Timeframe: Baseline, Month 12

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=19 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort	2.03 Percentage of body mass Interval -0.67 to 4.73	—	—

SECONDARY outcome

Timeframe: Baseline, Month 12

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=5 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort	3.32 Percentage of body mass Standard Deviation 3.867	0.58 Percentage of body mass Standard Deviation 4.711	—

SECONDARY outcome

Timeframe: Baseline, Month 12

Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / \[lean body mass (kg) + body fat (kg)\]\*100.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=19 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort	-3.09 Percentage of body fat Interval -4.33 to -1.85	—	—

SECONDARY outcome

Timeframe: Baseline, Month 12

Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / \[lean body mass (kg) + body fat (kg)\]\*100.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=6 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort	-4.59 Percentage of body fat Standard Deviation 4.490	1.34 Percentage of body fat Standard Deviation 3.238	—

SECONDARY outcome

Timeframe: Baseline, Month 12

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=6 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort n=19 Participants Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT) Change at Month 12, SAT	60.423 Centimeter square (cm^2) Standard Deviation 138.2001	102.222 Centimeter square (cm^2) Standard Deviation 211.9111	-13.764 Centimeter square (cm^2) Standard Deviation 31.0212
Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT) Change at Month 12, VAT	4.825 Centimeter square (cm^2) Standard Deviation 13.5401	-13.403 Centimeter square (cm^2) Standard Deviation 28.5490	-4.040 Centimeter square (cm^2) Standard Deviation 16.8243

SECONDARY outcome

Timeframe: Baseline, Month 6

Population: Efficacy evaluable set included all participants assigned to study intervention and who took at least one dose of study intervention and had at least one efficacy evaluation.

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=20 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only	2.38 Percentage of body mass Interval 1.3 to 3.46	—	—

SECONDARY outcome

Timeframe: From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months)

Population: Full analysis set (FAS) included all participants assigned to study intervention and who took at least one dose of study intervention.

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

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=7 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort n=20 Participants Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events TEAEs	5 Participants	7 Participants	19 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Treatment Emergent SAEs	0 Participants	1 Participants	3 Participants

SECONDARY outcome

Timeframe: From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months)

Population: FAS included all participants assigned to study intervention and who took at least one dose of study intervention.

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=7 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort n=20 Participants Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Number of Participants With Laboratory Test Abnormalities	5 Participants	3 Participants	16 Participants

SECONDARY outcome

Timeframe: 12 months

Population: FAS included all participants assigned to study intervention and who took at least one dose of study intervention.

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.

Outcome measures

Outcome measures
Measure	GH Naive Pediatric Cohort n=6 Participants Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=7 Participants Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Bone Maturation	4 Participants	0 Participants	—

Adverse Events

GH Naive Pediatric Cohort

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

GH Treated Pediatric Cohort

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Adult Cohort

Serious events: 3 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	GH Naive Pediatric Cohort n=6 participants at risk Participants 18 years or younger, naive to GH treatment, received somatropin 0.245 mg/kg/week subcutaneously. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	GH Treated Pediatric Cohort n=7 participants at risk Participants 18 years or younger, who continued GH treatment for at least 2 years with stable dose for the last 6 months and were on GH at time of inclusion, received somatropin 0.084 mg/kg/week subcutaneously. The dosage was adjusted according to participants symptoms and IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.	Adult Cohort n=20 participants at risk Adult participants who were off from GH treatment for at least 1 year, initially received somatropin 0.042 mg/kg/week subcutaneously. Dose was titrated up to 0.084 mg/kg/week from Month 1 visit. The dosage was adjusted according to participants symptoms and serum IGF-1 levels with maximum dose up to 1.6 mg/day. Treatment period was of 12 months and extension period was of 36 months. Participants were followed up to 28 days.
Injury, poisoning and procedural complications Ankle fracture	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	14.3% 1/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.
Injury, poisoning and procedural complications Brain contusion	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	5.0% 1/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.
Injury, poisoning and procedural complications Skull fractured base	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	5.0% 1/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.
Vascular disorders Hypertension	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	5.0% 1/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.
Cardiac disorders Sinus node dysfunction	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	5.0% 1/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.
Nervous system disorders Seizure	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	5.0% 1/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.
Psychiatric disorders Affect lability	0.00% 0/6 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	0.00% 0/7 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.	5.0% 1/20 • From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) Same event may appear as both non-SAE and SAE. What is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other participant, or a participant may have experienced both SAE and non-SAE. Analysis population include all participants assigned to study intervention and who took at least one dose of study intervention.

Other adverse events

Additional Information

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Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
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Restriction type: OTHER