Primary outcomeLeast squares mean· Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort
| Group | Value (Percentage of body mass) |
|---|---|
| Adult Cohort | 3.09 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataStudy of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
NCTNCT04697381 (opens ClinicalTrials.gov in a new tab)Other idA6281323Other id2024-000101-32Other idNCT04697381
Overview
- Status
- Completed
- Phase
- Phase 3
- Enrollment
- 33 participants (actual)
- Sponsor
- Pfizer · Industry
- Design
- Not randomized · Open label (no masking)
- Countries
1 country
Japan
- Start
- 9 Feb 2021
- Primary completion
- 6 Dec 2022
- Completion
- 15 Apr 2024
- First posted
- 6 Jan 2021
- Last update posted
- 14 Jan 2026
- Conditions
- Prader-Willi Syndrome
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
20212022202320242025
- Ph 3
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
NCT046973819 Feb 2021 to 15 Apr 2024
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- somatropin - GH naïve pediatric cohortBiological
- somatropin - GH treated cohortBiological
- somatropin - adult cohortBiological
Arms3
Experimental
somatropin - GH naïve pediatric cohort
All participants will receive somatropin.
Experimental
somatropin - GH treated pediatric cohort
All participants will receive somatropin
Experimental
somatropin - adult cohort
All participants will receive somatropin
Primary outcomes2
Outcomes the sponsor listed as primary.
Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort
Time frame · Baseline, Month 12
Registry description
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.
Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
Time frame · Baseline, Month 12
Registry description
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.
Secondary outcomes(9)
Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort
Time frame · Baseline, Month 12
Registry description
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.
Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort
Time frame · Baseline, Month 12
Registry description
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.
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort
Time frame · Baseline, Month 12
Registry description
Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / \[lean body mass (kg) + body fat (kg)\]\*100.
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
Time frame · Baseline, Month 12
Registry description
Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / \[lean body mass (kg) + body fat (kg)\]\*100.
Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)
Time frame · Baseline, Month 12
Registry description
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.
Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only
Time frame · Baseline, Month 6
Registry description
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.
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Time frame · From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months)
Registry description
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
Number of Participants With Laboratory Test Abnormalities
Time frame · From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months)
Registry description
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.
Bone Maturation
Time frame · 12 months
Registry description
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.
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Primary outcomeMean· Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
| Group | Value (Percentage of body mass) | Spread |
|---|---|---|
| GH Naive Pediatric Cohort | 4.59 | 4.490 |
| GH Treated Pediatric Cohort | -1.34 | 3.238 |
Secondary and other outcome results(6)
Secondary outcomeLeast squares mean· Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort
| Group | Value (Percentage of body mass) |
|---|---|
| Adult Cohort | 2.03 |
Secondary outcomeMean· Baseline, Month 12
Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort
| Group | Value (Percentage of body mass) | Spread |
|---|---|---|
| GH Naive Pediatric Cohort | 3.32 | 3.867 |
| GH Treated Pediatric Cohort | 0.58 | 4.711 |
Secondary outcomeLeast squares mean· Baseline, Month 12
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort
| Group | Value (Percentage of body fat) |
|---|---|
| Adult Cohort | -3.09 |
Secondary outcomeMean· Baseline, Month 12
Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
| Group | Value (Percentage of body fat) | Spread |
|---|---|---|
| GH Naive Pediatric Cohort | -4.59 | 4.490 |
| GH Treated Pediatric Cohort | 1.34 | 3.238 |
Secondary outcomeMean· Baseline, Month 12
Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)
| Group | Value (Centimeter square (cm^2)) | Spread |
|---|---|---|
| GH Naive Pediatric Cohort | 60.423 | 138.2001 |
| GH Treated Pediatric Cohort | 102.222 | 211.9111 |
| Adult Cohort | -13.764 | 31.0212 |
Secondary outcomeLeast squares mean· Baseline, Month 6
Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only
| Group | Value (Percentage of body mass) |
|---|---|
| Adult Cohort | 2.38 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| GH Naive Pediatric CohortParticipants 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. | 0 / 6 | 0 / 6 | 5 / 6 |
| GH Treated Pediatric CohortParticipants 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. | 0 / 7 | 1 / 7 | 7 / 7 |
| Adult CohortAdult 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. | 0 / 20 | 3 / 20 | 19 / 20 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies1
Studies that cite this trial.
More studies
1 study on this page
Indexing hint: Clinical trial · 2025
Improvement in body composition of Japanese participants with Prader-Willi syndrome following somatropin treatment: an open-label, multi cohort Phase 3 study.
Kawai et al. 2025Endocrine journalcited by 1
PMID 40436776PMC12340246DOI 10.1507/endocrj.ej24-0659PUB-40436776
Free to read · publisher termsCC BY-NC-ND
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 0 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
1. Male or female participants with documentation of genetically confirmed diagnosis of PWS.
2. No plan to initiate a new treatment that may affect the body composition, such as gonadal hormone replacement therapy.
3. Currently on appropriate diet and exercise programs and willing to continue throughout the study period at the discretion of the investigator.
4. Participants, and if required by local/site regulations their parent(s)/legal guardian(s) must be willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
5. Evidence of a personally signed and dated ICD (and written assent where applicable based on age and country regulation) indicating that the participant or a legally acceptable representative/parent(s)/legal guardian has been informed of all pertinent aspects of the study. Refer to Appendix 1 for the detailed process of obtaining consent.
For inclusion of GH naïve pediatric cohort, participants must meet criteria 6 to 8:
6. 18 years or younger.
7. Naïve to GH treatment.
8. Tanner stage 1 (for testes in males, for breasts in females).
For inclusion of GH treated pediatric cohort, participants must meet criteria 9 and 10:
9. Continued GH treatment for at least 2 years with stable dose for the last 6 months and being on GH at time of inclusion. The recent dose should be higher than 0.084 mg/kg/week.
10. Participants who are about to complete GH treatment for his/her short stature (eg, due to meeting the treatment stopping criteria defined as a height SDS more than -2.5 for Japanese adult standards).
For inclusion of adult cohort, participants must meet criteria 11 to 13:
11. 18 years of chronological age or older at Day 1 visit.
12. Off from GH treatment for at least 1 year.
13. Serum IGF-I level within +2 SDS, adjusted for age and sex.
Exclusion Criteria:
1. Participants with uncontrolled diabetes at the discretion of the investigator.
2. Participants with malignant tumors.
3. Participants with severe obesity or serious respiratory impairment.
4. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
5. Previous administration with an investigational drug within 30 days (or as determined by the local requirement) or 5 half- lives preceding the first dose of study intervention used in this study (whichever is longer).
6. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.
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