Trial · NCT04697381
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
NCT04697381, Phase 3, Completed, Pfizer; results first posted 2024-06-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS. Trials, record NCT04697381. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04697381
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
- NCT number
- NCT04697381
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 33 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Prader-Willi Syndrome
- Interventions
- somatropin - GH naïve pediatric cohort, somatropin - GH treated cohort, somatropin - adult cohort
- First posted
- 2021-01-06
- Last updated
- 2026-01-14
- Other registries' IDs
- eudract:2024-000101-32
- Results first posted
- 2024-06-05
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort | 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 kilogram (kg) / (lean body mass \[kg\] + fat mass \[kg\]) \*100. |
| Primary | Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | 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. |
| Secondary | Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort | 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. |
| Secondary | Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort | 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. |
| Secondary | Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort | 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. |
| Secondary | Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | 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. |
| Secondary | Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT) | 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. |
| Secondary | Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only | Baseline, Month 6 | 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 | Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) | 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 |
| Secondary | Number of Participants With Laboratory Test Abnormalities | From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) | 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. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort | Baseline, Month 12 | 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. | Percentage of body mass | LEAST_SQUARES_MEAN |
| PRIMARY | Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | Baseline, Month 12 | 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. | Percentage of body mass | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort | Baseline, Month 12 | 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. | Percentage of body mass | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort | Baseline, Month 12 | 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. | Percentage of body mass | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort | Baseline, Month 12 | 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. | Percentage of body fat | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | Baseline, Month 12 | 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. | Percentage of body fat | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT) | Baseline, Month 12 | 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. | Centimeter square (cm^2) | MEAN |
| SECONDARY | Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only | Baseline, Month 6 | 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. | Percentage of body mass | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) | Full analysis set (FAS) included all participants assigned to study intervention and who took at least one dose of study intervention. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Laboratory Test Abnormalities | From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months) | FAS included all participants assigned to study intervention and who took at least one dose of study intervention. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group27
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort | Adult Cohort | 3.09 | 95% Confidence Interval 1.85 to 4.33 | Percentage of body mass | |
| Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | GH Naive Pediatric Cohort | 4.59 | Standard Deviation 4.490 | Percentage of body mass | |
| Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | GH Treated Pediatric Cohort | -1.34 | Standard Deviation 3.238 | Percentage of body mass | |
| Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort | Adult Cohort | 2.03 | 95% Confidence Interval -0.67 to 4.73 | Percentage of body mass | |
| Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort | GH Naive Pediatric Cohort | 3.32 | Standard Deviation 3.867 | Percentage of body mass | |
| Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort | GH Treated Pediatric Cohort | 0.58 | Standard Deviation 4.711 | Percentage of body mass | |
| Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort | Adult Cohort | -3.09 | 95% Confidence Interval -4.33 to -1.85 | Percentage of body fat | |
| Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | GH Naive Pediatric Cohort | -4.59 | Standard Deviation 4.490 | Percentage of body fat | |
| Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort | GH Treated Pediatric Cohort | 1.34 | Standard Deviation 3.238 | Percentage of body fat | |
| Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT) | GH Naive Pediatric Cohort | Change at Month 12, SAT | 60.423 | Standard Deviation 138.2001 | Centimeter square (cm^2) |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period | STARTED | GH Naive Pediatric Cohort | 6 | |
| Treatment Period | STARTED | GH Treated Pediatric Cohort | 7 | |
| Treatment Period | STARTED | Adult Cohort | 20 | |
| Treatment Period | COMPLETED | GH Naive Pediatric Cohort | 6 | |
| Treatment Period | COMPLETED | GH Treated Pediatric Cohort | 7 | |
| Treatment Period | COMPLETED | Adult Cohort | 20 | |
| Treatment Period | NOT COMPLETED | GH Naive Pediatric Cohort | 0 | |
| Treatment Period | NOT COMPLETED | GH Treated Pediatric Cohort | 0 | |
| Treatment Period | NOT COMPLETED | Adult Cohort | 0 | |
| Extension Period | STARTED | GH Naive Pediatric Cohort | 6 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events342
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Ear pain | Ear and labyrinth disorders | GH Naive Pediatric Cohort | 1 | 6 | |
| other | Ear pain | Ear and labyrinth disorders | GH Treated Pediatric Cohort | 0 | 7 | |
| other | Ear pain | Ear and labyrinth disorders | Adult Cohort | 1 | 20 | |
| other | Vertigo | Ear and labyrinth disorders | GH Naive Pediatric Cohort | 0 | 6 | |
| other | Vertigo | Ear and labyrinth disorders | GH Treated Pediatric Cohort | 0 | 7 | |
| other | Vertigo | Ear and labyrinth disorders | Adult Cohort | 2 | 20 | |
| other | Abdominal pain | Gastrointestinal disorders | GH Naive Pediatric Cohort | 2 | 6 | |
| other | Abdominal pain | Gastrointestinal disorders | GH Treated Pediatric Cohort | 0 | 7 | |
| other | Abdominal pain | Gastrointestinal disorders | Adult Cohort | 1 | 20 | |
| other | Anal prolapse | Gastrointestinal disorders | GH Naive Pediatric Cohort | 0 | 6 |
1 to 10 of 342Source ClinicalTrials.gov
See all 342 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | GH Naive Pediatric Cohort | 0 | 6 |
| Total, all-cause mortality | GH Treated Pediatric Cohort | 0 | 7 |
| Total, all-cause mortality | Adult Cohort | 0 | 20 |
| Total, other adverse events | GH Naive Pediatric Cohort | 5 | 6 |
| Total, other adverse events | GH Treated Pediatric Cohort | 7 | 7 |
| Total, other adverse events | Adult Cohort | 19 | 20 |
| Total, serious adverse events | GH Naive Pediatric Cohort | 0 | 6 |
| Total, serious adverse events | GH Treated Pediatric Cohort | 1 | 7 |
| Total, serious adverse events | Adult Cohort | 3 | 20 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40436776 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| GH Naive Pediatric Cohort vs GH Treated Pediatric Cohort: Bone Maturation |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04697381
GET https://conductscience.org/api/trials/v1/NCT04697381/results?page=2