Phase 3
N=33
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
Prader-Willi Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04697381 ↗Enrolled (actual)
33
Serious AEs
12.1%
Results posted
Jun 2024
Primary outcome: Primary: 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
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- somatropin - GH naïve pediatric cohort (Biological); somatropin - GH treated cohort (Biological); somatropin - adult cohort (Biological)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort |
3.09 | — |
| PRIMARY Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort |
4.59; -1.34 | — |
| SECONDARY Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort |
2.03 | — |
| SECONDARY Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort |
3.32; 0.58 | — |
| SECONDARY Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort |
-3.09 | — |
| SECONDARY Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort |
-4.59; 1.34 | — |
| SECONDARY Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT) |
60.423; 102.222; -13.764; 4.825; -13.403; -4.040 | — |
| SECONDARY Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only |
2.38 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events |
5; 7; 19; 0; 1; 3 | — |
| SECONDARY Number of Participants With Laboratory Test Abnormalities |
5; 3; 16 | — |
| SECONDARY Bone Maturation |
4; 0 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Male or female participants with documentation of genetically confirmed diagnosis of PWS.
- No plan to initiate a new treatment that may affect the body composition, such as gonadal hormone replacement therapy.
- Currently on appropriate diet and exercise programs and willing to continue throughout the study period at the discretion of the investigator.
- 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.
- 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:
- 18 years or younger.
- Naïve to GH treatment.
- 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:
- 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.
- 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:
- 18 years of chronological age or older at Day 1 visit.
- Off from GH treatment for at least 1 year.
- Serum IGF-I level within +2 SDS, adjusted for age and sex.
Exclusion Criteria
- Participants with uncontrolled diabetes at the discretion of the investigator.
- Participants with malignant tumors.
- Participants with severe obesity or serious respiratory impairment.
- 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.
- 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).
- 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.
Data sourced from ClinicalTrials.gov (NCT04697381). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.