Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
Prader-Willi Syndrome
About this trial
This is an interventional treatment trial for Prader-Willi Syndrome
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.
Sites / Locations
- Kanagawa Children's Medical Center
- Osaka Women's and Children's Hospital
- Dokkyo Medical University Saitama Medical Center
- Hamamatsu University Hospital
- National Center for Child Health and Development
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
somatropin - GH naïve pediatric cohort
somatropin - GH treated pediatric cohort
somatropin - adult cohort
All participants will receive somatropin.
All participants will receive somatropin
All participants will receive somatropin