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

Korábbi kezelés

• 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).

Más betegség

• Participants with uncontrolled diabetes at the discretion of the investigator.
• Participants with malignant tumors.
• Participants with severe obesity or serious respiratory impairment.

Laborértékek

• 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.

Egyéb

• 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.

• 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.