CompletedPhase 3

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

Japan33 participantsStarted 2021-02-09

Plain-language 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.

Who can participate

Age range0 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.
✓. 18 years or younger.
✓. Naïve to GH treatment.
✓. Tanner stage 1 (for testes in males, for breasts in females).

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.

What they're measuring

Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort

Timeframe: Baseline, Month 12

Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort

Timeframe: Baseline, Month 12

Trial details

NCT IDNCT04697381

SponsorPfizer

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-12-06

Results submitted2023-11-29

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