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

JRCT ID: jRCT2031200351

Registered date:08/02/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Prader-Willi Syndrome
• Date of first enrollment: 06/02/2021
• Target sample size: 30
• Study type: Interventional

Intervention(s)

Biological: somatropin - GH naive pediatric cohort
somatropin 0.245 mg/kg/week
Biological: somatropin - GH treated cohort
somatropin 0.084 mg/kg/week
Biological: somatropin - adult cohort
somatropin 0.084 mg/kg/week

Outcome(s)

Primary Outcome

Primary Outcome Measures :
1.Change from baseline to Month 12 in lean body mass (%) measured by DEXA [ Time Frame: 52 weeks ]

Secondary Outcome

Secondary Outcome Measures :
1.Change from baseline to Month 12 in lean body mass (%) measured by BIA [ Time Frame: 52 weeks ]
2.Change from baseline to Month 12 in body fat (%) measured by DEXA [ Time Frame: 52 weeks ]
3.Change from baseline to Month 12 in adipose tissue distribution measured by abdominal CT [ Time Frame: 52 weeks ]
4.Change from baseline to Month 6 in lean body mass (%) measured by DEXA (adult cohort only) [ Time Frame: 26 weeks ]
5.Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) [ Time Frame: Baseline up to 50 months ]
6.Number of Participants With Treatment Emergent Treatment-Related Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 50 months ]
7.Number of Participants With Clinically Significant Change From Baseline in Laboratory Abnormalities [ Time Frame: Baseline up to 48 months ]
8.Bone maturation [ Time Frame: 52 weeks ]

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: Not applicable
• Gender: Both

Include criteria

Inclusion Criteria:
1.Male or female participants with documentation of genetically confirmed diagnosis of PWS.
2.No plan to initiate a new treatment that may affect the body composition, such as gonadal hormone replacement therapy.
3.Currently on appropriate diet and exercise programs and willing to continue throughout the study period at the discretion of the investigator.
4.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.
5.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 naive pediatric cohort, participants must meet criteria 6 to 8:
6.18 years or younger.
7.Naive to GH treatment.
8.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:
9.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.
10.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:
11.18 years of chronological age or older at Day 1 visit.
12.Off from GH treatment for at least 1 year.
13.Serum IGF-I level within +2 SDS, adjusted for age and sex.

Exclude criteria

Exclusion Criteria:
1.Participants with uncontrolled diabetes at the discretion of the investigator.
2.Participants with malignant tumors.
3.Participants with severe obesity or serious respiratory impairment.
4.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.
5.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).
6.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.

Related Information

• Primary Sponsor: Kawai Norisuke
• Secondary ID(s): NCT04697381