NCT04697381已完成3 期
A PHASE 3 MULTICENTER, OPEN LABEL, MULTI COHORT STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SOMATROPIN IN JAPANESE PARTICIPANTS WITH PRADER-WILLI SYNDROME (PWS)
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 33
- 试验地点
- 5
- 主要终点
- Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
somatropin - GH naïve pediatric cohort
Experimental
All participants will receive somatropin.
干预措施: somatropin - GH naïve pediatric cohort (Biological)
somatropin - GH treated pediatric cohort
Experimental
All participants will receive somatropin
干预措施: somatropin - GH treated cohort (Biological)
somatropin - adult cohort
Experimental
All participants will receive somatropin
干预措施: somatropin - adult cohort (Biological)
结局指标
主要结局
Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
时间窗: Baseline, Month 12
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass (kg) / (lean body mass \[kg\] + fat mass \[kg\])\*100.
Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort
时间窗: Baseline, Month 12
Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass kilogram (kg) / (lean body mass \[kg\] + fat mass \[kg\]) \*100.
次要结局
- Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort(Baseline, Month 12)
- Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort(Baseline, Month 12)
- Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only(Baseline, Month 6)
- Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort(Baseline, Month 12)
- Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort(Baseline, Month 12)
- Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)(Baseline, Month 12)
- Bone Maturation(12 months)
- Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events(From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months))
- Number of Participants With Laboratory Test Abnormalities(From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months))
研究者
研究点 (5)
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