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

Prader-Willi Syndrome Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04697381
• Other study ID #: A6281323
• Status: Completed
• Phase: Phase 3
• Start date: February 9, 2021
• Est. completion date: April 15, 2024

Study information

• Verified date: June 2024
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 33
• Est. completion date: April 15, 2024
• Est. primary completion date: December 6, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years and older

Eligibility

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 naïve pediatric cohort, participants must meet criteria 6 to 8:

6. 18 years or younger.

7. Naïve 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.

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 Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

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

Locations

Country	Name	City	State
Japan	Hamamatsu University Hospital	Hamamatsu	Shizuoka
Japan	Osaka Women's and Children's Hospital	Izumi	Osaka
Japan	Dokkyo Medical University Saitama Medical Center	Koshigaya	Saitama
Japan	National Center for Child Health and Development	Setagaya-ku	Tokyo
Japan	Kanagawa Children's Medical Center	Yokohama	Kanagawa

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort	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.	Baseline, Month 12
Primary	Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort	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.	Baseline, Month 12
Secondary	Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort	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.	Baseline, Month 12
Secondary	Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort	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.	Baseline, Month 12
Secondary	Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort	Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / [lean body mass (kg) + body fat (kg)]*100.	Baseline, Month 12
Secondary	Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort	Body fat was assessed by DEXA scan. and calculated as body fat (%) = body fat (kg) / [lean body mass (kg) + body fat (kg)]*100.	Baseline, Month 12
Secondary	Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)	Adipose tissue distribution was measured by abdominal CT. Areas of subcutaneous adipose tissue (SAT) (centimeter square [cm^2]), visceral adipose tissue (VAT) (cm^2) were measured at the level of the umbilicus by abdominal CT.	Baseline, Month 12
Secondary	Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only	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.	Baseline, Month 6
Secondary	Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events	An adverse event was any untoward medical occurrence in administered medicinal product, event need not necessarily have a causal relationship with product treatment or usage. A serious adverse event was any untoward medical occurrence in a participant administered a medicinal or nutritional product (including pediatric formulas) at any dose that: resulted in death; was life-threatening; required inpatient hospitalization or prolongation of hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); resulted in congenital anomaly/birth defect. Treatment emergent AEs were events emerged during treatment period and were absent before treatment or that worsened relative to pretreatment state.	From dose 1 up to 28 days after end of study treatment (maximum treatment period of 12 months; maximum duration for AEs collection of 13 months)
Secondary	Number of Participants With Laboratory Test Abnormalities	Criteria for abnormal laboratory values for chemistry parameters: alanine aminotransferase, alkaline phosphatase greater than (>) 3.0*upper limit of normal (ULN), albumin > 1.2*ULN, urea nitrogen millimoles per liter (mmol/L) > 1.3*ULN, HDL cholesterol mmol/L less than (<) 0.8* lower limit of normal (LLN), LDL cholesterol mmol/L >1.2*ULN, triglycerides mmol/L > 1.3*ULN, thyrotropin milliunits per liter (mU/L) <0.8*LLN and >1.2*ULN, glucose (mmol/L) >1.5*ULN, hemoglobin A1C liter of cells per liter of blood (L/L) >1.3*ULN.	12 months
Secondary	Bone Maturation	Bone maturation is the process whereby the tissue undergoes changes from the embryonic rudiment of bone to the adult form. Bone maturation was calculated as bone age divided by chronological age. Participants with bone maturation value greater than 1 is presented in this outcome measure.	12 months

External Links

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