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

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)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Biological: somatropin - GH naïve pediatric cohort; Biological: somatropin - GH treated cohort; Biological: somatropin - adult cohort

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Kanagawa
    • Yokohama, Kanagawa, Japan, 232-8555
      • Kanagawa Children's Medical Center
  • Osaka
    • Izumi, Osaka, Japan, 594-1101
      • Osaka Women's and Children's Hospital
  • Saitama
    • Koshigaya, Saitama, Japan, 343-8555
      • Dokkyo Medical University Saitama Medical Center
  • Shizuoka
    • Hamamatsu, Shizuoka, Japan, 431-3192
      • Hamamatsu University Hospital
  • Tokyo
    • Setagaya-ku, Tokyo, Japan, 157-8535
      • National Center for Child Health and Development

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: somatropin - GH naïve pediatric cohort; All participants will receive somatropin.	Biological: somatropin - GH naïve pediatric cohort; somatropin 0.245 mg/kg/week
Experimental: somatropin - GH treated pediatric cohort; All participants will receive somatropin	Biological: somatropin - GH treated cohort; somatropin 0.084 mg/kg/week
Experimental: somatropin - adult cohort; All participants will receive somatropin	Biological: somatropin - adult cohort; somatropin 0.084 mg/kg/week

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from baseline to Month 12 in lean body mass (%) measured by DEXA	52 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): February 6, 2021
• Primary Completion (Actual): December 6, 2022
• Study Completion (Estimated): May 16, 2024

Study Registration Dates

• First Submitted: January 4, 2021
• First Submitted That Met QC Criteria: January 4, 2021
• First Posted (Actual): January 6, 2021

Study Record Updates

• Last Update Posted (Actual): July 7, 2023
• Last Update Submitted That Met QC Criteria: July 5, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome
• Other Study ID Numbers: A6281323

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No